Donald B. Kohn, MD - Publications

Affiliations: 
MIMG University of California, Los Angeles, Los Angeles, CA 
Area:
Stem cell gene therapy
Website:
http://www.mimg.ucla.edu/faculty/kohn/index.html

432 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2024 Mudde AC, Kuo CY, Kohn DB, Booth C. What a Clinician Needs to Know about Genome Editing: Status and Opportunities for Inborn Errors of Immunity. The Journal of Allergy and Clinical Immunology. in Practice. PMID 38246560 DOI: 10.1016/j.jaip.2024.01.019  0.344
2023 Kohn DB. Gene therapy for adenosine deaminase severe combined immune deficiency-An unexpected journey of four decades. Immunological Reviews. PMID 38033164 DOI: 10.1111/imr.13293  0.403
2023 Kohn DB, Chen YY, Spencer MJ. Successes and challenges in clinical gene therapy. Gene Therapy. 30: 738-746. PMID 37935854 DOI: 10.1038/s41434-023-00390-5  0.432
2023 Frost IM, Mendoza AM, Chiou TT, Kim P, Aizenberg J, Kohn DB, De Oliveira SN, Weiss PS, Jonas SJ. Fluorinated Silane-Modified Filtroporation Devices Enable Gene Knockout in Human Hematopoietic Stem and Progenitor Cells. Acs Applied Materials & Interfaces. PMID 37616579 DOI: 10.1021/acsami.3c07045  0.36
2023 Bell JA, Collon K, Mayfield C, Gallo MC, Chang SW, Sugiyama O, Tang AH, Hollis R, Chopra S, Kohn DB, Lieberman JR. Biodistribution of lentiviral transduced adipose-derived stem cells for "ex-vivo" regional gene therapy for bone repair. Gene Therapy. PMID 37568039 DOI: 10.1038/s41434-023-00415-z  0.324
2023 Segura EER, Ayoub PG, Hart KL, Kohn DB. Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials. Viruses. 15. PMID 36992422 DOI: 10.3390/v15030713  0.384
2023 McAuley GE, Yiu G, Chang PC, Newby GA, Campo-Fernandez B, Fitz-Gibbon ST, Wu X, Kang SL, Garibay A, Butler J, Christian V, Wong RL, Everette KA, Azzun A, Gelfer H, ... ... Kohn DB, et al. Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing. Cell. PMID 36944331 DOI: 10.1016/j.cell.2023.02.027  0.425
2023 McDonald CL, Qasba P, Anderson DG, Bao G, Colvin RA, Kohn DB, Malik P, Mitchell MJ, Pu WT, Rawlings DJ, Williams DA, Flotte TR. Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. Human Gene Therapy. 34: 83-89. PMID 36794978 DOI: 10.1089/hum.2022.29233.tga  0.469
2023 Eapen M, Brazauskas R, Williams DA, Walters MC, St Martin A, Jacobs BL, Antin JH, Bona K, Chaudhury S, Coleman-Cowger VH, DiFronzo NL, Esrick EB, Field JJ, Fitzhugh CD, Kanter J, ... ... Kohn DB, et al. Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease. Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology. JCO2201203. PMID 36623245 DOI: 10.1200/JCO.22.01203  0.311
2022 Dvorak CC, Haddad E, Heimall J, Dunn E, Buckley RH, Kohn DB, Cowan MJ, Pai SY, Griffith LM, Cuvelier GDE, Eissa H, Shah AJ, O'Reilly RJ, Pulsipher MA, Wright NAM, et al. The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions. The Journal of Allergy and Clinical Immunology. PMID 36456361 DOI: 10.1016/j.jaci.2022.10.022  0.331
2022 Wong RL, Sackey S, Brown D, Senadheera S, Masiuk K, Quintos JP, Colindres N, Riggan L, Morgan RA, Malech HL, Hollis RP, Kohn DB. Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease Recapitulates Endogenous CYBB Regulation and Expression. Blood. PMID 36332160 DOI: 10.1182/blood.2022016074  0.33
2022 Collon K, Bell JA, Gallo MC, Chang SW, Bougioukli S, Sugiyama O, Tassey J, Hollis R, Heckmann N, Oakes DA, Longjohn DB, Evseenko D, Kohn DB, Lieberman JR. Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential. Gene Therapy. PMID 36216880 DOI: 10.1038/s41434-022-00367-w  0.389
2022 White SL, Hart K, Kohn DB. Diverse Approaches to Gene Therapy of Sickle Cell Disease. Annual Review of Medicine. PMID 36067800 DOI: 10.1146/annurev-med-042921-021707  0.455
2022 Labrosse R, Boufaied I, Bourdin B, Gona S, Randolph HE, Logan BR, Bourbonnais S, Berthe C, Chan W, Buckley RH, Parrott RE, Cuvelier GDE, Kapoor N, Chandra S, Dávila Saldaña BJ, ... ... Kohn DB, et al. Aberrant T-cell exhaustion in SCID survivors with poor T-cell reconstitution post transplant. The Journal of Allergy and Clinical Immunology. PMID 35987350 DOI: 10.1016/j.jaci.2022.08.004  0.35
2022 White SL, Lee TD, Toy T, Carroll JE, Polsky L, Campo Fernandez B, Davila A, Kohn DB, Chang VY. Evaluation of Clonal Hematopoiesis in Pediatric ADA-SCID Gene Therapy Participants. Blood Advances. PMID 35914227 DOI: 10.1182/bloodadvances.2022007803  0.327
2022 Collon K, Gallo M, Bell J, Chang S, Rodman J, Sugiyama O, Kohn DB, Lieberman JR. Improving Lentiviral Transduction of Human Adipose-Derived Mesenchymal Stem Cells. Human Gene Therapy. PMID 35859364 DOI: 10.1089/hum.2022.117  0.455
2022 Magis W, DeWitt MA, Wyman SK, Vu JT, Heo SJ, Shao SJ, Hennig F, Romero ZG, Campo-Fernandez B, Said S, McNeill MS, Rettig GR, Sun Y, Wang Y, Behlke MA, ... Kohn DB, et al. High-level correction of the sickle mutation is amplified during erythroid differentiation. Iscience. 25: 104374. PMID 35633935 DOI: 10.1016/j.isci.2022.104374  0.308
2021 Han J, Tam K, Tam C, Hollis RP, Kohn DB. Improved lentiviral vector titers from a multi-gene knockout packaging line. Molecular Therapy Oncolytics. 23: 582-592. PMID 34938858 DOI: 10.1016/j.omto.2021.11.012  0.344
2021 Li YR, Zhou Y, Kim YJ, Zhu Y, Ma F, Yu J, Wang YC, Chen X, Li Z, Zeng S, Wang X, Lee D, Ku J, Tsao T, Hardoy C, ... ... Kohn DB, et al. Development of allogeneic HSC-engineered iNKT cells for off-the-shelf cancer immunotherapy. Cell Reports. Medicine. 2: 100449. PMID 34841295 DOI: 10.1016/j.xcrm.2021.100449  0.428
2021 Abou-El-Enein M, Angelis A, Appelbaum FR, Andrews NC, Bates SE, Bierman AS, Brenner MK, Cavazzana M, Caligiuri MA, Clevers H, Cooke E, Daley GQ, Dzau VJ, Ellis LM, Fineberg HV, ... ... Kohn DB, et al. Evidence generation and reproducibility in cell and gene therapy research: A call to action. Molecular Therapy. Methods & Clinical Development. 22: 11-14. PMID 34377737 DOI: 10.1016/j.omtm.2021.06.012  0.333
2021 Baloh CH, Borkar SA, Chang KF, Yao J, Hershfield MS, Parikh SH, Kohn DB, Goodenow MM, Sleasman JW, Yin L. Normal IgH Repertoire Diversity in an Infant with ADA Deficiency After Gene Therapy. Journal of Clinical Immunology. PMID 34184208 DOI: 10.1007/s10875-021-01034-2  0.415
2021 Reinhardt BC, Habib O, Shaw KL, Garabedian EK, Carbonaro-Sarracino DA, Terrazas DR, Campo-Fernandez B, De Oliveira S, Moore TB, Ikeda A, Engel BC, Podsakoff GM, Hollis RP, Fernandes A, Jackson CR, ... ... Kohn DB, et al. Long-term Outcomes after Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency (ADA SCID). Blood. PMID 33974038 DOI: 10.1182/blood.2020010260  0.441
2021 Gray DH, Villegas I, Long J, Santos J, Keir A, Abele A, Kuo CY, Kohn DB. Optimizing Integration and Expression of Transgenic Bruton's Tyrosine Kinase for CRISPR-Cas9-Mediated Gene Editing of X-Linked Agammaglobulinemia. The Crispr Journal. 4: 191-206. PMID 33876953 DOI: 10.1089/crispr.2020.0080  0.468
2021 Carbonaro-Sarracino DA, Chun K, Clark DN, Kaufman ML, Jin X, Wang X, Kohn DB. Gene delivery using AAV8 for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID. Molecular Therapy. Methods & Clinical Development. 20: 765-778. PMID 33738330 DOI: 10.1016/j.omtm.2021.02.007  0.417
2021 Cannon P, Asokan A, Czechowicz A, Hammond P, Kohn DB, Lieber A, Malik P, Marks P, Porteus M, Verhoeyen E, Weissman D, Weissman I, Kiem HP. Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development National Institutes of Health/Bill & Melinda Gates Foundation Expert Scientific Roundtable Webinar Meeting. Human Gene Therapy. PMID 33427035 DOI: 10.1089/hum.2020.263  0.562
2020 Benitez EK, Lomova Kaufman A, Cervantes L, Clark DN, Ayoub PG, Senadheera S, Osborne K, Sanchez JM, Crisostomo RV, Wang X, Reuven N, Shaul Y, Hollis RP, Romero Z, Kohn DB. Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells. Frontiers in Genome Editing. 2: 601541. PMID 34713224 DOI: 10.3389/fgeed.2020.601541  0.322
2020 Han J, Tam K, Ma F, Tam C, Aleshe B, Wang X, Quintos JP, Morselli M, Pellegrini M, Hollis RP, Kohn DB. β-Globin Lentiviral Vectors Have Reduced Titers due to Incomplete Vector RNA Genomes and Lowered Virion Production. Stem Cell Reports. PMID 33186538 DOI: 10.1016/j.stemcr.2020.10.007  0.393
2020 Yang P, Chou SJ, Li J, Hui W, Liu W, Sun N, Zhang RY, Zhu Y, Tsai ML, Lai HI, Smalley M, Zhang X, Chen J, Romero Z, Liu D, ... ... Kohn DB, et al. Supramolecular nanosubstrate-mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies. Science Advances. 6. PMID 33097539 DOI: 10.1126/sciadv.abb7107  0.333
2020 Kuo CY, Garabedian E, Puck J, Cowan MJ, Sullivan KE, Buckley RH, Cunningham-Rundles C, Marsh R, Candotti F, Kohn DB. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry. Journal of Clinical Immunology. PMID 32880085 DOI: 10.1007/S10875-020-00857-9  0.408
2020 Kuo CY, Kohn DB. Overview of the current status of gene therapy for primary immune deficiencies (PIDs). The Journal of Allergy and Clinical Immunology. 146: 229-233. PMID 32771134 DOI: 10.1016/J.Jaci.2020.05.024  0.402
2020 Masterman KA, Haigh OL, Tullett KM, Leal-Rojas IM, Walpole C, Pearson FE, Cebon J, Schmidt C, O'Brien L, Rosendahl N, Daraj G, Caminschi I, Gschweng EH, Hollis RP, Kohn DB, et al. Human CLEC9A antibodies deliver NY-ESO-1 antigen to CD141 dendritic cells to activate naïve and memory NY-ESO-1-specific CD8 T cells. Journal For Immunotherapy of Cancer. 8. PMID 32737142 DOI: 10.1136/Jitc-2020-000691  0.375
2020 Bradford KL, Pearl M, Kohn DB, Weng P, Yadin O, Bowles V, De Oliveira SN, Moore TB. AT1R Activating Autoantibodies in Hematopoietic Stem Cell Transplantation. Biology of Blood and Marrow Transplantation : Journal of the American Society For Blood and Marrow Transplantation. PMID 32736008 DOI: 10.1016/J.Bbmt.2020.07.029  0.372
2020 Bradford KL, Liu S, Krajinovic M, Ansari M, Garabedian E, Tse J, Wang X, Shaw KL, Gaspar HB, Candotti F, Kohn DB. Busulfan Pharmacokinetics in ADA SCID Gene Therapy. Biology of Blood and Marrow Transplantation : Journal of the American Society For Blood and Marrow Transplantation. PMID 32653625 DOI: 10.1016/J.Bbmt.2020.07.004  0.397
2020 Bosticardo M, Pala F, Calzoni E, Delmonte OM, Dobbs K, Gardner CL, Sacchetti N, Kawai T, Garabedian EK, Draper D, Bergerson JRE, DeRavin SS, Freeman AF, Güngör T, Hartog N, ... ... Kohn DB, et al. Artificial thymic organoids represent a reliable tool to study T-cell differentiation in patients with severe T-cell lymphopenia. Blood Advances. 4: 2611-2616. PMID 32556283 DOI: 10.1182/Bloodadvances.2020001730  0.48
2020 Morgan RA, Ma F, Unti MJ, Brown D, Ayoub PG, Tam C, Lathrop L, Aleshe B, Kurita R, Nakamura Y, Senadheera S, Wong RL, Hollis RP, Pellegrini M, Kohn DB. Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences. Molecular Therapy. Methods & Clinical Development. 17: 999-1013. PMID 32426415 DOI: 10.1016/J.Omtm.2020.04.006  0.446
2020 Burroughs L, Petrovic A, Brazauskas R, Liu X, Griffith LM, Ochs HD, Bleesing J, Edwards S, Dvorak CC, Chaudhury S, Prockop S, Quinones R, Goldman F, Quigg T, Chandrakasan S, ... ... Kohn DB, et al. Excellent Outcomes Following Hematopoietic Cell Transplantation for Wiskott-Aldrich Syndrome: A PIDTC Report. Blood. PMID 32268350 DOI: 10.1182/Blood.2019002939  0.375
2020 Chan AY, Leiding JW, Liu X, Logan BR, Burroughs LM, Allenspach EJ, Skoda-Smith S, Uzel G, Notarangelo LD, Slatter M, Gennery AR, Smith AR, Pai SY, Jordan MB, Marsh RA, ... ... Kohn DB, et al. Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey. Frontiers in Immunology. 11: 239. PMID 32153572 DOI: 10.3389/Fimmu.2020.00239  0.402
2020 Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, et al. Lentiviral gene therapy for X-linked chronic granulomatous disease. Nature Medicine. PMID 31988463 DOI: 10.1038/S41591-019-0735-5  0.492
2020 Carbonaro-Sarracino DA, Tarantal AF, Lee CCI, Kaufman ML, Wandro S, Jin X, Martinez M, Clark DN, Chun K, Koziol C, Hardee CL, Wang X, Kohn DB. Dosing and Re-Administration of Lentiviral Vector for Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice. Molecular Therapy. Methods & Clinical Development. 16: 78-93. PMID 31871959 DOI: 10.1016/J.Omtm.2019.11.004  0.32
2020 Benitez EK, Lomova Kaufman A, Cervantes L, Clark DN, Ayoub PG, Senadheera S, Osborne K, Sanchez JM, Crisostomo RV, Wang X, Reuven N, Shaul Y, Hollis RP, Romero Z, Kohn DB. Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells Frontiers in Genome Editing. 2. DOI: 10.3389/fgeed.2020.601541  0.325
2020 Gregory GL, Wienert B, Schwab M, Lianoglou BR, Hollis RP, Kohn DB, Conklin BR, MacKenzie TC. Investigating Zeta Globin Gene Expression to Develop a Potential Therapy for Alpha Thalassemia Major Blood. 136: 3-4. DOI: 10.1182/blood-2020-142922  0.31
2020 Kohn DB, Rao GR, Almarza E, Terrazas D, Nicoletti E, Fernandes A, Kuo C, De Oliveira SN, Moore TB, Law KM, Beard BC, Sevilla J, Mesa-Núñez C, Bueren JA, Schwartz JD. A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1 Blood. 136: 15-15. DOI: 10.1182/BLOOD-2020-142484  0.343
2020 Agarwal R, Weinberg KI, Kwon H, Le A, Long-Boyle JR, Kohn DB, Bradford K, De Oliveira S, Bertaina A, Czechowicz A, Porteus M, Shyr DC, Weinacht KG, Shah AJ, Mavers M, et al. First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency Blood. 136: 10-10. DOI: 10.1182/blood-2020-137762  0.434
2020 Thakar MS, Logan B, Buckley RH, Haddad E, Dvorak CC, O'Reilly RJ, Kapoor N, Satter LF, Martinez C, Pai S, Heimall J, Jyonouchi S, Sullivan KE, Chandra S, Smith AR, ... ... Kohn DB, et al. Transplantation Outcomes for Children with Severe Combined Immune Deficiency (SCID) Have Improved over Time: A 36-Year Summary Report By the Primary Immune Deficiency Treatment Consortium (PIDTC) Biology of Blood and Marrow Transplantation. 26: S18-S19. DOI: 10.1016/J.Bbmt.2019.12.083  0.311
2019 Farrell AT, Panepinto J, Desai AA, Kassim AA, Lebensburger J, Walters MC, Bauer DE, Blaylark RM, DiMichele DM, Gladwin MT, Green NS, Hassell K, Kato GJ, Klings ES, Kohn DB, et al. End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings. Blood Advances. 3: 4002-4020. PMID 31809537 DOI: 10.1182/Bloodadvances.2019000883  0.514
2019 Morgan RA, Unti MJ, Aleshe B, Brown D, Osborne KS, Koziol C, Smith OB, O'Brien R, Tam C, Miyahira E, Ruiz M, Quintos JP, Senadheera S, Hollis RP, Kohn DB. Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 31628051 DOI: 10.1016/J.Ymthe.2019.09.020  0.44
2019 Zhu Y, Smith DJ, Zhou Y, Li YR, Yu J, Lee D, Wang YC, Di Biase S, Wang X, Hardoy C, Ku J, Tsao T, Lin LJ, Pham AT, Moon H, ... ... Kohn DB, et al. Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer. Cell Stem Cell. PMID 31495780 DOI: 10.1016/J.Stem.2019.08.004  0.84
2019 Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, ... ... Kohn DB, et al. Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. Journal of Clinical Immunology. PMID 31376032 DOI: 10.1007/S10875-019-00659-8  0.325
2019 Romero Z, Lomova A, Said S, Miggelbrink A, Kuo CY, Campo-Fernandez B, Hoban MD, Masiuk KE, Clark DN, Long J, Sanchez JM, Velez M, Miyahira E, Zhang R, Brown D, ... ... Kohn DB, et al. Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 31178391 DOI: 10.1016/J.Ymthe.2019.05.014  0.58
2019 Masiuk KE, Zhang R, Osborne K, Hollis RP, Campo-Fernandez B, Kohn DB. PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a β-Globin Lentiviral Vector. Molecular Therapy. Methods & Clinical Development. 13: 390-398. PMID 31024981 DOI: 10.1016/J.Omtm.2019.03.005  0.537
2019 Hazim RA, Karumbayaram S, Jiang M, Dimashkie A, Lopes VS, Li D, Burgess BL, Vijayaraj P, Alva-Ornelas JA, Zack JA, Kohn DB, Gomperts BN, Pyle AD, Lowry WE, Williams DS. Correction to: Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization. Stem Cell Research & Therapy. 10: 52. PMID 30755264 DOI: 10.1186/S13287-019-1147-7  0.371
2019 Soni S, Kohn DB. Chemistry, manufacturing and controls for gene modified hematopoietic stem cells. Cytotherapy. PMID 30745225 DOI: 10.1016/J.Jcyt.2018.12.001  0.526
2019 Amatuni GS, Currier RJ, Church JA, Bishop T, Grimbacher E, Nguyen AA, Agarwal-Hashmi R, Aznar CP, Butte MJ, Cowan MJ, Dorsey MJ, Dvorak CC, Kapoor N, Kohn DB, Markert ML, et al. Newborn Screening for Severe Combined Immunodeficiency and T-cell Lymphopenia in California, 2010-2017. Pediatrics. PMID 30683812 DOI: 10.1542/Peds.2018-2300  0.316
2019 Kwon HS, Logan AC, Chhabra A, Pang WW, Czechowicz A, Tate K, Le A, Poyser J, Hollis R, Kelly BV, Kohn DB, Weissman IL, Prohaska SS, Shizuru JA. Anti-human CD117 antibody-mediated bone marrow niche clearance in non-human primates and humanized NSG mice. Blood. PMID 30617195 DOI: 10.1182/Blood-2018-06-853879  0.416
2019 Kohn DB, Shaw KL, Garabedian E, Carbonaro-Sarracino DA, Moore TB, Oliveira SND, Tse J, Shupien S, Terrazas D, Davila A, Icreverzi A, Yu A, Chun KM, Casas CE, Barman P, et al. Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort Blood. 134: 3345-3345. DOI: 10.1182/Blood-2019-123432  0.505
2019 Walters M, Magis W, DeWitt M, Wyman S, Vu JT, Romero ZG, Fernandez CB, Kohn DB, Boffelli D, Corn JE, Martin DI. IN VIVO SELECTION FOR CORRECTED BETA-GLOBIN ALLELES AFTER CRISPR/CAS9 EDITING IN HUMAN SICKLE HEMATOPOIETIC STEM CELLS ENHANCES THERAPEUTIC POTENTIAL Hemasphere. 3: 380. DOI: 10.1097/01.HS9.0000561688.44273.20  0.305
2018 Masiuk KE, Laborada J, Roncarolo MG, Hollis RP, Kohn DB. Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome. Cell Stem Cell. PMID 30639036 DOI: 10.1016/J.Stem.2018.12.003  0.585
2018 Kohn DB. Gene therapy for blood diseases. Current Opinion in Biotechnology. 60: 39-45. PMID 30599357 DOI: 10.1016/J.Copbio.2018.11.016  0.52
2018 Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F. Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease. Molecular Therapy. Methods & Clinical Development. 11: 167-179. PMID 30533448 DOI: 10.1016/J.Omtm.2018.10.014  0.854
2018 Puig-Saus C, Parisi G, Garcia-Diaz A, Krystofinski PE, Sandoval S, Zhang R, Champhekar AS, McCabe J, Cheung-Lau GC, Truong NA, Vega-Crespo A, Komenan MDS, Pang J, Macabali MH, Saco JD, ... ... Kohn DB, et al. IND-enabling studies for a clinical trial to genetically program a persistent cancer-targeted immune system. Clinical Cancer Research : An Official Journal of the American Association For Cancer Research. PMID 30409823 DOI: 10.1158/1078-0432.Ccr-18-0963  0.571
2018 Lomova A, Clark DN, Campo-Fernandez B, Flores-Bjurström C, Kaufman ML, Fitz-Gibbon S, Wang X, Miyahira EY, Brown D, DeWitt MA, Corn JE, Hollis RP, Romero Z, Kohn DB. Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair. Stem Cells (Dayton, Ohio). PMID 30372555 DOI: 10.1002/Stem.2935  0.528
2018 Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, et al. T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency. Genome Medicine. 10: 70. PMID 30261899 DOI: 10.1186/S13073-018-0580-Z  0.503
2018 Urbinati F, Campo Fernandez B, Masiuk KE, Poletti V, Hollis RP, Koziol C, Kaufman M, Brown D, Mavilio F, Kohn DB. Gene Therapy for Sickle Cell Disease: a lentiviral vector comparison study. Human Gene Therapy. PMID 30198339 DOI: 10.1089/Hum.2018.061  0.857
2018 Kohn DB, Hershfield MS, Puck JM, Aiuti A, Blincoe A, Gaspar HB, Notarangelo LD, Grunebaum E. Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency. The Journal of Allergy and Clinical Immunology. PMID 30194989 DOI: 10.1016/J.Jaci.2018.08.024  0.44
2018 Hu P, Bi Y, Ma H, Suwanmanee T, Zeithaml B, Fry NJ, Kohn DB, Kafri T. Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization. Gene Therapy. PMID 30190607 DOI: 10.1038/S41434-018-0039-2  0.372
2018 Haddad E, Logan BR, Griffith LM, Buckley RH, Parrot RE, Prockop SE, Small TN, Chaisson J, Dvorak CC, Murnane M, Kapoor N, Abdel-Azim H, Hanson IC, Martinez C, Bleesing JJH, ... ... Kohn DB, et al. SCID genotype and 6-month post-transplant CD4 count predict survival and immune recovery: a PIDTC retrospective study. Blood. PMID 30154114 DOI: 10.1182/Blood-2018-03-840702  0.401
2018 Kuo CY, Long JD, Campo-Fernandez B, de Oliveira S, Cooper AR, Romero Z, Hoban MD, Joglekar AV, Lill GR, Kaufman ML, Fitz-Gibbon S, Wang X, Hollis RP, Kohn DB. Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome. Cell Reports. 23: 2606-2616. PMID 29847792 DOI: 10.1016/J.Celrep.2018.04.103  0.833
2018 Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Abdel-Azim H, Prockop SE, Shyr D, Decaluwe H, Hanson IC, Gillio A, Dávila Saldaña BJ, Eibel H, Hopkins G, ... ... Kohn DB, et al. B cell differentiation and IL-21 response in / SCID patients after hematopoietic stem cell transplantation. Blood. PMID 29728406 DOI: 10.1182/Blood-2017-10-809822  0.429
2018 Dunbar CE, High KA, Joung JK, Kohn DB, Ozawa K, Sadelain M. Gene therapy comes of age. Science (New York, N.Y.). 359. PMID 29326244 DOI: 10.1126/Science.Aan4672  0.468
2018 Velez M, Sanchez J, Miggelbrink A, Romero Z, Kohn D. Using HUDEP Cells as a Model for Gene Editing for -Thalassemia and Sickle Cell Disease Experimental Hematology. 64. DOI: 10.1016/J.Exphem.2018.06.167  0.487
2018 Seet C, Li S, Chick B, Casero D, Kim J, Gschweng E, Chen H, Zhu Y, Lopez S, Miao R, Montel-Hagen A, Kohn D, Sehl M. Notch Signaling Regulates the Differentiation of CLEC9A+ Dendritic Cells (cDC1) From Human and Mouse Hematopoietic Stem/Progenitor Cells Experimental Hematology. 64. DOI: 10.1016/J.Exphem.2018.06.145  0.445
2018 Sanchez J, Velez M, Miggelbrink A, Romero Z, Kohn D. Comparing Two Gene Therapy Approaches for the Treatment of Sickle Cell Disease: Gene Correction vs. Allelic Disruption Experimental Hematology. 64. DOI: 10.1016/J.Exphem.2018.06.139  0.463
2018 Leiding JW, Logan BR, Yin Z, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs L, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GD, Thakar M, Chen K, ... ... Kohn DB, et al. Resolution of CGD Related Colitis after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Chronic Granulomatous Disease—Early Results From the 6903 Study of the Primary Immune Deficiency Treatment Consortium (PIDTC) Biology of Blood and Marrow Transplantation. 24: S80-S81. DOI: 10.1016/J.Bbmt.2017.12.624  0.417
2017 Long J, Hoban MD, Cooper AR, Kaufman ML, Kuo CY, Campo-Fernandez B, Lumaquin D, Hollis RP, Wang X, Kohn DB, Romero Z. Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 29221806 DOI: 10.1016/J.Ymthe.2017.11.001  0.706
2017 Morgan RA, Gray D, Lomova A, Kohn DB. Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned. Cell Stem Cell. 21: 574-590. PMID 29100011 DOI: 10.1016/J.Stem.2017.10.010  0.542
2017 Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Pulsipher MA, Parikh S, Martinez C, Kapoor N, O'Reilly R, Boyer M, Pai SY, Goldman F, et al. Immune Reconstitution and Survival of 100 SCID Patients Post Hematopoietic Cell Transplant: A PIDTC Natural History Study. Blood. PMID 29021228 DOI: 10.1182/Blood-2017-05-781849  0.42
2017 Hazim RA, Karumbayaram S, Jiang M, Dimashkie A, Lopes VS, Li D, Burgess BL, Vijayaraj P, Alva-Ornelas JA, Zack JA, Kohn DB, Gomperts BN, Pyle AD, Lowry WE, Williams DS. Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization. Stem Cell Research & Therapy. 8: 217. PMID 28969679 DOI: 10.1186/S13287-017-0652-9  0.479
2017 Kohn DB. Gene therapy: WAS (not) just for kids. Blood. 130: 1278-1279. PMID 28912292 DOI: 10.1182/Blood-2017-08-798496  0.503
2017 Kohn DB. Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy. Hematology/Oncology Clinics of North America. 31: 721-735. PMID 28895843 DOI: 10.1016/J.Hoc.2017.06.006  0.531
2017 Bradford KL, Moretti FA, Carbonaro-Sarracino DA, Gaspar HB, Kohn DB. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations. Journal of Clinical Immunology. PMID 28842866 DOI: 10.1007/S10875-017-0433-3  0.485
2017 Urbinati F, Wherley J, Geiger S, Fernandez BC, Kaufman ML, Cooper A, Romero Z, Marchioni F, Reeves L, Read E, Nowicki B, Grassman E, Viswanathan S, Wang X, Hollis RP, ... Kohn DB, et al. Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease. Cytotherapy. PMID 28733131 DOI: 10.1016/J.Jcyt.2017.06.002  0.84
2017 Masiuk KE, Brown D, Laborada J, Hollis RP, Urbinati F, Kohn DB. Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 28663101 DOI: 10.1016/J.Ymthe.2017.05.023  0.843
2017 Seet CS, He C, Bethune MT, Li S, Chick B, Gschweng EH, Zhu Y, Kim K, Kohn DB, Baltimore D, Crooks GM, Montel-Hagen A. Generation of mature T cells from human hematopoietic stem and progenitor cells in artificial thymic organoids. Nature Methods. PMID 28369043 DOI: 10.1038/Nmeth.4237  0.606
2017 Cooper AR, Lill GR, Shaw K, Carbonaro D, Davila A, Sokolic R, Candotti F, Pellegrini M, Kohn DB. Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients. Blood. PMID 28351939 DOI: 10.1182/Blood-2016-12-756734  0.7
2017 Shaw KL, Garabedian E, Mishra S, Barman P, Davila A, Carbonaro D, Shupien S, Silvin C, Geiger S, Nowicki B, Smogorzewska EM, Brown B, Wang X, de Oliveira S, Choi Y, ... ... Kohn DB, et al. Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency. The Journal of Clinical Investigation. PMID 28346229 DOI: 10.1172/Jci90367  0.769
2017 Kohn DB, Kuo CY. New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing. The Journal of Allergy and Clinical Immunology. 139: 726-732. PMID 28270364 DOI: 10.1016/J.Jaci.2017.01.007  0.534
2017 Kohn DB, Gaspar HB. How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID). Journal of Clinical Immunology. PMID 28194615 DOI: 10.1007/S10875-017-0373-Y  0.479
2017 Seet CS, Li S, Chick B, Casero D, Gschweng EH, Zhu Y, Miao R, Montel-Hagen A, Kohn DB, Sehl M. Notch Signaling Promotes the Differentiation of Human CLEC9A+ Dendritic Cells from Hematopoietic Stem and Progenitor Cells Blood. 130: 624-624. DOI: 10.1182/Blood.V130.Suppl_1.624.624  0.405
2017 Kasbekar S, Bueren J, Thrasher AJ, Kohn DB, Sevilla J, Schwartz JD, Almarza E. Leukocyte Adhesion Deficiency-I (LAD-I): A Comprehensive Review of Published Cases Blood. 130: 4821-4821. DOI: 10.1182/Blood.V130.Suppl_1.4821.4821  0.355
2017 Puig-Saus C, Parisi G, Krystofinski P, Garcia-Diaz A, Sandoval S, McCabe J, Zhang R, Cheung-Lau G, Truong N, Saco J, Komenan S, Vega-Crespo A, Macabali MH, Comin-Anduix B, Berent-Maoz B, ... Kohn D, et al. Abstract 3765: IND-Enabling GLP study to support a clinical trial of dual adoptive cell therapy combining stem cells and T cells engineered with an NY-ESO-1 TCR Cancer Research. 77: 3765-3765. DOI: 10.1158/1538-7445.Am2017-3765  0.537
2017 Lobry T, Sharma J, Ur S, Lau A, Rocca C, Kohn DB, Carbonaro D, Cabrera B, Hernandez L, Cherqui S. Towards a phase I clinical trial for autologous hematopoietic stem cells transplantation in cystinosis Molecular Genetics and Metabolism. 120. DOI: 10.1016/J.Ymgme.2016.11.209  0.41
2017 Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Puck J, Prockop SE, Kapoor N, Abdel-Azim H, Hanson IC, Martinez C, Bleesing J, Chandra S, Smith AR, ... ... Kohn DB, et al. Genotype, Phenotype and T Cell Counts at One Year Predict Survival and Long Term Immune Reconstitution after Transplantation in Severe Combined Immune Deficiency (SCID)—The Primary Immune Deficiency Treatment Consortium (PIDTC) Biology of Blood and Marrow Transplantation. 23: S133-S134. DOI: 10.1016/J.Bbmt.2017.01.052  0.327
2016 Bjurström CF, Mojadidi M, Phillips J, Kuo C, Lai S, Lill GR, Cooper A, Kaufman M, Urbinati F, Wang X, Hollis RP, Kohn DB. Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases. Molecular Therapy. Nucleic Acids. 5: e351. PMID 28131278 DOI: 10.1038/Mtna.2016.52  0.843
2016 Bethune MT, Gee MH, Bunse M, Lee MS, Gschweng EH, Pagadala MS, Zhou J, Cheng D, Heath JR, Kohn DB, Kuhns MS, Uckert W, Baltimore D. Domain-swapped T cell receptors improve the safety of TCR gene therapy. Elife. 5. PMID 27823582 DOI: 10.7554/Elife.19095  0.581
2016 DeWitt MA, Magis W, Bray NL, Wang T, Berman JR, Urbinati F, Heo SJ, Mitros T, Muñoz DP, Boffelli D, Kohn DB, Walters MC, Carroll D, Martin DI, Corn JE. Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells. Science Translational Medicine. 8: 360ra134. PMID 27733558 DOI: 10.1126/Scitranslmed.Aaf9336  0.849
2016 Punwani D, Kawahara M, Yu J, Sanford U, Roy S, Patel K, Carbonaro DA, Karlen AD, Khan S, Cornetta KG, Rothe M, Schambach A, Kohn DB, Malech HL, McIvor RS, et al. Lentivirus Mediated Correction of Artemis-deficient Severe Combined Immunodeficiency. Human Gene Therapy. PMID 27611239 DOI: 10.1089/Hum.2016.064  0.546
2016 Smith DJ, Lin LJ, Moon H, Pham AT, Wang X, Liu S, Ji S, Rezek V, Shimizu S, Ruiz M, Lam J, Janzen DM, Memarzadeh S, Kohn DB, Zack J, et al. Propagating Humanized BLT Mice for the Study of Human Immunology and Immunotherapy. Stem Cells and Development. PMID 27608727 DOI: 10.1089/scd.2016.0193  0.319
2016 Hoban MD, Lumaquin D, Kuo CY, Romero Z, Long J, Ho M, Young CS, Mojadidi M, Fitz-Gibbon S, Cooper AR, Lill GR, Urbinati F, Campo-Fernandez B, Bjurstrom CF, Pellegrini M, ... ... Kohn DB, et al. CRISPR/Cas9-mediated correction of the sickle mutation in human CD34+ cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 27406980 DOI: 10.1038/Mt.2016.148  0.843
2016 Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Shearer WT, Burroughs LM, Torgerson TR, Decaluwe H, Haddad E. Primary Immune Deficiency Treatment Consortium (PIDTC) update. The Journal of Allergy and Clinical Immunology. PMID 27262745 DOI: 10.1016/J.Jaci.2016.01.051  0.313
2016 Kuo CY, Kohn DB. Gene Therapy for the Treatment of Primary Immune Deficiencies. Current Allergy and Asthma Reports. 16: 39. PMID 27056559 DOI: 10.1007/S11882-016-0615-8  0.496
2016 Kohn DB, Porteus MH, Scharenberg AM. Ethical and regulatory aspects of genome editing. Blood. PMID 27053531 DOI: 10.1182/Blood-2016-01-678136  0.422
2016 Chin CJ, Cooper AR, Lill GR, Evseenko D, Zhu Y, He CB, Casero D, Pellegrini M, Kohn DB, Crooks GM. Genetic Tagging During Human Mesoderm Differentiation Reveals Tripotent Lateral Plate Mesodermal Progenitors. Stem Cells (Dayton, Ohio). PMID 26934332 DOI: 10.1002/Stem.2351  0.65
2016 Young CS, Hicks MR, Ermolova NV, Nakano H, Jan M, Younesi S, Karumbayaram S, Kumagai-Cresse C, Wang D, Zack JA, Kohn DB, Nakano A, Nelson SF, Miceli MC, Spencer MJ, et al. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells. Cell Stem Cell. PMID 26877224 DOI: 10.1016/J.Stem.2016.01.021  0.375
2016 Hoban MD, Romero Z, Cost GJ, Mendel M, Holmes M, Kohn DB. Delivery of Genome Editing Reagents to Hematopoietic Stem/Progenitor Cells. Current Protocols in Stem Cell Biology. 36: 5B.4.1-5B.4.10. PMID 26840227 DOI: 10.1002/9780470151808.Sc05B04S36  0.506
2016 Bethune MT, Gee MH, Bunse M, Lee MS, Gschweng EH, Pagadala MS, Zhou J, Cheng D, Heath JR, Kohn DB, Kuhns MS, Uckert W, Baltimore D. Author response: Domain-swapped T cell receptors improve the safety of TCR gene therapy Elife. DOI: 10.7554/Elife.19095.020  0.385
2016 Seet CS, He C, Bethune M, Li S, Chick B, Gschweng E, Zhu Y, Kim K, Baltimore D, Kohn DB, Montel-Hagen A, Crooks GM. Artificial Thymic Organoids Permit Allelic Exclusion and Efficient Generation of Naïve TCR-Engineered T-Cells from Human Hematopoietic Stem Cells In Vitro Blood. 128: 4553-4553. DOI: 10.1182/Blood.V128.22.4553.4553  0.608
2016 Bjurström CF, Mojadidi M, Lomova A, Lai S, Fitz-Gibbon S, Pellegrini M, Cooper AR, Lill GR, Kaufman M, Cost GJ, Holmes MC, Hollis RP, Kohn DB. Preservation of Gene Edited Hematopoietic Stem Cells By Transient Overexpression of BCL-2 mRNA Blood. 128: 3636-3636. DOI: 10.1182/Blood.V128.22.3636.3636  0.739
2016 Masiuk KE, Brown D, Urbinati F, Hollis RP, Kohn DB. Enrichment of Hematopoietic Stem Cells Using Immunomagnetic Beads to Facilitate Gene Therapy Blood. 128: 2313-2313. DOI: 10.1182/Blood.V128.22.2313.2313  0.843
2016 Carbonaro-Sarracino DA, Tarantal AF, Lee CI, Kaufman ML, Martinez M, Clark DN, Koziol C, Kohn DB. 764. Dosing and Re-Administration of Intravenous Lentiviral Vector for Liver-Directed Gene Transfer in Young Rhesus Monkeys and ADA-Deficient Mice Molecular Therapy. 24: S302-S303. DOI: 10.1016/S1525-0016(16)33572-9  0.314
2016 Eichler FS, Duncan C, Amartino H, Aziz-Bose R, Baruchel A, Dansereau C, Dalle J, Gaspar HB, Gissen P, Kohn DB, Larghero J, Lund TC, Miller WP, Musolino PL, O'Meara T, et al. 250. A Phase 2/3 Study of the Efficacy and Safety of Ex Vivo Gene Therapy with Lenti-D TM Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy Molecular Therapy. 24: S98-S99. DOI: 10.1016/S1525-0016(16)33059-3  0.344
2016 Urbinati F, Poletti V, Fernandez BC, Hollis RP, Koziol C, Kaufman ML, Brown D, Miccio A, Antoniou M, Kohn DB, Mavilio F. 230. Designing High-Titer Lentiviral Vectors for Gene Therapy of Sickle-Cell Disease Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33039-8  0.86
2016 Long J, Hoban MD, Kuo CY, Fernandez BC, Cooper AR, Lumaquin D, Hollis RP, Kohn DB, Romero Z. 118. Characterization of Chromosomal Alterations Using a Zinc-Finger Nuclease Targeting the Beta-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)32927-6  0.646
2016 Lobry T, Sharma J, Ur S, Lau A, Rocca C, Kohn DB, Carbonaro D, Hernandez L, Cherqui S. 32. Towards a Phase I Clinical Trial for Cystinosis Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)32841-6  0.505
2016 Kohn D. Gene therapy for blood cell diseases with autologous hematopoietic stem cells Experimental Hematology. 44. DOI: 10.1016/J.Exphem.2016.06.013  0.547
2016 Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Puck J, Fleisher T, Griffith LM, Kohn DB, Pulsipher MA, Shearer W, Hanson IC, Kapoor N, O'Reilly RJ, Boyer M, Pai S, et al. Poor T Cell Reconstitution at 100 Days after T Cell-Replete Hematopoietic Cell Transplantation (HCT) for SCID Is Associated with Later Risk of Death or Need for 2nd Transplant in the 6901 Prospective Study of the Pidtc Biology of Blood and Marrow Transplantation. 22: S101-S102. DOI: 10.1016/J.Bbmt.2015.11.404  0.407
2015 Abdel-Azim H, Mahadeo KM, Zhao Q, Khazal S, Kohn DB, Crooks GM, Shah AJ, Kapoor N. Unrelated donor hematopoietic stem cell transplantation for the treatment of non-malignant genetic diseases: An alemtuzumab based regimen is associated with cure of clinical disease; earlier clearance of alemtuzumab may be associated with graft rejection. American Journal of Hematology. 90: 1021-6. PMID 26242764 DOI: 10.1002/Ajh.24141  0.388
2015 Kohn DB. Gene therapy outpaces haplo for SCID-X1. Blood. 125: 3521-2. PMID 26045591 DOI: 10.1182/Blood-2015-04-641720  0.427
2015 Romero Z, Campo-Fernandez B, Wherley J, Kaufman ML, Urbinati F, Cooper AR, Hoban MD, Baldwin KM, Lumaquin D, Wang X, Senadheera S, Hollis RP, Kohn DB. The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells. Molecular Therapy. Methods & Clinical Development. 2: 15012. PMID 26029723 DOI: 10.1038/Mtm.2015.12  0.832
2015 Corrigan-Curay J, O'Reilly M, Kohn DB, Cannon PM, Bao G, Bushman FD, Carroll D, Cathomen T, Joung JK, Roth D, Sadelain M, Scharenberg AM, von Kalle C, Zhang F, Jambou R, et al. Genome editing technologies: defining a path to clinic. Molecular Therapy : the Journal of the American Society of Gene Therapy. 23: 796-806. PMID 25943494 DOI: 10.1038/Mt.2015.54  0.317
2015 Hoban MD, Cost GJ, Mendel MC, Romero Z, Kaufman ML, Joglekar AV, Ho M, Lumaquin D, Gray D, Lill GR, Cooper AR, Urbinati F, Senadheera S, Zhu A, Liu PQ, ... ... Kohn DB, et al. Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells. Blood. 125: 2597-604. PMID 25733580 DOI: 10.1182/Blood-2014-12-615948  0.849
2015 Urbinati F, Hargrove PW, Geiger S, Romero Z, Wherley J, Kaufman ML, Hollis RP, Chambers CB, Persons DA, Kohn DB, Wilber A. Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells. Experimental Hematology. 43: 346-51. PMID 25681747 DOI: 10.1016/J.Exphem.2015.01.009  0.861
2015 Baldwin K, Urbinati F, Romero Z, Campo-Fernandez B, Kaufman ML, Cooper AR, Masiuk K, Hollis RP, Kohn DB. Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy. Stem Cells (Dayton, Ohio). 33: 1532-42. PMID 25588820 DOI: 10.1002/Stem.1957  0.828
2015 Awe JP, Gschweng EH, Vega-Crespo A, Voutila J, Williamson MH, Truong B, Kohn DB, Kasahara N, Byrne JA. Putative immunogenicity expression profiling using human pluripotent stem cells and derivatives. Stem Cells Translational Medicine. 4: 136-45. PMID 25575527 DOI: 10.5966/Sctm.2014-0117  0.518
2015 Cooper AR, Lill GR, Gschweng EH, Kohn DB. Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter. Nucleic Acids Research. 43: 682-90. PMID 25520191 DOI: 10.1093/Nar/Gku1312  0.616
2015 Mahadeo KM, Weinberg KI, Abdel-Azim H, Miklos DB, Killen R, Kohn D, Crooks GM, Shah AJ, Kharbanda S, Agarwal R, Kapoor N. A reduced-toxicity regimen is associated with durable engraftment and clinical cure of nonmalignant genetic diseases among children undergoing blood and marrow transplantation with an HLA-matched related donor. Biology of Blood and Marrow Transplantation : Journal of the American Society For Blood and Marrow Transplantation. 21: 440-4. PMID 25459642 DOI: 10.1016/J.Bbmt.2014.11.005  0.36
2015 Carbonaro-Sarracino DA, Kaufman ML, Jin X, Geiger S, Clark D, Kohn DB. 442. Decreased Survival and Engraftment in Adult ADA-Deficient Mice Co-Transplanted With Uncorrected Lineage-Positive Bone Marrow Cells Combined With Gene-Corrected Lineage-Negative Bone Marrow Cells Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)34051-5  0.678
2015 Hoban MD, Lumaquin D, Kuo C, Romero Z, Young C, Ho M, Long J, Coss N, Bjurstrom C, Mojadidi M, Hollis RP, Kohn DB. 338. Evaluation of TALENs and the CRISPR/Cas9 Nuclease System To Correct the Sickle Cell Disease Mutation Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33947-8  0.444
2015 Punwani D, Stillion M, Yu J, Malech HL, Carbonaro D, Kohn DB, McIvor S, Puck JM, Cowan MJ. 292. Lentivirus Vector Mediated Gene Correction in Artemis-Deficient Severe Combined Immunodeficiency Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33901-6  0.568
2015 Gaspar HB, Buckland K, Carbonaro DA, Shaw K, Barman P, Davila A, Gilmour KC, Booth C, Terrazs D, Cornetta K, Paruzynski A, Schmidt M, Sokolic R, Candotti F, Thrasher AJ, ... Kohn DB, et al. C-8. Immunological and Metabolic Correction After Lentiviral Vector Gene Therapy for ADA Deficiency Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33866-7  0.566
2015 Cooper AR, Shaw KL, Lill GR, Sokolic R, Candotti F, Kohn DB. 240. Stable and Clinically Benign Clonal Dominance in an ADA-SCID Patient Treated With Retroviral Gene Therapy Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33845-X  0.697
2015 Kuo CY, Hoban MD, Joglekar AV, Young CS, Kleinman J, Abele AN, Kohn DB. 121. Targeted Gene Therapy in the Treatment of X-Linked Hyper IgM Syndrome Molecular Therapy. 23: S50. DOI: 10.1016/S1525-0016(16)33726-1  0.744
2015 Hoban MD, Mendel MC, Romero Z, Kaufman ML, Joglekar AV, Ho M, Lumaquin D, Gray D, Lill GR, Cooper AR, Urbinati F, Senadheera S, Zhu A, Liu P, Paschon DE, ... ... Kohn DB, et al. 115. Correction of the Sickle-Cell Disease Mutation in Human Hematopoietic Stem/Progenitor Cells Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33720-0  0.818
2015 Lobry T, Ur S, Lau A, Sharma J, Rocca CJ, Kohn DB, Carbonaro D, Cabrera B, Cherqui S. 84. Pharmacology/Toxicology Studies for Gene-Modified Hematopoietic Stem Cell Transplantation for Cystinosis Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33689-9  0.55
2015 Shaw KL, Garabedian E, Sokolic R, Barman P, Davila A, Silvin C, Oliveira Sd, Shah AJ, Terrazas D, Carbonaro D, Geiger S, Mishra S, Cooper A, Smogorzewska M, Jagadeesh J, ... ... Kohn DB, et al. 30. Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID) Using a γ-Retroviral Vector Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33634-6  0.744
2015 Hacke K, Treger J, Bogan B, Rezek V, Narukawa M, Shimizu S, Liu P, Reik A, Lieber A, Crooks G, Kohn D, An DS, Kitchen S, Gregory P, Cost G, et al. A novel in vivo chemoselection strategy for genetically modified hematopoietic stem cells Cytotherapy. 17: S52. DOI: 10.1016/J.Jcyt.2015.03.486  0.431
2015 Kuo CY, Hoban MD, Joglekar AV, Kohn DB. Site Specific Gene Correction of Defects in CD40 Ligand Using the Crispr/Cas9 Genome Editing Platform Journal of Allergy and Clinical Immunology. 135: AB17. DOI: 10.1016/J.Jaci.2014.12.987  0.735
2015 Bjurström CF, Mojadidi M, Campo B, Reik A, Holmes MC, Cost G, Gregory PD, Hoban MD, Hollis RP, Kohn DB. Identifying mechanisms that limit efficient site-specific gene modification by homology-directed repair in hematopoietic stem cells Experimental Hematology. 43. DOI: 10.1016/J.Exphem.2015.06.086  0.471
2015 Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Griffith LM, Puck J, Parikh S, O'Reilly RJ, Pai S, Hanson IC, Martinez C, Pulsipher MA, Kapoor N, Goldman F, Kletzel M, ... ... Kohn DB, et al. Early Hematopoietic Cell Transplant (HCT) Outcomes of Children with Severe Combined Immunodeficiency Disease (SCID): The First Seventy Four Patients of the Primary Immune Deficiency Treatment Consortium (PIDTC) Prospective Study 6901 Biology of Blood and Marrow Transplantation. 21: S289-S291. DOI: 10.1016/J.Bbmt.2014.11.459  0.379
2015 Abdel-Azim H, Zhao Q, Mahadeo KM, Khazal S, Kohn DB, Shah AJ, Kapoor N. Unrelated Donor Hematopoietic Stem Cell Transplantation for Treatment of Non-Malignant Genetic Diseases Using a Myeloablative Reduced Toxicity Conditioning Regimen Biology of Blood and Marrow Transplantation. 21. DOI: 10.1016/J.Bbmt.2014.11.369  0.422
2015 Kohn DB, Shaw KL, Sokolic R, Carbonaro DA, Davila A, Barman P, Garabedian E, Silvin C, Oliveira SD, Shah AJ, Moore TB, Hershfield M, Thrasher A, Gaspar HR, Candotti F. Autologous Transplant/Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency Biology of Blood and Marrow Transplantation. 21. DOI: 10.1016/J.Bbmt.2014.11.123  0.367
2014 Corrigan-Curay J, Kiem HP, Baltimore D, O'Reilly M, Brentjens RJ, Cooper L, Forman S, Gottschalk S, Greenberg P, Junghans R, Heslop H, Jensen M, Mackall C, June C, Press O, ... ... Kohn DB, et al. T-Cell Immunotherapy: Looking Forward: T Cell Immunotherapy: Optimizing Trial Design Bethesda, Maryland 10-11 September 2013. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 1564-1574. PMID 28153204 DOI: 10.1038/mt.2014.148  0.426
2014 Kohn DB. Eliminating SCID row: new approaches to SCID. Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program. 2014: 475-80. PMID 25696897 DOI: 10.1182/Asheducation-2014.1.475  0.515
2014 Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, ... ... Kohn DB, et al. A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. The New England Journal of Medicine. 371: 1407-17. PMID 25295500 DOI: 10.1056/Nejmoa1404588  0.528
2014 Corrigan-Curay J, Kiem HP, Baltimore D, O'Reilly M, Brentjens RJ, Cooper L, Forman S, Gottschalk S, Greenberg P, Junghans R, Heslop H, Jensen M, Mackall C, June C, Press O, ... ... Kohn DB, et al. T-cell immunotherapy: looking forward. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 1564-74. PMID 25186558 DOI: 10.1038/Mt.2014.148  0.525
2014 Kohn DB, Hollis RP. Envelope, please. And the award goes to…. Blood. 124: 1203-4. PMID 25147372 DOI: 10.1182/Blood-2014-06-583823  0.517
2014 Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich AH, Jyonouchi S, Sullivan KE, Small TN, Burroughs L, Skoda-Smith S, Haight AE, ... ... Kohn DB, et al. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. The New England Journal of Medicine. 371: 434-46. PMID 25075835 DOI: 10.1056/Nejmoa1401177  0.359
2014 Gschweng EH, McCracken MN, Kaufman ML, Ho M, Hollis RP, Wang X, Saini N, Koya RC, Chodon T, Ribas A, Witte ON, Kohn DB. HSV-sr39TK positron emission tomography and suicide gene elimination of human hematopoietic stem cells and their progeny in humanized mice. Cancer Research. 74: 5173-83. PMID 25038231 DOI: 10.1158/0008-5472.Can-14-0376  0.521
2014 Carbonaro Sarracino D, Tarantal AF, Lee CC, Martinez M, Jin X, Wang X, Hardee CL, Geiger S, Kahl CA, Kohn DB. Effects of vector backbone and pseudotype on lentiviral vector-mediated gene transfer: studies in infant ADA-deficient mice and rhesus monkeys. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 1803-16. PMID 24925206 DOI: 10.1038/Mt.2014.88  0.622
2014 O'Reilly M, Federoff HJ, Fong Y, Kohn DB, Patterson AP, Ahmed N, Asokan A, Boye SE, Crystal RG, De Oliveira S, Gargiulo L, Harper SQ, Ikeda Y, Jambou R, Montgomery M, et al. Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013. Human Gene Therapy. 25: 488-97. PMID 24773122 DOI: 10.1089/Hum.2014.045  0.366
2014 Kohn LA, Seet CS, Scholes J, Codrea F, Chan R, Zaidi-Merchant S, Zhu Y, De Oliveira S, Kapoor N, Shah A, Abdel-Azim H, Kohn DB, Crooks GM. Human lymphoid development in the absence of common γ-chain receptor signaling. Journal of Immunology (Baltimore, Md. : 1950). 192: 5050-8. PMID 24771849 DOI: 10.4049/Jimmunol.1303496  0.402
2014 Kim WS, Zhu Y, Deng Q, Chin CJ, He CB, Grieco AJ, Dravid GG, Parekh C, Hollis RP, Lane TF, Bouhassira EE, Kohn DB, Crooks GM. Erythropoiesis from human embryonic stem cells through erythropoietin-independent AKT signaling. Stem Cells (Dayton, Ohio). 32: 1503-14. PMID 24677652 DOI: 10.1002/Stem.1677  0.418
2014 Chodon T, Comin-Anduix B, Chmielowski B, Koya RC, Wu Z, Auerbach M, Ng C, Avramis E, Seja E, Villanueva A, McCannel TA, Ishiyama A, Czernin J, Radu CG, Wang X, ... ... Kohn DB, et al. Adoptive transfer of MART-1 T-cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanoma. Clinical Cancer Research : An Official Journal of the American Association For Cancer Research. 20: 2457-65. PMID 24634374 DOI: 10.1158/1078-0432.Ccr-13-3017  0.569
2014 Joglekar AV, Stein L, Ho M, Hoban MD, Hollis RP, Kohn DB. Dissecting the mechanism of histone deacetylase inhibitors to enhance the activity of zinc finger nucleases delivered by integrase-defective lentiviral vectors. Human Gene Therapy. 25: 599-608. PMID 24568341 DOI: 10.1089/Hum.2013.211  0.73
2014 Gschweng E, De Oliveira S, Kohn DB. Hematopoietic stem cells for cancer immunotherapy. Immunological Reviews. 257: 237-49. PMID 24329801 DOI: 10.1111/Imr.12128  0.488
2014 Shearer WT, Dunn E, Notarangelo LD, Dvorak CC, Puck JM, Logan BR, Griffith LM, Kohn DB, O'Reilly RJ, Fleisher TA, Pai SY, Martinez CA, Buckley RH, Cowan MJ. Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience. The Journal of Allergy and Clinical Immunology. 133: 1092-8. PMID 24290292 DOI: 10.1016/J.Jaci.2013.09.044  0.424
2014 Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, ... ... Kohn DB, et al. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 607-22. PMID 24256635 DOI: 10.1038/Mt.2013.265  0.787
2014 Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJ, Boyle M, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, et al. Primary Immune Deficiency Treatment Consortium (PIDTC) report. The Journal of Allergy and Clinical Immunology. 133: 335-47. PMID 24139498 DOI: 10.1016/J.Jaci.2013.07.052  0.37
2014 Oliveira SND, Ryan C, Wherley J, Tu A, Elashoff D, Cooper LJ, Hollis RP, Kohn DB. Abstract B70: Modification of hematopoietic stem cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy Cancer Research. 74. DOI: 10.1158/1538-7445.Pedcan-B70  0.418
2014 Kuo CY, Joglekar A, Kohn DB. Patient Specific Targeted Gene Therapy In The Treatment Of X-Linked Hyper-IgM Syndrome Journal of Allergy and Clinical Immunology. 133: AB162. DOI: 10.1016/J.Jaci.2013.12.588  0.671
2014 Pai S, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich A, Jyonouchi S, Small T, Burroughs L, Haight AE, Pulsipher MA, Chan KW, ... ... Kohn DB, et al. Retrospective Study of 240 Patients with Severe Combined Immunodeficiency Transplanted from 2000-2009: A Report from the Primary Immune Deficiency Treatment Consortium of North America Biology of Blood and Marrow Transplantation. 20: S24-S25. DOI: 10.1016/J.Bbmt.2013.12.007  0.316
2013 Stoyanova T, Cooper AR, Drake JM, Liu X, Armstrong AJ, Pienta KJ, Zhang H, Kohn DB, Huang J, Witte ON, Goldstein AS. Prostate cancer originating in basal cells progresses to adenocarcinoma propagated by luminal-like cells. Proceedings of the National Academy of Sciences of the United States of America. 110: 20111-6. PMID 24282295 DOI: 10.1073/Pnas.1320565110  0.633
2013 De Oliveira SN, Ryan C, Giannoni F, Hardee CL, Tremcinska I, Katebian B, Wherley J, Sahaghian A, Tu A, Grogan T, Elashoff D, Cooper LJ, Hollis RP, Kohn DB. Modification of hematopoietic stem/progenitor cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy. Human Gene Therapy. 24: 824-39. PMID 23978226 DOI: 10.1089/Hum.2012.202  0.549
2013 Awe JP, Lee PC, Ramathal C, Vega-Crespo A, Durruthy-Durruthy J, Cooper A, Karumbayaram S, Lowry WE, Clark AT, Zack JA, Sebastiano V, Kohn DB, Pyle AD, Martin MG, Lipshutz GS, et al. Generation and characterization of transgene-free human induced pluripotent stem cells and conversion to putative clinical-grade status. Stem Cell Research & Therapy. 4: 87. PMID 23890092 DOI: 10.1186/Scrt246  0.712
2013 Romero Z, Urbinati F, Geiger S, Cooper AR, Wherley J, Kaufman ML, Hollis RP, de Assin RR, Senadheera S, Sahagian A, Jin X, Gellis A, Wang X, Gjertson D, Deoliveira S, ... ... Kohn DB, et al. β-globin gene transfer to human bone marrow for sickle cell disease. The Journal of Clinical Investigation. PMID 23863630 DOI: 10.1172/Jci67930  0.859
2013 Joglekar AV, Hollis RP, Kuftinec G, Senadheera S, Chan R, Kohn DB. Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 1705-17. PMID 23857176 DOI: 10.1038/Mt.2013.106  0.698
2013 Dvorak CC, Cowan MJ, Logan BR, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Shearer WT, O'Reilly RJ, Fleisher TA, Pai SY, Hanson IC, Pulsipher MA, Fuleihan R, Filipovich A, et al. The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901. Journal of Clinical Immunology. 33: 1156-64. PMID 23818196 DOI: 10.1007/S10875-013-9917-Y  0.386
2013 Kwan A, Church JA, Cowan MJ, Agarwal R, Kapoor N, Kohn DB, Lewis DB, McGhee SA, Moore TB, Stiehm ER, Porteus M, Aznar CP, Currier R, Lorey F, Puck JM. Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: results of the first 2 years. The Journal of Allergy and Clinical Immunology. 132: 140-50. PMID 23810098 DOI: 10.1016/J.Jaci.2013.04.024  0.364
2013 O'Reilly M, Kohn DB, Bartlett J, Benson J, Brooks PJ, Byrne BJ, Camozzi C, Cornetta K, Crystal RG, Fong Y, Gargiulo L, Gopal-Srivastava R, High KA, Jacobson SG, Jambou RC, et al. Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Human Gene Therapy. 24: 355-62. PMID 23517518 DOI: 10.1089/Hum.2013.064  0.367
2013 Giannoni F, Hardee CL, Wherley J, Gschweng E, Senadheera S, Kaufman ML, Chan R, Bahner I, Gersuk V, Wang X, Gjertson D, Baltimore D, Witte ON, Economou JS, Ribas A, ... Kohn DB, et al. Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 1044-54. PMID 23380815 DOI: 10.1038/Mt.2013.8  0.616
2013 De Oliveira SN, Wang J, Ryan C, Morrison SL, Kohn DB, Hollis RP. A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors. Journal of Translational Medicine. 11: 23. PMID 23360526 DOI: 10.1186/1479-5876-11-23  0.426
2013 McCracken MN, Gschweng EH, Nair-Gill E, McLaughlin J, Cooper AR, Riedinger M, Cheng D, Nosala C, Kohn DB, Witte ON. Long-term in vivo monitoring of mouse and human hematopoietic stem cell engraftment with a human positron emission tomography reporter gene. Proceedings of the National Academy of Sciences of the United States of America. 110: 1857-62. PMID 23319634 DOI: 10.1073/Pnas.1221840110  0.692
2013 Harrison F, Yeagy BA, Rocca CJ, Kohn DB, Salomon DR, Cherqui S. Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 433-44. PMID 23089735 DOI: 10.1038/Mt.2012.214  0.5
2013 Kohn DB, Pai SY, Sadelain M. Gene therapy through autologous transplantation of gene-modified hematopoietic stem cells. Biology of Blood and Marrow Transplantation : Journal of the American Society For Blood and Marrow Transplantation. 19: S64-9. PMID 23032601 DOI: 10.1016/J.Bbmt.2012.09.021  0.525
2013 Hacein-Bey-Abina S, Bushman F, Armant M, Blanche S, Blondeau J, Caccavelli L, Daley H, Dansereau C, deOliveira S, Guo D, Hopkins G, Lim A, Sci M, London WB, Malani N, ... ... Kohn DB, et al. Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector Blood. 122: 715-715. DOI: 10.1182/Blood.V122.21.715.715  0.68
2013 Hoban MD, Joglekar AV, Gray D, Kaufman ML, Urbinati F, Senadheera S, Cost G, Reik A, Holmes MC, Gregory PD, Hollis RP, Kohn DB. Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34+ Cells Blood. 122: 2904-2904. DOI: 10.1182/Blood.V122.21.2904.2904  0.851
2013 Gschweng EH, McCracken MN, Chodon T, Koya RC, Kaufman ML, Ho M, Saini N, Hollis RP, Ribas A, De Oliveira SN, Witte ON, Kohn DB. A Pre-Clinical Model Of Hematopoietic Stem Cell Based Immunotherapy For Cancer Utilizing The NY-ESO-1 T-Cell Receptor and sr39TK PET Reporter / Suicide Gene Blood. 122: 2020-2020. DOI: 10.1182/Blood.V122.21.2020.2020  0.559
2013 Larson SM, Tu A, Senadheera S, Ho M, Kohn DB, deOliveira S. Anti-CD19 Chimeric Antigen Receptor Controlled By The Suicide Gene HSVsr39TK In Hematopoietic Stem Cells For Immunotherapy Of B-Lineage Malignancies Blood. 122: 1659-1659. DOI: 10.1182/Blood.V122.21.1659.1659  0.588
2013 Kim WS, Zhu Y, Deng Q, Grieco AJ, Dravid GG, Parekh C, Hollis RP, Lane T, Bouhassira EE, Kohn DB, Crooks GM. Inducing Definitive Erythropoiesis From Human Embryonic Stem Cells Through a Novel Intracellular MPL Dimerization Strategy Blood. 122: 1172-1172. DOI: 10.1182/Blood.V122.21.1172.1172  0.457
2013 Kuftinec G, Wherley J, Kohn DB. Gene Therapy of Genetic Diseases of Blood Cells Reviews in Cell Biology and Molecular Medicine. DOI: 10.1002/3527600906.Mcb.201200019  0.503
2012 Tarantal AF, Giannoni F, I Lee CC, Wherley J, Sumiyoshi T, Martinez M, Kahl CA, Elashoff D, Louie SG, Kohn DB. Nonmyeloablative Conditioning Regimen to Increase Engraftment of Gene-modified Hematopoietic Stem Cells in Young Rhesus Monkeys. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 1033-1045. PMID 28160620 DOI: 10.1038/mt.2011.312  0.46
2012 Karumbayaram S, Lee P, Azghadi SF, Cooper AR, Patterson M, Kohn DB, Pyle A, Clark A, Byrne J, Zack JA, Plath K, Lowry WE. From skin biopsy to neurons through a pluripotent intermediate under Good Manufacturing Practice protocols. Stem Cells Translational Medicine. 1: 36-43. PMID 23197638 DOI: 10.5966/Sctm.2011-0001  0.658
2012 Candotti F, Shaw KL, Muul L, Carbonaro D, Sokolic R, Choi C, Schurman SH, Garabedian E, Kesserwan C, Jagadeesh GJ, Fu PY, Gschweng E, Cooper A, Tisdale JF, Weinberg KI, ... ... Kohn DB, et al. Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood. 120: 3635-46. PMID 22968453 DOI: 10.1182/Blood-2012-02-400937  0.768
2012 Carbonaro DA, Jin X, Wang X, Yu XJ, Rozengurt N, Kaufman ML, Wang X, Gjertson D, Zhou Y, Blackburn MR, Kohn DB. Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction. Blood. 120: 3677-87. PMID 22833548 DOI: 10.1182/Blood-2012-02-408591  0.481
2012 Corrigan-Curay J, Cohen-Haguenauer O, O'Reilly M, Ross SR, Fan H, Rosenberg N, Somia N, King N, Friedmann T, Dunbar C, Aiuti A, Naldini L, Baum C, von Kalle C, Kiem HP, ... ... Kohn DB, et al. Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 1084-94. PMID 22652996 DOI: 10.1038/Mt.2012.93  0.408
2012 Tarantal AF, Giannoni F, Lee CC, Wherley J, Sumiyoshi T, Martinez M, Kahl CA, Elashoff D, Louie SG, Kohn DB. Nonmyeloablative conditioning regimen to increase engraftment of gene-modified hematopoietic stem cells in young rhesus monkeys. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 1033-45. PMID 22294147 DOI: 10.1038/Mt.2011.312  0.55
2012 Parekh C, Sahaghian A, Kim W, Scholes J, Ge S, Zhu Y, Asgharzadeh S, Hollis R, Kohn D, Ji L, Malvar J, Wang X, Crooks G. Novel pathways to erythropoiesis induced by dimerization of intracellular C-Mpl in human hematopoietic progenitors. Stem Cells (Dayton, Ohio). 30: 697-708. PMID 22290824 DOI: 10.1002/Stem.1046  0.421
2012 Logan AC, Czechowicz A, Kelley BV, Thway TM, Magana I, Krampf MR, Poyser J, Hollis RP, Kohn DB, Weissman IL, Shizuru JA. Anti-CD117 (c-Kit) Monoclonal Antibodies Deplete Human Hematopoietic Stem Cells and Facilitate Their Replacement in Humanized NOD/SCID/IL2Rγ−/− Mice: A Non-Toxic Conditioning Regimen for Allotransplantation Blood. 120: 4099-4099. DOI: 10.1182/Blood.V120.21.4099.4099  0.494
2012 Kim WS, Dravid GG, Zhu Y, Parekh C, Deng Q, Hollis RP, Lane T, Kohn DB. Efficient Erythropoiesis From Human Embryonic Stem Cells Through Dimerization of Intracellular MPL. Blood. 120: 2291-2291. DOI: 10.1182/Blood.V120.21.2291.2291  0.446
2012 Baldwin KM, Urbinati F, Romero-Garcia Z, Kohn DB. Increasing Transduction Efficiency in Human Hematopoietic Stem Cells for Gene Therapy Blood. 120: 2052-2052. DOI: 10.1182/Blood.V120.21.2052.2052  0.845
2011 Kohn DB, Dotti G, Brentjens R, Savoldo B, Jensen M, Cooper LJ, June CH, Rosenberg S, Sadelain M, Heslop HE. CARs on Track in the Clinic: Workshop of the Blood and Marrow Transplant Clinical Trials Network Subcommittee on Cell and Gene Therapy Washington DC, 18 May 2010. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 432-438. PMID 28160821 DOI: 10.1038/mt.2011.1  0.308
2011 Vatakis DN, Koya RC, Nixon CC, Wei L, Kim SG, Avancena P, Bristol G, Baltimore D, Kohn DB, Ribas A, Radu CG, Galic Z, Zack JA. Antitumor activity from antigen-specific CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells. Proceedings of the National Academy of Sciences of the United States of America. 108: E1408-16. PMID 22123951 DOI: 10.1073/Pnas.1115050108  0.606
2011 Shaw KL, Kohn DB. A tale of two SCIDs. Science Translational Medicine. 3: 97ps36. PMID 21865536 DOI: 10.1126/Scitranslmed.3002594  0.455
2011 Cooper AR, Patel S, Senadheera S, Plath K, Kohn DB, Hollis RP. Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration. Journal of Virological Methods. 177: 1-9. PMID 21784103 DOI: 10.1016/J.Jviromet.2011.06.019  0.624
2011 Sokolic R, Maric I, Kesserwan C, Garabedian E, Hanson IC, Dodds M, Buckley R, Issekutz AC, Kamani N, Shaw K, Tan B, Bali P, Hershfield MS, Kohn DB, Wayne AS, et al. Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency. Blood. 118: 2688-94. PMID 21725047 DOI: 10.1182/Blood-2011-01-329359  0.385
2011 Kohn DB, Dotti G, Brentjens R, Savoldo B, Jensen M, Cooper LJ, June CH, Rosenberg S, Sadelain M, Heslop HE. CARs on track in the clinic. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 432-8. PMID 21358705 DOI: 10.1038/Mt.2011.1  0.418
2011 Horwitz EM, Horowitz MM, DiFronzo NL, Kohn DB, Heslop HE. Guidance for developing phase II cell therapy trial proposals for consideration by the Blood and Marrow Transplant Clinical Trials Network. Biology of Blood and Marrow Transplantation : Journal of the American Society For Blood and Marrow Transplantation. 17: 192-6. PMID 21182973 DOI: 10.1016/J.Bbmt.2010.08.020  0.399
2011 Liu R, Zheng L, Dwyre DM, Urbinati F, Kohn DB, Matthews D, Chan J, Satake N. Laser Tweezers Raman Spectroscopy As a Novel Red Blood Cell Functional Assay for Sickle Cell Disease Blood. 118: 4847-4847. DOI: 10.1182/Blood.V118.21.4847.4847  0.806
2011 Oliveira SND, Giannoni F, Hardee C, Sahaghian A, Cooper LJN, Kohn DB. Gene Transfer to Hematopoietic Stem/Progenitor Cells As a Novel Approach for Immunotherapy Against B-Lineage Malignancies: In Vivo Xenograft Model, Blood. 118: 4168-4168. DOI: 10.1182/Blood.V118.21.4168.4168  0.567
2011 Urbinati F, Garcia ZR, Geiger S, de Assin RR, Kuftinec G, Hollis RP, Oliveira SND, Wherley J, Cooper A, Townes TM, Crooks GM, Read EJ, Walters MC, Meiselman HJ, Coates TD, ... ... Kohn DB, et al. Preclinical Studies for Sickle Cell Disease Gene Therapy Using Bone Marrow CD34+ Cells Modified with a βAS3-Globin Lentiviral Vector Blood. 118: 3119-3119. DOI: 10.1182/Blood.V118.21.3119.3119  0.867
2011 Pai S, Notarangelo LD, Harris C, Cattaneo F, Wladkowski M, Armant M, Kao GS, Wielgosz M, Nienhuis AW, Modlich U, Brugman M, Schambach A, Rothe M, Thornhill SI, Gaspar HB, ... ... Kohn DB, et al. Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient Blood. 118: 164-164. DOI: 10.1182/Blood.V118.21.164.164  0.7
2010 Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V, Crooks GM, Kohn DB, Gregory PD, Holmes MC, Cannon PM. Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nature Biotechnology. 28: 839-47. PMID 20601939 DOI: 10.1038/Nbt.1663  0.422
2010 Becker PS, Taylor JA, Trobridge GD, Zhao X, Beard BC, Chien S, Adair J, Kohn DB, Wagner JE, Shimamura A, Kiem HP. Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector. Gene Therapy. 17: 1244-52. PMID 20485382 DOI: 10.1038/Gt.2010.62  0.514
2010 Pais E, Park J, Alexy T, Nikolian V, Ge S, Shaw K, Senadheera S, Hardee CL, Skelton D, Hollis R, Crooks GM, Kohn DB. Regulated expansion of human pancreatic beta-cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 1389-96. PMID 20389286 DOI: 10.1038/Mt.2010.63  0.374
2010 Shah AJ, Kohn DB. Neurocognitive function of patients with severe combined immunodeficiency. Immunology and Allergy Clinics of North America. 30: 143-51. PMID 20113891 DOI: 10.1016/J.Iac.2009.10.003  0.378
2010 Kohn DB. Update on gene therapy for immunodeficiencies. Clinical Immunology (Orlando, Fla.). 135: 247-54. PMID 20071242 DOI: 10.1016/J.Clim.2009.12.003  0.51
2010 Oliveira SND, Wang J, Hollis RP, Kohn DB. CD19 Fc-Fusion Protein for Detection of Cells Expressing Anti-CD19 Chimeric Antigen Receptors. Blood. 116: 3756-3756. DOI: 10.1182/Blood.V116.21.3756.3756  0.43
2010 Ikeda AK, Kohn DB. Hematopoietic Stem Cell Transplantation and Gene Therapy for Primary Immune Deficiency Diseases Pediatric Allergy: Principles and Practice Expert Consult: Second Edition. 223-231. DOI: 10.1016/B978-1-4377-0271-2.00020-1  0.411
2009 Griffith LM, Cowan MJ, Notarangelo LD, Puck JM, Buckley RH, Candotti F, Conley ME, Fleisher TA, Gaspar HB, Kohn DB, Ochs HD, O'Reilly RJ, Rizzo JD, Roifman CM, Small TN, et al. Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management. The Journal of Allergy and Clinical Immunology. 124: 1152-60.e12. PMID 20004776 DOI: 10.1016/J.Jaci.2009.10.022  0.346
2009 Sumiyoshi T, Holt NG, Hollis RP, Ge S, Cannon PM, Crooks GM, Kohn DB. Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system. Human Gene Therapy. 20: 1607-26. PMID 19689196 DOI: 10.1089/Hum.2009.109  0.569
2009 Shaw KL, Pais E, Ge S, Hardee C, Skelton D, Hollis RP, Crooks GM, Kohn DB. Lentiviral vectors with amplified beta cell-specific gene expression. Gene Therapy. 16: 998-1008. PMID 19440227 DOI: 10.1038/Gt.2009.49  0.41
2009 Sondergaard CS, Haldrup C, Beer C, Andersen B, Kohn DB, Pedersen L. Preloading potential of retroviral vectors is packaging cell clone dependent and centrifugation onto CH-296 ensures highest transduction efficiency. Human Gene Therapy. 20: 337-49. PMID 19281432 DOI: 10.1089/Hum.2008.140  0.43
2009 Kohn DB, Candotti F. Gene therapy fulfilling its promise. The New England Journal of Medicine. 360: 518-21. PMID 19179320 DOI: 10.1056/Nejme0809614  0.561
2009 Gruber TA, Shah AJ, Hernandez M, Crooks GM, Abdel-Azim H, Gupta S, McKnight S, White D, Kapoor N, Kohn DB. Clinical and genetic heterogeneity in Omenn syndrome and severe combined immune deficiency. Pediatric Transplantation. 13: 244-50. PMID 18822103 DOI: 10.1111/J.1399-3046.2008.00970.X  0.34
2009 Lin M, Epport K, Azen C, Parkman R, Kohn DB, Shah AJ. Long-term neurocognitive function of pediatric patients with severe combined immune deficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT). Journal of Clinical Immunology. 29: 231-7. PMID 18807155 DOI: 10.1007/S10875-008-9250-Z  0.431
2009 Sokolic R, Podsakoff G, Muul L, Engel B, Jagadeesh J, Garabedian E, Kesserwan C, Carbonaro D, Choi YC, Shaw K, Hershfield M, Wayne A, Kohn D, Candotti F. Immune Reconstitution After Gene Therapy (GTx) for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID) Biology of Blood and Marrow Transplantation. 15: 27. DOI: 10.1016/J.Bbmt.2008.12.081  0.342
2008 Griffith LM, Cowan MJ, Kohn DB, Notarangelo LD, Puck JM, Schultz KR, Buckley RH, Eapen M, Kamani NR, O'Reilly RJ, Parkman R, Roifman CM, Sullivan KE, Filipovich AH, Fleisher TA, et al. Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. The Journal of Allergy and Clinical Immunology. 122: 1087-96. PMID 18992926 DOI: 10.1016/J.Jaci.2008.09.045  0.372
2008 Chhabra A, Yang L, Wang P, Comin-Anduix B, Das R, Chakraborty NG, Ray S, Mehrotra S, Yang H, Hardee CL, Hollis R, Dorsky DI, Koya R, Kohn DB, Ribas A, et al. CD4+CD25- T cells transduced to express MHC class I-restricted epitope-specific TCR synthesize Th1 cytokines and exhibit MHC class I-restricted cytolytic effector function in a human melanoma model. Journal of Immunology (Baltimore, Md. : 1950). 181: 1063-70. PMID 18606658 DOI: 10.4049/Jimmunol.181.2.1063  0.55
2008 Shah AJ, Epport K, Azen C, Killen R, Wilson K, De Clerck D, Crooks G, Kapoor N, Kohn DB, Parkman R, Weinberg KI. Progressive declines in neurocognitive function among survivors of hematopoietic stem cell transplantation for pediatric hematologic malignancies. Journal of Pediatric Hematology/Oncology. 30: 411-8. PMID 18525456 DOI: 10.1097/Mph.0B013E318168E750  0.333
2008 Bauer G, Dao MA, Case SS, Meyerrose T, Wirthlin L, Zhou P, Wang X, Herrbrich P, Arevalo J, Csik S, Skelton DC, Walker J, Pepper K, Kohn DB, Nolta JA. In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 1308-15. PMID 18461052 DOI: 10.1182/Blood.V110.11.2595.2595  0.472
2008 Bonde J, Wirthlin L, Kohn DB, Nolta JA. Human hematopoietic cell culture, transduction, and analyses. Current Protocols in Human Genetics / Editorial Board, Jonathan L. Haines ... [Et Al.]. Unit 13.7. PMID 18428422 DOI: 10.1002/0471142905.Hg1307S56  0.456
2008 Singh H, Manuri PR, Olivares S, Dara N, Dawson MJ, Huls H, Hackett PB, Kohn DB, Shpall EJ, Champlin RE, Cooper LJ. Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system. Cancer Research. 68: 2961-71. PMID 18413766 DOI: 10.1158/0008-5472.Can-07-5600  0.462
2008 Kahl CA, Cannon PM, Oldenburg J, Tarantal AF, Kohn DB. Tissue-specific restriction of cyclophilin A-independent HIV-1- and SIV-derived lentiviral vectors. Gene Therapy. 15: 1079-89. PMID 18385767 DOI: 10.1038/Gt.2008.50  0.341
2008 Carbonaro DA, Jin X, Cotoi D, Mi T, Yu XJ, Skelton DC, Dorey F, Kellems RE, Blackburn MR, Kohn DB. Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion. Blood. 111: 5745-54. PMID 18356486 DOI: 10.1182/Blood-2007-08-103663  0.447
2008 Abdel-Azim H, Zhu Y, Hollis R, Wang X, Ge S, Hao QL, Smbatyan G, Kohn DB, Rosol M, Crooks GM. Expansion of multipotent and lymphoid-committed human progenitors through intracellular dimerization of Mpl. Blood. 111: 4064-74. PMID 18174381 DOI: 10.1182/Blood-2007-08-107466  0.462
2008 Kohn DB. Gene therapy for childhood immunological diseases. Bone Marrow Transplantation. 41: 199-205. PMID 17994122 DOI: 10.1038/Sj.Bmt.1705895  0.537
2008 Taylor JA, Vojtech L, Bahner I, Kohn DB, Laer DV, Russell DW, Richard RE. Foamy virus vectors expressing anti-HIV transgenes efficiently block HIV-1 replication. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 46-51. PMID 17955023 DOI: 10.1038/Sj.Mt.6300335  0.351
2008 Sumiyoshi T, Hollis RP, Holt N, Kohn DB. Optimization of the Sleeping Beauty Transposon System to Achieve Stable Transgene Expression in Human CD34+ Hematopoietic Progenitor Cells Blood. 112: 3527-3527. DOI: 10.1182/Blood.V112.11.3527.3527  0.537
2008 Podsakoff G, Engel BC, Sokolic R, Carbonaro DA, Muul L, Garabedian E, Ireland J, Hershfield M, Wayne A, Dunbar C, Candotti F, Kohn DB. 68: Gene Therapy for Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Comparative Results with or without PEG-ADA Withdrawal and Myelosuppressive Chemotherapy Biology of Blood and Marrow Transplantation. 14: 27-28. DOI: 10.1016/J.Bbmt.2007.12.076  0.375
2008 Abdel-Azim H, Zhu Y, Ge S, Kohn DB, Crooks GM. 1: Targeted in vivo Expansion of Human Multipotent and Lymphoid Progenitors Biology of Blood and Marrow Transplantation. 14: 1. DOI: 10.1016/J.Bbmt.2007.12.007  0.345
2007 Rossi JJ, June CH, Kohn DB. Genetic therapies against HIV. Nature Biotechnology. 25: 1444-54. PMID 18066041 DOI: 10.1038/Nbt1367  0.4
2007 Rodriguez RT, Velkey JM, Lutzko C, Seerke R, Kohn DB, O'Shea KS, Firpo MT. Manipulation of OCT4 levels in human embryonic stem cells results in induction of differential cell types. Experimental Biology and Medicine (Maywood, N.J.). 232: 1368-80. PMID 17959850 DOI: 10.3181/0703-Rm-63  0.461
2007 Higashimoto T, Urbinati F, Perumbeti A, Jiang G, Zarzuela A, Chang LJ, Kohn DB, Malik P. The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors. Gene Therapy. 14: 1298-304. PMID 17597793 DOI: 10.1038/sj.gt.3302979  0.784
2007 Shah AJ, Kapoor N, Crooks GM, Weinberg KI, Azim HA, Killen R, Kuo L, Rushing T, Kohn DB, Parkman R. The effects of Campath 1H upon graft-versus-host disease, infection, relapse, and immune reconstitution in recipients of pediatric unrelated transplants. Biology of Blood and Marrow Transplantation : Journal of the American Society For Blood and Marrow Transplantation. 13: 584-93. PMID 17448918 DOI: 10.1016/J.Bbmt.2007.01.076  0.338
2007 Kohn DB. Lentiviral vectors ready for prime-time. Nature Biotechnology. 25: 65-6. PMID 17211402 DOI: 10.1038/Nbt0107-65  0.386
2007 Blumenthal M, Skelton D, Pepper KA, Jahn T, Methangkool E, Kohn DB. Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 183-92. PMID 17164790 DOI: 10.1038/Sj.Mt.6300015  0.452
2007 Bahner I, Sumiyoshi T, Kagoda M, Swartout R, Peterson D, Pepper K, Dorey F, Reiser J, Kohn DB. Lentiviral vector transduction of a dominant-negative Rev gene into human CD34+ hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replication. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 76-85. PMID 17164778 DOI: 10.1038/Sj.Mt.6300025  0.415
2007 Engel BC, Podsakoff GM, Ireland JL, Smogorzewska EM, Carbonaro DA, Wilson K, Shah A, Kapoor N, Sweeney M, Borchert M, Crooks GM, Weinberg KI, Parkman R, Rosenblatt HM, Wu SQ, ... ... Kohn DB, et al. Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report. Blood. 109: 503-6. PMID 16973956 DOI: 10.1182/Blood-2006-06-031476  0.506
2007 Fischer-Lougheed JY, Tarantal AF, Shulkin I, Mitsuhashi N, Kohn DB, Lee CC, Kearns-Jonker M. Gene therapy to inhibit xenoantibody production using lentiviral vectors in non-human primates. Gene Therapy. 14: 49-57. PMID 16886002 DOI: 10.1038/Sj.Gt.3302818  0.395
2007 Sokolic R, Podsakoff G, Muul L, Engel B, Jagadeesh J, Garabedian E, Carbonaro D, Tuschong L, Ireland J, Hershfield M, Tisdale J, Dunbar C, Wayne A, Kohn D, Candotti F. Comparative Results of Gene Therapy for Adenosine Deaminase Deficiency with or without PEG-ADA Withdrawal and Myelosuppressive Chemotherapy. Blood. 110: 501-501. DOI: 10.1182/Blood.V110.11.501.501  0.484
2007 Singh H, Manuri PR, Olivares S, Dara N, Dawson MJ, Huls H, Lee DA, Hackett PB, Kohn DB, Kebriaei P, Shpall EJ, Champlin R, Cooper L. CD19-Specific T Cells for Treatment of Pediatric Acute Lymphocytic Leukemia Using Sleeping Beauty Transposition. Blood. 110: 2820-2820. DOI: 10.1182/Blood.V110.11.2820.2820  0.481
2007 Kohn DB. Gene therapy as salvage Blood. 110: 4. DOI: 10.1182/Blood-2007-04-082875  0.489
2006 Hollis RP, Nightingale SJ, Wang X, Pepper KA, Yu XJ, Barsky L, Crooks GM, Kohn DB. Stable gene transfer to human CD34(+) hematopoietic cells using the Sleeping Beauty transposon. Experimental Hematology. 34: 1333-43. PMID 16982326 DOI: 10.1016/J.Exphem.2006.05.023  0.568
2006 Anilkumar G, Barwe SP, Christiansen JJ, Rajasekaran SA, Kohn DB, Rajasekaran AK. Association of prostate-specific membrane antigen with caveolin-1 and its caveolae-dependent internalization in microvascular endothelial cells: implications for targeting to tumor vasculature. Microvascular Research. 72: 54-61. PMID 16713605 DOI: 10.1016/J.Mvr.2006.03.004  0.322
2006 Carbonaro DA, Jin X, Petersen D, Wang X, Dorey F, Kil KS, Aldrich M, Blackburn MR, Kellems RE, Kohn DB. In vivo transduction by intravenous injection of a lentiviral vector expressing human ADA into neonatal ADA gene knockout mice: a novel form of enzyme replacement therapy for ADA deficiency. Molecular Therapy : the Journal of the American Society of Gene Therapy. 13: 1110-20. PMID 16651028 DOI: 10.1016/J.Ymthe.2006.02.013  0.416
2006 Nightingale SJ, Hollis RP, Pepper KA, Petersen D, Yu XJ, Yang C, Bahner I, Kohn DB. Transient gene expression by nonintegrating lentiviral vectors. Molecular Therapy : the Journal of the American Society of Gene Therapy. 13: 1121-32. PMID 16556511 DOI: 10.1016/J.Ymthe.2006.01.008  0.514
2006 Kahl CA, Tarantal AF, Lee CI, Jimenez DF, Choi C, Pepper K, Petersen D, Fletcher MD, Leapley AC, Fisher J, Burns TS, Ultsch MN, Dorey FJ, Kohn DB. Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector. Experimental Hematology. 34: 369-81. PMID 16543071 DOI: 10.1016/J.Exphem.2005.12.005  0.521
2006 Jang JE, Shaw K, Yu XJ, Petersen D, Pepper K, Lutzko C, Kohn DB. Specific and stable gene transfer to human embryonic stem cells using pseudotyped lentiviral vectors. Stem Cells and Development. 15: 109-17. PMID 16522168 DOI: 10.1089/Scd.2006.15.109  0.48
2006 Unwalla HJ, Li HT, Bahner I, Li MJ, Kohn D, Rossi JJ. Novel Pol II fusion promoter directs human immunodeficiency virus type 1-inducible coexpression of a short hairpin RNA and protein Journal of Virology. 80: 1863-1873. PMID 16439542 DOI: 10.1128/Jvi.80.4.1863-1873.2006  0.308
2006 Mitsuhashi N, Fischer-Lougheed J, Shulkin I, Kleihauer A, Kohn DB, Weinberg KI, Starnes VA, Kearns-Jonker M. Tolerance induction by lentiviral gene therapy with a nonmyeloablative regimen. Blood. 107: 2286-93. PMID 16291598 DOI: 10.1182/Blood-2005-03-1172  0.423
2006 Methangkool E, Blumenthal M, Kohn D. 437 In Vitro Sensitivity Of Hsv-Tk Transduced Cells To Ganciclovir, Acyclovir, And Foscarnet. Journal of Investigative Medicine. 54. DOI: 10.2310/6650.2005.X0004.436  0.529
2006 Shah AJ, Kapoor N, Crooks GM, Kohn DB, Weinberg KI, Killen R, Kuo L, Parkman R. Campath 1H Versus ATG for the Prophylaxis of Graft Versus Host Disease Does Not Increase the Risk of Relapse or Infections. Blood. 108: 2888-2888. DOI: 10.1182/Blood.V108.11.2888.2888  0.332
2006 Shaw KL, Pepper K, Petersen D, Ge S, Kohn DB. 369. Development of an Amplifiable Gene Expression System in Lentivirus Vectors Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.428  0.424
2006 Kahl CA, Cannon PM, Oldenburg J, Kohn DB. 368. Lentiviral Vector Strain of Origin and Target Cell Species-Specific Restriction Factors Influence Transduction Efficiency Molecular Therapy. 13: S140. DOI: 10.1016/J.Ymthe.2006.08.427  0.49
2006 Ireland JL, Podsakoff GM, Kohn DB, Engel BC. 319. Comparison of Serum-Free Media for Optimal Ex Vivo Transduction of Human CD34+ Cells Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.375  0.457
2006 Carbonaro DA, Jin X, Pepper K, Kohn DB. 1090. Enzyme Replacement Therapy with Pegylated Adenosine Deaminase (PEG-ADA) Does Not Impede Immune Reconstitution Following Transplantation of Gene-Corrected Bone Marrow Cells in the Murine Model of ADA-SCID Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.1192  0.496
2006 Carbonaro DA, Jin X, Yu X, Skelton D, Kohn DB. 1089. Neonatal BMT of ADA-Deficient SCID Mice Results in Phenotypic Correction Despite Low Levels of Engraftment and Absence of the Selective T Lymphoid Expansion Seen in γc Gene Knock-Out Mice Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.1191  0.457
2005 Podsakoff GM, Engel BC, Kohn DB. Perspectives on gene therapy for immune deficiencies. Biology of Blood and Marrow Transplantation : Journal of the American Society For Blood and Marrow Transplantation. 11: 972-6. PMID 16338618 DOI: 10.1016/J.Bbmt.2005.08.034  0.472
2005 Shah AJ, Kapoor N, Crooks GM, Parkman R, Weinberg KI, Wilson K, Kohn DB. Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B. Pediatrics. 116: 1022-5. PMID 16199719 DOI: 10.1542/Peds.2005-0867  0.392
2005 Chan B, Wara D, Bastian J, Hershfield MS, Bohnsack J, Azen CG, Parkman R, Weinberg K, Kohn DB. Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). Clinical Immunology (Orlando, Fla.). 117: 133-43. PMID 16112907 DOI: 10.1016/J.Clim.2005.07.006  0.376
2005 Tarantal AF, McDonald RJ, Jimenez DF, Lee CC, O'Shea CE, Leapley AC, Won RH, Plopper CG, Lutzko C, Kohn DB. Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 87-98. PMID 15963924 DOI: 10.1016/J.Ymthe.2005.01.019  0.384
2005 Podsakoff GM, Engel BC, Carbonaro DA, Choi C, Smogorzewska EM, Bauer G, Selander D, Csik S, Wilson K, Betts MR, Koup RA, Nabel GJ, Bishop K, King S, Schmidt M, ... ... Kohn DB, et al. Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 77-86. PMID 15963923 DOI: 10.1016/J.Ymthe.2005.02.024  0.431
2005 Gruber TA, Skelton DC, Kohn DB. Recombinant murine interleukin-12 elicits potent antileukemic immune responses in a murine model of Philadelphia chromosome-positive acute lymphoblastic leukemia. Cancer Gene Therapy. 12: 818-24. PMID 15877085 DOI: 10.1038/Sj.Cgt.7700839  0.347
2005 Lee CC, Jimenez DF, Kohn DB, Tarantal AF. Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta). Human Gene Therapy. 16: 417-25. PMID 15871673 DOI: 10.1089/Hum.2005.16.417  0.483
2005 Kobayashi H, Carbonaro D, Pepper K, Petersen D, Ge S, Jackson H, Shimada H, Moats R, Kohn DB. Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. Molecular Therapy : the Journal of the American Society of Gene Therapy. 11: 776-89. PMID 15851016 DOI: 10.1016/J.Ymthe.2004.10.006  0.398
2005 Jimenez DF, Lee CI, O'Shea CE, Kohn DB, Tarantal AF. HIV-1-derived lentiviral vectors and fetal route of administration on transgene biodistribution and expression in rhesus monkeys. Gene Therapy. 12: 821-30. PMID 15815706 DOI: 10.1038/Sj.Gt.3302464  0.371
2005 Abdel-Azim H, Zhu Y, Hollis R, Wang X, Hao Q, Barsky L, Kohn D. Human Progenitor and Stem Cell Expansion through Selective, Reversible Cytokine Receptor Signaling. Blood. 106: 31-31. DOI: 10.1182/Blood.V106.11.31.31  0.55
2005 Nightingale S, Hollis RP, Pepper K, Kohn DB. 1102. Stable, Non-Viral Gene Transfer to Human CD34+ Hematopoietic Progenitor Cells Using the Sleeping Beauty Transposon Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.650  0.574
2005 Unwalla HJ, Bahner I, Li H, Li M, Kohn D, Rossi JJ, Gokhgauzer Y. 998. The HIV-1 LTR-hsp70 Fusion Promoter Directs HIV-1 Inducible Expression of Anti-Rev siRNA along with HIV-1 Rev Transdominant Mutant RevM10 Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.545  0.394
2005 Taylor J, Bahner I, Vojtech L, Olson E, Allen J, Laer Dv, Kohn DB, Russell DW, Richard RE. 987. Foamy Virus Vectors That Block HIV Infection Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.531  0.369
2005 Blumenthal M, Skelton D, Carbonaro DA, Pepper KA, Kohn DB. 805. Effective Suicide Gene Therapy of Leukemia in a Novel Model of Retroviral Insertion Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.342  0.474
2005 Tarantal AF, Lee CI, Jimenez DF, Gambhir SS, Kohn DB, Rossi JJ, Cherry SR. 743. Lentiviral Vector Gene Transfer in Monkeys: In Vivo. Detection of Gene Expression Longitudinally Using MicroPET and Optical Imaging Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.283  0.352
2005 Lutzko C, Yu X, Senadheera D, Kohn DB. 463. Efficient Lentiviral Gene Transfer and Expression in Human Embryonic Stem Cells Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.003  0.536
2005 Kahl CA, Tarantal AF, Lee CI, Jimenez DF, Choi C, Pepper K, Peterson D, Fletcher MD, Leapley AC, Kohn DB. 462. Busulfan Dose Escalation to Increase Gene Marking of Hematopoietic Stem Cells by Lentiviral Vectors in Infant Rhesus Monkeys Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.002  0.445
2005 Humblet-Baron S, Anover S, Kipp K, Zhu Q, Ye P, Zhang W, Ovechkina Y, Khim S, Astrakhan A, Strom T, Kohn D, Candotti F, Vyas Y, Ochs H, Miao C, et al. 341. Lentiviral Vector-Mediated Gene Therapy as Treatment for Wiskott-Aldrich Syndrome (WAS): Pre-Clinical Studies in Human Cell Lines and WASp -/- Mice Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.344  0.452
2005 Shaw KL, Pepper K, Petersen D, Kaartinen V, Kohn DB. 90. In Vitro and In Vivo Gene Expression of Lentiviral Vectors in CD4+ T Cells Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.068  0.466
2005 Carbonaro DA, Jin X, Petersen D, Kohn DB. 37. A Novel Form of Enzyme Replacement Therapy for ADA-Deficiency: In Vivo Transduction by Neonatal Injection of Lentivirus Expressing ADA Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.037  0.479
2005 Kohn D, Kahl C, Lee C, Jiminez D, Choi C, Pepper K, Petersen D, Fletcher M, Leapley A, Burns T, Ultsch M, Tarantal A. Busulfan dose escalation to increase gene marking of hematopoietic stem cells by lentiviral vectors in infant rhesus monkeys Biology of Blood and Marrow Transplantation. 11: 82. DOI: 10.1016/J.Bbmt.2004.12.243  0.436
2004 Logan AC, Nightingale SJ, Haas DL, Cho GJ, Pepper KA, Kohn DB. Factors influencing the titer and infectivity of lentiviral vectors. Human Gene Therapy. 15: 976-88. PMID 15585113 DOI: 10.1089/Hum.2004.15.976  0.418
2004 Zheng Y, Ryazantsev S, Ohmi K, Zhao HZ, Rozengurt N, Kohn DB, Neufeld EF. Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB. Molecular Genetics and Metabolism. 82: 286-95. PMID 15308126 DOI: 10.1016/J.Ymgme.2004.06.004  0.375
2004 Logan AC, Haas DL, Kafri T, Kohn DB. Integrated self-inactivating lentiviral vectors produce full-length genomic transcripts competent for encapsidation and integration. Journal of Virology. 78: 8421-36. PMID 15280451 DOI: 10.1128/Jvi.78.16.8421-8436.2004  0.343
2004 Kearns-Jonker M, Fischer-Lougheed J, Shulkin I, Kleihauer A, Mitsuhashi N, Kohn DB, Weinberg K, D'Apice AJ, Starnes VA, Cramer DV. Use of lentiviral vectors to induce long-term tolerance to gal(+) heart grafts. Transplantation. 77: 1748-54. PMID 15201677 DOI: 10.1097/01.Tp.0000131174.52424.4A  0.441
2004 Lee CI, Cowan MJ, Kohn DB, Tarantal AF. Simian immunodeficiency virus infection of hematopoietic stem cells and bone marrow stromal cells. Journal of Acquired Immune Deficiency Syndromes (1999). 36: 553-61. PMID 15097297 DOI: 10.1097/00126334-200405010-00002  0.437
2004 Shah AJ, Lenarsky C, Kapoor N, Crooks GM, Kohn DB, Parkman R, Epport K, Wilson K, Weinberg K. Busulfan and cyclophosphamide as a conditioning regimen for pediatric acute lymphoblastic leukemia patients undergoing bone marrow transplantation. Journal of Pediatric Hematology/Oncology. 26: 91-7. PMID 14767194 DOI: 10.1097/00043426-200402000-00004  0.319
2004 Lee CI, Kohn DB, Ekert JE, Tarantal AF. Morphological analysis and lentiviral transduction of fetal monkey bone marrow-derived mesenchymal stem cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. 9: 112-23. PMID 14741784 DOI: 10.1016/J.Ymthe.2003.09.019  0.474
2004 Gruber TA, Skelton DC, Kohn DB. Interleukin 12 in Combination with Tumor Cell Vaccines Elicits Anti-Leukemic Immune Responses in a Murine Model of Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia. Blood. 104: 4482-4482. DOI: 10.1182/Blood.V104.11.4482.4482  0.437
2004 Hollis RP, Nightingale S, Calos MP, Kohn DB. 1037. Site-Specific Genomic Integration in Hematopoietic Cells Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.982  0.534
2004 Jimenez DF, Lee CI, O'Shea CE, Kohn DB, Tarantal AF. 988. Effect of Lentiviral Promoter and Fetal Route of Administration on Transgene Biodistribution and Expression in Rhesus Monkeys (Macaca mulatta) Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.926  0.415
2004 Lee CI, Jimenez DF, Kohn DB, Tarantal AF. 984. Fetal Administration of Lentiviral Vectors and the Potential for Germ Cell Transduction in Rhesus Monkeys (Macaca mulatta) Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.922  0.389
2004 Jin X, Carbonaro DA, Senadherra D, Petersen D, Kwan S, Kohn DB. 905. The endogenous WASp promoter fragments in SIN lentiviral vectors drive expression preferentially in T cells Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.811  0.405
2004 Miao CH, Zhu Q, Zhou L, Humblet-Baron S, Lin D, Kohn DB, Candotti F, Ochs HD, Rawlings DJ. 904. Lentiviral transduction of Wiskott-Aldrich Syndrome (WAS) deficient, t-cells leads to long-term and progressive WAS protein expression and functional reconstitution Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.810  0.521
2004 Kobayashi H, Petersen D, Carbonaro D, Skelton D, Ge S, Luzko C, Cannon P, Dussel MO, Rosol M, Moats R, Shimada H, Kohn DB. 696. Comparative study of lentiviral vector-mediated gene therapy in neonatal and young-adult MPS I mice by a single intravenous injection Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.647  0.402
2004 Zheng Y, Ryazantsev S, Ohmi K, Zhao H, Rozengurt N, Kohn DB, Neufeld EF. 693. Retrovirally Transduced Bone Marrow Has Effect on Brain Pathology in Mouse Model of Mucopolysaccharidosis IIIB Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.608  0.405
2004 Gruber TA, Skelton DC, Pepper K, Peterson D, Kohn DB. 574. Systemic IL-12 Eliminates Pre-Established Leukemia in a Murine Model of Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.504  0.427
2004 Bahner I, Cho G, Engels B, Peterson D, Pepper K, Kohn DB. 545. Lentiviral vector mediated gene transfer of the anti HIV gene revM10 into primitive hematopoietic cells for gene therapy of HIV infection Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.499  0.432
2004 Nightingale S, Hollis RP, Yang C, Bahner I, Pepper KA, Kohn DB. 416. Transient Gene Expression by Non-Integrating Lentiviral (NIL) Vectors Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.435  0.555
2004 Shaw KL, Pepper K, Petersen D, Kohn DB. 408. Developing Lentiviral Vectors for Specific Gene Expression in CD4+ T Cells Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.396  0.463
2004 Carbonaro DA, Jin X, Senadherra D, Pepper K, Petersen D, Yu X, Skelton D, Kohn DB. 177. Lentiviral Vector Mediated ADA Gene Transfer in ADA-Deficient SCID Mice Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.137  0.493
2003 Engel BC, Kohn DB, Podsakoff GM. Update on gene therapy of inherited immune deficiencies. Current Opinion in Molecular Therapeutics. 5: 503-7. PMID 14601519  0.375
2003 Engel BC, Kohn DB. Gene therapy for inborn and acquired immune deficiency disorders. Acta Haematologica. 110: 60-70. PMID 14583666 DOI: 10.1159/000072455  0.413
2003 Goto H, Yang B, Petersen D, Pepper KA, Alfaro PA, Kohn DB, Reynolds CP. Transduction of green fluorescent protein increased oxidative stress and enhanced sensitivity to cytotoxic drugs in neuroblastoma cell lines. Molecular Cancer Therapeutics. 2: 911-7. PMID 14555710  0.338
2003 Smogorzewska EM, Weinberg KI, Kohn DB. [Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments]. Medycyna Wieku Rozwojowego. 7: 27-34. PMID 13130167  0.443
2003 Zheng Y, Rozengurt N, Ryazantsev S, Kohn DB, Satake N, Neufeld EF. Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow. Molecular Genetics and Metabolism. 79: 233-44. PMID 12948739 DOI: 10.1016/S1096-7192(03)00116-1  0.381
2003 Wang X, Rosol M, Ge S, Peterson D, McNamara G, Pollack H, Kohn DB, Nelson MD, Crooks GM. Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging. Blood. 102: 3478-82. PMID 12946998 DOI: 10.1182/Blood-2003-05-1432  0.462
2003 Haas DL, Lutzko C, Logan AC, Cho GJ, Skelton D, Jin Yu X, Pepper KA, Kohn DB. The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells. Journal of Virology. 77: 9439-50. PMID 12915559 DOI: 10.1128/Jvi.77.17.9439-9450.2003  0.513
2003 Kohn DB, Sadelain M, Dunbar C, Bodine D, Kiem HP, Candotti F, Tisdale J, Riviére I, Blau CA, Richard RE, Sorrentino B, Nolta J, Malech H, Brenner M, Cornetta K, et al. American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. 8: 180-7. PMID 12907140 DOI: 10.1016/S1525-0016(03)00212-0  0.512
2003 Kurre P, Morris J, Thomasson B, Kohn DB, Kiem HP. Scaffold attachment region-containing retrovirus vectors improve long-term proviral expression after transplantation of GFP-modified CD34+ baboon repopulating cells. Blood. 102: 3117-9. PMID 12869503 DOI: 10.1182/Blood-2003-03-0962  0.505
2003 Kohn DB, Sadelain M, Glorioso JC. Occurrence of leukaemia following gene therapy of X-linked SCID. Nature Reviews. Cancer. 3: 477-88. PMID 12835668 DOI: 10.1038/Nrc1122  0.425
2003 Kohn DB, Gänsbacher B. Letter to the editors of Nature from the American Society of Gene Therapy (ASGT) and the European Society of Gene Therapy (ESGT). The Journal of Gene Medicine. 5: 641. PMID 12825204 DOI: 10.1002/Jgm.446  0.362
2003 Lutzko C, Senadheera D, Skelton D, Petersen D, Kohn DB. Lentivirus vectors incorporating the immunoglobulin heavy chain enhancer and matrix attachment regions provide position-independent expression in B lymphocytes. Journal of Virology. 77: 7341-51. PMID 12805432 DOI: 10.1128/Jvi.77.13.7341-7351.2003  0.415
2003 Schmidt M, Glimm H, Wissler M, Hoffmann G, Olsson K, Sellers S, Carbonaro D, Tisdale JF, Leurs C, Hanenberg H, Dunbar CE, Kiem HP, Karlsson S, Kohn DB, Williams D, et al. Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing. Annals of the New York Academy of Sciences. 996: 112-21. PMID 12799289 DOI: 10.1111/J.1749-6632.2003.Tb03239.X  0.444
2003 Schmidt M, Carbonaro DA, Speckmann C, Wissler M, Bohnsack J, Elder M, Aronow BJ, Nolta JA, Kohn DB, von Kalle C. Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Nature Medicine. 9: 463-8. PMID 12640448 DOI: 10.1038/Nm844  0.549
2003 Kohn DB, Sadelain M, Glorioso JC. Correction: Occurrence of leukaemia following gene therapy of X-linked SCID Nature Reviews Cancer. 3: 883-883. DOI: 10.1038/Nrc1236  0.381
2003 Kohn D, Podsakoff G, Engel B, Carbonaro D, Smogorzewska M, Choi C, Muul L, Schurman S, Durnhar C, Weinberg K, Candotti F. 156A clicnical trial of gene transfer to bone marrow CD34+ cells from ADA-deficient SCID subjects Biology of Blood and Marrow Transplantation. 9: 112. DOI: 10.1016/S1083-8791(03)80156-8  0.421
2002 Logan AC, Lutzko C, Kohn DB. Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Current Opinion in Biotechnology. 13: 429-36. PMID 12459333 DOI: 10.1016/S0958-1669(02)00346-4  0.515
2002 Grove JE, Lutzko C, Priller J, Henegariu O, Theise ND, Kohn DB, Krause DS. Marrow-derived cells as vehicles for delivery of gene therapy to pulmonary epithelium American Journal of Respiratory Cell and Molecular Biology. 27: 645-651. PMID 12444022 DOI: 10.1165/Rcmb.2002-0056Rc  0.479
2002 Price MA, Case SS, Carbonaro DA, Yu XJ, Petersen D, Sabo KM, Curran MA, Engel BC, Margarian H, Abkowitz JL, Nolan GP, Kohn DB, Crooks GM. Expression from second-generation feline immunodeficiency virus vectors is impaired in human hematopoietic cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. 6: 645-52. PMID 12409263 DOI: 10.1016/S1525-0016(02)90725-2  0.493
2002 Meertens L, Zhao Y, Rosic-Kablar S, Li L, Chan K, Dobson H, Gartley C, Lutzko C, Hopwood J, Kohn D, Kruth S, Hough MR, Dubé ID. In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression. Human Gene Therapy. 13: 1809-20. PMID 12396614 DOI: 10.1089/104303402760372918  0.399
2002 Yates F, Malassis-Séris M, Stockholm D, Bouneaud C, Larousserie F, Noguiez-Hellin P, Danos O, Kohn DB, Fischer A, de Villartay JP, Cavazzana-Calvo M. Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency. Blood. 100: 3942-9. PMID 12393742 DOI: 10.1182/Blood-2002-03-0782  0.532
2002 Kurre P, Morris J, Andrews RG, Kohn DB, Kiem HP. Kinetics of fluorescence expression in nonhuman primates transplanted with GFP retrovirus-modified CD34 cells Molecular Therapy. 6: 83-90. PMID 12095307 DOI: 10.1006/Mthe.2002.0623  0.483
2002 Shah AJ, Kapoor N, Weinberg KI, Crooks GM, Kohn DB, Lenarsky C, Kaufman F, Epport K, Wilson K, Parkman R. Second hematopoietic stem cell transplantation in pediatric patients: overall survival and long-term follow-up. Biology of Blood and Marrow Transplantation : Journal of the American Society For Blood and Marrow Transplantation. 8: 221-8. PMID 12014811 DOI: 10.1053/Bbmt.2002.V8.Pm12014811  0.371
2002 Kohn DB. Adenosine deaminase gene therapy protocol revisited Molecular Therapy. 5: 96-97. PMID 11829515 DOI: 10.1006/Mthe.2002.0535  0.389
2002 O'Rourke JP, Newbound GC, Kohn DB, Olsen JC, Bunnell BA. Comparison of gene transfer efficiencies and gene expression levels achieved with equine infectious anemia virus- and human immunodeficiency virus type 1-derived lentivirus vectors. Journal of Virology. 76: 1510-5. PMID 11773424 DOI: 10.1128/Jvi.76.3.1510-1515.2002  0.453
2002 Gruber TA, Skelton DC, Kohn DB. Requirement for NK cells in CD40 ligand-mediated rejection of Philadelphia chromosome-positive acute lymphoblastic leukemia cells. Journal of Immunology (Baltimore, Md. : 1950). 168: 73-80. PMID 11751948 DOI: 10.4049/Jimmunol.168.1.73  0.483
2001 Weinberg KI, Kapoor N, Shah AJ, Crooks GM, Kohn DB, Parkman R. Hematopoietic stem cell transplantation for severe combined immune deficiency Current Allergy and Asthma Reports. 1: 416-420. PMID 11892067 DOI: 10.1007/S11882-001-0026-2  0.46
2001 Tarantal AF, Lee CI, Ekert JE, McDonald R, Kohn DB, Plopper CG, Case SS, Bunnell BA. Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): Lung-targeting approaches Molecular Therapy. 4: 614-621. PMID 11735346 DOI: 10.1006/Mthe.2001.0497  0.358
2001 Kohn DB. Gene therapy for genetic haematological disorders and immunodeficiencies Journal of Internal Medicine. 249: 379-390. PMID 11298858 DOI: 10.1046/J.1365-2796.2001.00803.X  0.492
2001 Tarantal AF, O'Rourke JP, Case SS, Newbound GC, Li J, Lee CI, Baskin CR, Kohn DB, Bunnell BA. Rhesus monkey model for fetal gene transfer: Studies with retroviral-based vector systems Molecular Therapy. 3: 128-138. PMID 11237669 DOI: 10.1006/Mthe.2000.0255  0.462
2001 Nolta JA, Kohn DB. UNIT 13.7 Human Hematopoietic Cell Culture, Transduction, and Analyses Current Protocols in Human Genetics. DOI: 10.1002/0471142905.Hg1307S14  0.456
2001 Kohn DB. Progress in hematopoietic stem cell gene therapy Cancer Research, Therapy and Control. 11: 41-45.  0.43
2000 Bauer G, Selander D, Engel B, Carbonaro D, Csik S, Rawlings S, Church J, Kohn DB. Gene therapy for pediatric AIDS Annals of the New York Academy of Sciences. 918: 318-329. PMID 11131719 DOI: 10.1111/J.1749-6632.2000.Tb05501.X  0.482
2000 Cavazzana-Calvo M, Hacein-Bey S, De Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, Bousso P, Deist FL, Fischer A, Kohn DB. Gene therapy for XSCID: The first success of gene therapy Pediatric Research. 48: 578. PMID 11044472 DOI: 10.1203/00006450-200011000-00002  0.393
2000 Stripecke R, Cardoso AA, Pepper KA, Skelton DC, Yu XJ, Mascarenhas L, Weinberg KI, Nadler LM, Kohn DB. Lentiviral vectors for efficient delivery of CD80 and granulocyte-macrophage-colony-stimulating factor in human acute lymphoblastic leukemia and acute myeloid leukemia cells to induce antileukemic immune responses Blood. 96: 1317-1326. PMID 10942373 DOI: 10.1182/Blood.V96.4.1317.H8001317_1317_1326  0.488
2000 Engel BC, Bauer G, Pepper KA, Bockstoce DC, Yu XJ, Chen SY, Kohn DB. Intrakines - Evidence for a Trans-Cellular Mechanism of Action Molecular Therapy. 1: 165-170. PMID 10933927 DOI: 10.1006/Mthe.2000.0026  0.449
2000 Haas DL, Case SS, Crooks GM, Kohn DB. Critical factors influencing stable transduction of human CD34(+) cells with HIV-1-derived lentiviral vectors. Molecular Therapy : the Journal of the American Society of Gene Therapy. 2: 71-80. PMID 10899830 DOI: 10.1006/Mthe.2000.0094  0.526
2000 Islam TC, Brandén LJ, Kohn DB, Islam KB, Smith CIE. BTK mediated apoptosis, a possible mechanism for failure to generate high titer retroviral producer clones Journal of Gene Medicine. 2: 204-209. PMID 10894266 DOI: 10.1002/(Sici)1521-2254(200005/06)2:3<204::Aid-Jgm104>3.0.Co;2-5  0.323
2000 Halene S, Kohn DB. Gene therapy using hematopoietic stem cells: Sisyphus approaches the crest Human Gene Therapy. 11: 1259-1267. PMID 10890736 DOI: 10.1089/10430340050032366  0.546
2000 Rosenberg SA, Blaese RM, Brenner MK, Deisseroth AB, Ledley FD, Lotze MT, Wilson JM, Nabel GJ, Cornetta K, Economou JS, Freeman SM, Riddell SR, Brenner M, Oldfield E, Gansbacher B, ... ... Kohn D, et al. Human gene marker/therapy clinical protocols. Human Gene Therapy. 11: 919-79. PMID 10779168 DOI: 10.1089/10430340050015536  0.608
2000 Parkman R, Weinberg K, Nolta J, Kapoor N, Kohn D. Gene Therapy for Adenosine Deaminase Deficiency Annual Review of Medicine. 51: 33-47. PMID 10774451 DOI: 10.1146/Annurev.Med.51.1.33  0.524
2000 Huang MM, Tsuboi S, Wong A, Yu XJ, Oh-Eda M, Derry JM, Francke U, Fukuda M, Weinberg KI, Kohn DB. Expression of human Wiskott-Aldrich syndrome protein in patients' cells leads to partial correction of a phenotypic abnormality of cell surface glycoproteins. Gene Therapy. 7: 314-20. PMID 10694812 DOI: 10.1038/sj.gt.3301085  0.366
2000 Stripecke R, Cardoso AA, Pepper KA, Skelton DC, Yu X, Mascarenhas L, Weinberg KI, Nadler LM, Kohn DB. Lentiviral vectors for efficient delivery of CD80 and granulocyte-macrophage– colony-stimulating factor in human acute lymphoblastic leukemia and acute myeloid leukemia cells to induce antileukemic immune responses Blood. 96: 1317-1326. DOI: 10.1182/BLOOD.V96.4.1317.H8001317_1317_1326  0.387
2000 Kohn DB, Weinberg KI, Parkman R. Gene therapy for T-cell immunodeficiencies Immunology and Allergy Clinics of North America. 20: 221-235. DOI: 10.1016/S0889-8561(05)70143-0  0.482
2000 Kohn DB, Weinberg KI, Parkman R. GENE THERAPY FOR T-CELL IMMUNODEFICIENCIES Immunology and Allergy Clinics of North America. 20: 221-235. DOI: 10.1016/S0889-8561(05)70143-0  0.35
2000 Dao M, Tsark E, Ertl D, Wang X, Kim T, Malik P, Kohn D, Nolta J. Engineered cytokine secretion from human mesenchymal stem cells influences in vivo hematopoiesis Experimental Hematology. 28: 59. DOI: 10.1016/S0301-472X(00)00272-1  0.606
2000 Arevalo JMG, Case SS, Kohn DB, Nolta JA. Effects of allogeneic support cells on human hematopoietic stem cell engraftment in a novel immune deficient mouse transplant system Blood. 96: 172a-173a.  0.389
1999 Halene S, Wang L, Cooper RM, Bockstoce DC, Robbins PB, Kohn DB. Improved expression in hematopoietic and lymphoid cells in mice after transplantation of bone marrow transduced with a modified retroviral vector. Blood. 94: 3349-57. PMID 10552944 DOI: 10.1182/Blood.V94.10.3349.422K05_3349_3357  0.537
1999 Stripecke R, Skelton DC, Pattengale PK, Shimada H, Kohn DB. Combination of CD80 and granulocyte-macrophage colony-stimulating factor coexpression by a leukemia cell vaccine: Preclinical studies in a murine model recapitulating philadelphia chromosome-positive acute lymphoblastic leukemia Human Gene Therapy. 10: 2109-2122. PMID 10498243 DOI: 10.1089/10430349950017103  0.416
1999 Stripecke R, Del Carmen Villacres M, Skelton DC, Satake N, Halene S, Kohn DB. Immune response to green fluorescent protein: Implications for gene therapy Gene Therapy. 6: 1305-1312. PMID 10455440 DOI: 10.1038/Sj.Gt.3300951  0.479
1999 Crooks GM, Fuller J, Petersen D, Izadi P, Malik P, Pattengale PK, Kohn DB, Gasson JC. Constitutive HOXA5 expression inhibits erythropoiesis and increases myelopoiesis from human hematopoietic progenitors. Blood. 94: 519-28. PMID 10397719  0.586
1999 Kohn DB, Bauer G, Rice CR, Rothschild JC, Carbonaro DA, Valdez P, Hao Ql, Zhou C, Bahner I, Kearns K, Brody K, Fox S, Haden E, Wilson K, Salata C, et al. A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34(+) cells from the bone marrow of human immunodeficiency virus-1-infected children. Blood. 94: 368-71. PMID 10381536 DOI: 10.1182/Blood.V94.1.368.413A47_368_371  0.517
1999 Engel BC, Kohn DB. Stem cell directed gene therapy Frontiers in Bioscience : a Journal and Virtual Library. 4: e26-33. PMID 10228093  0.381
1999 Case SS, Price MA, Jordan CT, Yu XJ, Wang L, Bauer G, Haas DL, Xu D, Stripecke R, Naldini L, Kohn DB, Crooks GM. Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors. Proceedings of the National Academy of Sciences of the United States of America. 96: 2988-93. PMID 10077624 DOI: 10.1073/Pnas.96.6.2988  0.493
1999 Dunbar C, Chang L, Mullen C, Ramsey WJ, Carter C, Kohn D, Parkman R, Lenarsky C, Weinberg K, Wara D, Culver KW, Anderson WF, Leitman S, Fleisher T, Klein H, et al. Amendment to Clinical Research Project. Project 90-C-195. April 1, 1993. Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with autologous lymphocytes transduced with a human ADA gene. Human Gene Therapy. 10: 477-88. PMID 10048399 DOI: 10.1089/10430349950018913  0.335
1999 Lutzko C, Kruth S, Abrams-Ogg ACG, Lau K, Li L, Clark BR, Ruedy C, Nanji S, Foster R, Kohn D, Shull R, Dubé ID. Genetically Corrected Autologous Stem Cells Engraft, But Host Immune Responses Limit Their Utility in Canine -L-iduronidase Deficiency Blood. 93: 1895-1905. DOI: 10.1182/Blood.V93.6.1895.406K02_1895_1905  0.501
1998 Bauer G, Sauter S, Ibanez C, Rice CR, Valdez P, Jolly D, Kohn DB. Increased gene transfer into human CD34+ progenitor cells using retroviral vectors produced by a canine packaging cell line. Biology of Blood and Marrow Transplantation : Journal of the American Society For Blood and Marrow Transplantation. 4: 119-27. PMID 9923409 DOI: 10.1053/Bbmt.1998.V4.Pm9923409  0.489
1998 Dunbar CE, Kohn DB, Schiffmann R, Barton NW, Nolta JA, Esplin JA, Pensiero M, Long Z, Lockey C, Emmons RV, Csik S, Leitman S, Krebs CB, Carter C, Brady RO, et al. Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation. Human Gene Therapy. 9: 2629-40. PMID 9853529 DOI: 10.1089/Hum.1998.9.17-2629  0.553
1998 Wang L, Robbins PB, Carbonaro DA, Kohn DB. High-resolution analysis of cytosine methylation in the 5' long terminal repeat of retroviral vectors Human Gene Therapy. 9: 2321-2330. PMID 9829531 DOI: 10.1089/Hum.1998.9.16-2321  0.414
1998 Mascarenhas L, Stripecke R, Case SS, Xu D, Weinberg KI, Kohn DB. Gene delivery to human B-precursor acute lymphoblastic leukemia cells Blood. 92: 3537-3545. PMID 9808545 DOI: 10.1182/Blood.V92.10.3537.422K52_3537_3545  0.521
1998 Weinberg KI, Kohn DB. Gene therapy for congenital lymphoid immunodeficiency diseases Seminars in Hematology. 35: 354-366. PMID 9801264  0.358
1998 Kapoor N, Crooks G, Kohn DB, Parkman R. Hematopoietic stem cell transplantation for primary lymphoid immunodeficiencies Seminars in Hematology. 35: 346-353. PMID 9801263  0.357
1998 Verma S, Woffendin C, Bahner I, Ranga U, Xu L, Yang ZY, King SR, Kohn DB, Nabel GJ. Gene transfer into human umbilical cord blood-derived CD34+ cells by particle-mediated gene transfer Gene Therapy. 5: 692-699. PMID 9797875 DOI: 10.1038/Sj.Gt.3300656  0.545
1998 Stripecke R, Skelton DC, Gruber T, Afar D, Pattengale PK, Witte ON, Kohn DB. Immune response to Philadelphia chromosome-positive acute lymphoblastic leukemia induced by expression of CD80, interleukin 2, and granulocyte-macrophage colony-stimulating factor. Human Gene Therapy. 9: 2049-62. PMID 9759932 DOI: 10.1089/Hum.1998.9.14-2049  0.433
1998 Robbins PB, Skelton DC, Yu XJ, Halene S, Leonard EH, Kohn DB. Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells Proceedings of the National Academy of Sciences of the United States of America. 95: 10182-10187. PMID 9707621 DOI: 10.1073/Pnas.95.17.10182  0.443
1998 Kohn DB, Hershfield MS, Carbonaro D, Shigeoka A, Brooks J, Smogorzewska EM, Barsky LW, Chan R, Burotto F, Annett G, Nolta JA, Crooks G, Kapoor N, Elder M, Wara D, et al. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nature Medicine. 4: 775-80. PMID 9662367 DOI: 10.1038/nm0798-775  0.657
1998 Malik P, Fisher TC, Barsky LL, Zeng L, Izadi P, Hiti AL, Weinberg KI, Coates TD, Meiselman HJ, Kohn DB. An in vitro model of human red blood cell production from hematopoietic progenitor cells. Blood. 91: 2664-71. PMID 9531574 DOI: 10.1182/Blood.V91.8.2664.2664_2664_2671  0.681
1998 De Vos S, Kohn DB, Cho SK, McBride WH, Said JW, Koeffler HP. Immunotherapy against murine leukemia Leukemia. 12: 401-405. PMID 9529135 DOI: 10.1038/Sj.Leu.2400940  0.331
1998 Mascarenhas L, Stripecke R, Case SS, Xu D, Weinberg KI, Kohn DB. Gene Delivery to Human B-Precursor Acute Lymphoblastic Leukemia Cells Blood. 92: 3537-3545. DOI: 10.1182/blood.V92.10.3537.422k52_3537_3545  0.429
1997 Huang MM, Wong A, Yu X, Kakkis E, Kohn DB. Retrovirus-mediated transfer of the human α-L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts Gene Therapy. 4: 1150-1159. PMID 9425437  0.459
1997 Robbins PB, Yu XJ, Skelton DM, Pepper KA, Wasserman RM, Zhu L, Kohn DB. Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells Journal of Virology. 71: 9466-9474. PMID 9371608 DOI: 10.1128/Jvi.71.12.9466-9474.1997  0.457
1997 Bahner I, Kearns K, Coutinho S, Leonard EH, Kohn DB. Infection of human marrow stroma by human immunodeficiency virus-1 (HIV-1) is both required and sufficient for HIV-1-induced hematopoietic suppression in vitro: demonstration by gene modification of primary human stroma. Blood. 90: 1787-98. PMID 9292511 DOI: 10.1182/Blood.V90.5.1787  0.386
1997 Kohn DB, Parkman R. Gene therapy for newborns Faseb Journal. 11: 635-639. PMID 9240965 DOI: 10.1096/Fasebj.11.8.9240965  0.502
1997 Lu Y, Planelles V, Li X, Palaniappan C, Day B, Challita-Eid P, Amado R, Stephens D, Kohn DB, Bakker A, Fay P, Bambara RA, Rosenblatt JD. Inhibition of HIV-1 replication using a mutated tRNALys-3 primer. The Journal of Biological Chemistry. 272: 14523-31. PMID 9169409 DOI: 10.1074/Jbc.272.23.14523  0.304
1997 Bauer G, Valdez P, Kearns K, Bahner I, Wen SF, Zaia JA, Kohn DB. Inhibition of human immunodeficiency virus-1 (HIV-1) replication after transduction of granulocyte colony-stimulating factor-mobilized CD34+ cells from HIV-1-infected donors using retroviral vectors containing anti-HIV-1 genes. Blood. 89: 2259-67. PMID 9116267 DOI: 10.1182/Blood.V89.7.2259  0.357
1997 Kearns K, Bahner I, Bauer G, Wei SF, Valdez P, Wheeler S, Woods L, Miller R, Casciato D, Galpin J, Church J, Kohn DB. Suitability of bone marrow from HIV-1-infected donors for retrovirus-mediated gene transfer. Human Gene Therapy. 8: 301-11. PMID 9048197 DOI: 10.1089/Hum.1997.8.3-301  0.469
1997 Malik P, McQuiston SA, Yu XJ, Pepper KA, Krall WJ, Podsakoff GM, Kurtzman GJ, Kohn DB. Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line. Journal of Virology. 71: 1776-83. PMID 9032306 DOI: 10.1128/Jvi.71.3.1776-1783.1997  0.67
1997 Kohn DB. Gene therapy for haematopoietic and lymphoid disorders Clinical and Experimental Immunology, Supplement. 107: 54-57. PMID 9020937  0.427
1997 Dao MA, Hannum CH, Kohn DB, Nolta JA. FLT3 ligand preserves the ability of human CD34+ progenitors to sustain long-term hematopoiesis in immune-deficient mice after ex vivo retroviral- mediated transduction Blood. 89: 446-456. PMID 9002946 DOI: 10.1182/Blood.V89.2.446  0.438
1997 Nolta JA, Kohn DB. 15 – Haematopoietic stem cells for gene therapy Stem Cells. 447-462. DOI: 10.1016/B978-012563455-7/50016-7  0.556
1996 Kohn DB. Gene therapy for hematopoietic and immune disorders Bone Marrow Transplantation. 18: S55-S58. PMID 8971410  0.494
1996 Kido M, Rich KA, Yang G, Barrón E, Kohn DB, al-Ubaidi MR, Blanks JC, Lang G. Use of a retroviral vector with an internal opsin promoter to direct gene expression to retinal photoreceptor cells. Current Eye Research. 15: 833-44. PMID 8921226 DOI: 10.3109/02713689609017624  0.469
1996 Taylor N, Bacon KB, Smith S, Jahn T, Kadlecek TA, Uribe L, Kohn DB, Gelfand EW, Weiss A, Weinberg K. Reconstitution of T cell receptor signaling in ZAP-70-deficient cells by retroviral transduction of the ZAP-70 gene Journal of Experimental Medicine. 184: 2031-2036. PMID 8920891 DOI: 10.1084/Jem.184.5.2031  0.475
1996 Shull RM, Lu X, McEntee MF, Bright RM, Pepper KA, Kohn DB. Myoblast gene therapy in canine mucopolysaccharidosis. I: Abrogation by an immune response to alpha-L-iduronidase. Human Gene Therapy. 7: 1595-603. PMID 8864760 DOI: 10.1089/Hum.1996.7.13-1595  0.398
1996 Kohn DB, Sarver N. Gene Therapy for HIV-1 Infection Advances in Experimental Medicine and Biology. 394: 421-428. PMID 8815708 DOI: 10.1007/978-1-4757-9209-6_39  0.426
1996 Dunbar PIC, Kohn D, Karlsson SCS, Barton IN, Brady R, Cottler-Fox M, Emmons R, Esplin J, Leitman S, Lenarsky C, Nolta J, Parkman R, Pensiero M, Schifmann R, Tolstoshev P, et al. Retroviral Mediated Transfer of the cDNA for Human Glucocerebrosidase into Hematopoietic Stem Cells of Patients with Gaucher Disease. A Phase I Study. National Institutes of Health, Bethesda, Maryland Human Gene Therapy. 7: 231-253. PMID 8788174 DOI: 10.1089/Hum.1996.7.2-231  0.533
1996 Zhou C, Bahner I, Rossi JJ, Kohn DB. Expression of Hammerhead Ribozymes by Retroviral Vectors to Inhibit HIV-1 Replication: Comparison of RNA Levels and Viral Inhibition Antisense & Nucleic Acid Drug Development. 6: 17-24. PMID 8783792 DOI: 10.1089/Oli.1.1996.6.17  0.336
1996 Bahner I, Kearns K, Hao QL, Smogorzewska EM, Kohn DB. Transduction of human CD34+ hematopoietic progenitor cells by a retroviral vector expressing an RRE decoy inhibits human immunodeficiency virus type 1 replication in myelomonocytic cells produced in long-term culture. Journal of Virology. 70: 4352-60. PMID 8676458 DOI: 10.1128/Jvi.70.7.4352-4360.1996  0.455
1996 Nolta JA, Dao MA, Wells S, Smogorzewska EM, Kohn DB. Transduction of pluripotent human hematopoietic stem cells demonstrated by clonal analysis after engraftment in immune-deficient mice Proceedings of the National Academy of Sciences of the United States of America. 93: 2414-2419. PMID 8637888 DOI: 10.1073/Pnas.93.6.2414  0.509
1996 Jadus, Irwin M, Irwin, Horansky R, Sekhon S, Pepper K, Kohn D, Wepsic H. Macrophages can recognize and kill tumor cells bearing the membrane isoform of macrophage colony-stimulating factor Blood. 87: 5232-5241. DOI: 10.1182/Blood.V87.12.5232.Bloodjournal87125232  0.362
1996 Weinberg KI, Kohn DB. GENE THERAPY FOR CONGENITAL IMMUNODEFICIENCY DISEASES Immunology and Allergy Clinics of North America. 16: 453-476. DOI: 10.1016/S0889-8561(05)70256-3  0.429
1996 Malik P, McQuiston SA, Yu XJ, Pepper KA, Podsakoff GM, Kohn DB. Recombinant adeno-associated vkus (rAAV) médiates a high lavel of gene transfer but a low efficiency of integration in human hematopoietic cells Experimental Hematology. 24: 1054.  0.575
1995 Wells S, Malik P, Pensiero M, Kohn DB, Nolta JA. The presence of an autologous marrow stromal cell layer increases glucocerebrosidase gene transduction of long-term culture initiating cells (LTCICs) from the bone marrow of a patient with Gaucher disease. Gene Therapy. 2: 512-20. PMID 8593601  0.642
1995 Challita P-, Skelton D, El-Khoueiry A, Yu X, Weinberg K, Kohn DB. Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells. Journal of Virology. 69: 748-755. PMID 7815539 DOI: 10.1128/Jvi.69.2.748-755.1995  0.438
1995 Nolta JA, Smogorzewska EM, Kohn DB. Analysis of optimal conditions for retroviral-mediated transduction of primitive human hematopoietic cells Blood. 86: 101-110. PMID 7795215  0.413
1995 Kohn DB. The current status of gene therapy using hematopoietic stem cells. Current Opinion in Pediatrics. 7: 56-63. PMID 7728204 DOI: 10.1097/00008480-199502000-00011  0.522
1995 Malik P, Krall WJ, Yu XJ, Zhou C, Kohn DB. Retroviral-mediated gene expression in human myelomonocytic cells: a comparison of hematopoietic cell promoters to viral promoters. Blood. 86: 2993-3005. PMID 7579392 DOI: 10.1182/Blood.V86.8.2993.2993  0.694
1995 Hao QL, Malik P, Salazar R, Tang H, Gordon EM, Kohn DB. Expression of biologically active human factor IX in human hematopoietic cells after retroviral vector-mediated gene transduction. Human Gene Therapy. 6: 873-80. PMID 7578406 DOI: 10.1089/Hum.1995.6.7-873  0.629
1995 Kohn DB, Weinberg KI, Nolta JA, Heiss LN, Lenarsky C, Crooks GM, Hanley ME, Annett G, Brooks JS, El-Khoureiy A, Lawrence K, Wells S, Moen RC, Bastian J, Williams-Herman DE, et al. Engraftment of gene–modified umbilical cord blood cells in neonates with adenosine deaminase deficiency Nature Medicine. 1: 1017-1023. PMID 7489356 DOI: 10.1038/nm1095-1017  0.403
1995 Nolta J, Smogorzewska E, Kohn D. Analysis of optimal conditions for retroviral-mediated transduction of primitive human hematopoietic cells Blood. 86: 101-110. DOI: 10.1182/Blood.V86.1.101.Bloodjournal861101  0.51
1994 Challita P, Kohn DB. Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. Proceedings of the National Academy of Sciences of the United States of America. 91: 2567-2571. PMID 8146155 DOI: 10.1073/Pnas.91.7.2567  0.478
1994 Chen Z, Bahner IC, Larson GP, Zaia JA, Rossi JJ, Kohn DB. Inhibition of HIV-1 in human T-lymphocytes by retrovirally transduced anti-tat and rev hammerhead ribozymes Gene. 149: 33-39. PMID 7958986 DOI: 10.1016/0378-1119(94)90409-X  0.383
1994 Nolta JA, Hanley MB, Kohn DB. Sustained human hematopoiesis in immunodeficient mice by cotransplantation of marrow stroma expressing human interleukin-3: Analysis of gene transduction of long-lived progenitors Blood. 83: 3041-3051. PMID 7514050  0.404
1994 Krall W, Challita P, Perlmutter L, Skelton D, Kohn D. Cells expressing human glucocerebrosidase from a retroviral vector repopulate macrophages and central nervous system microglia after murine bone marrow transplantation Blood. 83: 2737-2748. DOI: 10.1182/Blood.V83.9.2737.Bloodjournal8392737  0.52
1994 Nolta JA, Hanley MB, Kohn DB. Sustained human hematopoiesis in immunodeficient mice by cotransplantation of marrow stroma expressing human interleukin-3: analysis of gene transduction of long-lived progenitors Blood. 83: 3041-3051. DOI: 10.1182/Blood.V83.10.3041.3041  0.502
1993 Gordon EM, Tang H, Salazar RL, Kohn DB. Expression of coagulation factor IX (Christmas factor) in human hepatoma (HepG2) cell cultures after retroviral vector-mediated transfer. The American Journal of Pediatric Hematology/Oncology. 15: 196-203. PMID 8388667 DOI: 10.1097/00043426-199305000-00007  0.357
1993 Bahner I, Zhou C, Yu XJ, Hao QL, Guatelli JC, Kohn DB. Comparison of trans-dominant inhibitory mutant human immunodeficiency virus type 1 genes expressed by retroviral vectors in human T lymphocytes Journal of Virology. 67: 3199-3207. PMID 8388497 DOI: 10.1128/Jvi.67.6.3199-3207.1993  0.358
1993 Weinberg K, Hershfield MS, Bastian J, Kohn D, Sender L, Parkman R, Lenarsky C. T lymphocyte ontogeny in adenosine deaminase-deficient severe combined immune deficiency after treatment with polyethylene glycol-modified adenosine deaminase. The Journal of Clinical Investigation. 92: 596-602. PMID 8349799 DOI: 10.1172/Jci116626  0.335
1993 Miller AR, Skotzko MJ, Rhoades K, Belldegrun AS, Tso CL, Kaboo R, McBride WH, Jacobs E, Kohn DB, Moen R. Simultaneous use of two retroviral vectors in human gene marking trials: feasibility and potential applications. Human Gene Therapy. 3: 619-24. PMID 1482701 DOI: 10.1089/Hum.1992.3.6-619  0.469
1993 Miller A, McBride W, Dubinett S, Dougherty G, Thacker J, Shau H, Kohn D, Moen R, Walker M, Chiu R. Transduction of human melanoma cell lines with the human interleukin-7 gene using retroviral-mediated gene transfer: comparison of immunologic properties with interleukin-2 Blood. 82: 3686-3694. DOI: 10.1182/Blood.V82.12.3686.Bloodjournal82123686  0.319
1993 Crooks G, Kohn D. Growth Factors Increase Amphotropic Retrovirus Binding to Human CD34+ Bone Marrow Progenitor Cells Blood. 82: 3290-3297. DOI: 10.1182/Blood.V82.11.3290.3290  0.471
1992 Nolta JA, Yu XJ, Bahner I, Kohn DB. Retroviral-mediated transfer of the human glucocerebrosidase gene into cultured gaucher bone marrow Journal of Clinical Investigation. 90: 342-348. PMID 1379609 DOI: 10.1172/Jci115868  0.534
1992 Economou IJS, Figlin RA, Jacobs E, Golub S, DeKernion J, Moen RC, Belldegrun A, Holmes EC, Kohn D, Shau H, McBride WH. The Treatment of Patients with Metastatic Melanoma and Renal Cell Cancer Using In Vitro Expanded and Genetically-Engineered (Neomycin Phosphotransferase) Bulk, CD8(+) and/or CD4(+) Tumor Infiltrating Lymphocytes and Bulk, CD8(+) and/or CD4(+) Peripheral Blood Leukocytes in Combination with Recombinant Interleukin-2 Alone, or with Recombinant Interleukin-2 and Recombinant Alpha Interferon Human Gene Therapy. 3: 411-430. PMID 1326336 DOI: 10.1089/Hum.1992.3.4-411  0.364
1992 Bedgood RM, Bahner I, Kohn DB, Stallcup MR. Two different genes coding for processable and nonprocessable forms of a viral envelope protein can account for the apparent hormonal stimulation of protein processing in W7MG1 lymphoma cells Molecular Endocrinology. 6: 459-467. PMID 1316543 DOI: 10.1210/Mend.6.3.1316543  0.376
1991 Kohn DB, Nolta JA, Weinthal J, Bahner I, Yu XJ, Lilley J, Crooks GM. Toward gene therapy for Gaucher disease. Human Gene Therapy. 2: 101-5. PMID 1911928 DOI: 10.1089/Hum.1991.2.2-101  0.542
1990 Ozkaynak MF, Lenarsky C, Kohn D, Weinberg K, Parkman R. Mycobacterium avium-intracellulare infections after allogeneic bone marrow transplantation in children. The American Journal of Pediatric Hematology/Oncology. 12: 220-4. PMID 2378416 DOI: 10.1097/00043426-199022000-00019  0.319
1990 Nolta JA, Sender LS, Barranger JA, Kohn DB. Expression of human glucocerebrosidase in murine long-term bone marrow cultures after retroviral vector-mediated transfer Blood. 75: 787-797. PMID 2297579  0.329
1990 Nolta JA, Kohn DB. Comparison of the effects of growth factors on retroviral vector-mediated gene transfer and the proliferative status of human hematopoietic progenitor cells Human Gene Therapy. 1: 257-268. PMID 2081193 DOI: 10.1089/Hum.1990.1.3-257  0.424
1990 Lenarsky C, Kohn D, Parkman R. Bone marrow transplantation for immunodeficiency and genetic diseases. Cancer Treatment and Research. 50: 167-184. PMID 1976349 DOI: 10.1007/978-1-4613-1493-6_10  0.398
1990 Nolta J, Sender LS, Barranger JA, Kohn DB. Expression of human glucocerebrosidase in murine long-term bone marrow cultures after retroviral vector-mediated transfer. Blood. 75: 787-797. DOI: 10.1182/Blood.V75.3.787.787  0.439
1990 Lenarsky C, Kohn DB, Weinberg KI, Parkman R. Bone marrow transplantation for genetic diseases. Hematology-Oncology Clinics of North America. 4: 589-602. DOI: 10.1016/S0889-8588(18)30480-5  0.372
1989 Kohn DB, Kantoff PW. Potential applications of gene therapy. Transfusion. 29: 812-820. PMID 2686109 DOI: 10.1046/J.1537-2995.1989.29990070187.X  0.384
1989 Kohn DB, Anderson WF, Blaese RM. Gene therapy for genetic diseases. Cancer Investigation. 7: 179-92. PMID 2676086 DOI: 10.3109/07357908909038283  0.538
1989 Kohn DB, Mitsuya H, Ballow M, Selegue JE, Barankiewicz J, Cohen A, Gelfand E, Anderson WF, Blaese RM. Establishment and characterization of adenosine deaminase-deficient human T cell lines. Journal of Immunology (Baltimore, Md. : 1950). 142: 3971-7. PMID 2497184  0.603
1988 Eglitis MA, Kohn DB, Moen RC, Blaese RM, Anderson WF. Infection of human hematopoietic progenitor cells using a retroviral vector with a xenotropic pseudotype. Biochemical and Biophysical Research Communications. 151: 201-6. PMID 3348773 DOI: 10.1016/0006-291X(88)90579-7  0.639
1988 Eglitis MA, Kantoff PW, McLachlin JR, Gillio A, Flake AW, Bordignon C, Moen RC, Karson EM, Zwiebel JA, Kohn DB. Gene therapy: efforts at developing large animal models for autologous bone marrow transplant and gene transfer with retroviral vectors. Ciba Foundation Symposium. 130: 229-46. PMID 3327664 DOI: 10.1002/9780470513507.ch14  0.355
1988 Fearon ER, Kohn DB, Winkelstein JA, Vogelstein B, Blaese RM. Carrier detection in the Wiskott Aldrich syndrome. Blood. 72: 1735-9. PMID 3263154  0.518
1988 Eglitis MA, Kantoff PW, Kohn DB, Karson E, Moen RC, Lothrop CD, Blaese RM, Anderson WF. Retroviral-mediated gene transfer into hemopoietic cells. Advances in Experimental Medicine and Biology. 241: 19-27. PMID 3146905 DOI: 10.1007/978-1-4684-5571-7_4  0.65
1988 Fearon E, Kohn D, Winkelstein J, Vogelstein B, Blaese R. Carrier detection in the Wiskott Aldrich syndrome Blood. 72: 1735-1739. DOI: 10.1182/Blood.V72.5.1735.Bloodjournal7251735  0.341
1987 Blaese RM, Kohn DB, Moen RC. The treatment of adenosine deaminase deficiency. Immunology Today. 8: 296-7. PMID 25290834 DOI: 10.1016/0167-5699(87)90015-6  0.49
1987 Kantoff PW, Gillio AP, McLachlin JR, Bordignon C, Eglitis MA, Kernan NA, Moen RC, Kohn DB, Yu SF, Karson E. Expression of human adenosine deaminase in nonhuman primates after retrovirus-mediated gene transfer. Journal of Experimental Medicine. 166: 219-234. PMID 3298525 DOI: 10.1084/Jem.166.1.219  0.482
1987 Gillio A, Bordignon C, Kernan N, Kantoff P, Eglitis M, McLachlin J, Karson E, Yu SF, Zwiebel J, Nienhuis A, Karlsson S, Blaese M, Kohn D, Armentano D, Gilboa E, et al. Retroviral vector-mediated gene transfer and expression in nonhuman primates following autologous bone marrow transplantation. Annals of the New York Academy of Sciences. 511: 406-417. PMID 2894194 DOI: 10.1111/J.1749-6632.1987.Tb36270.X  0.417
1987 Kohn DB, Selegue JE, Ballow M, Blaese RM. Establishment And Characterization Of Adenosine Deaminase (Ada)-Deficient T Cell Lines Pediatric Research. 21. DOI: 10.1203/00006450-198704010-00876  0.607
1986 Kantoff PW, Kohn DB, Mitsuya H, Armentano D, Sieberg M, Zwiebel JA, Eglitis MA, McLachlin JR, Wiginton DA, Hutton JJ. Correction of adenosine deaminase deficiency in cultured human T and B cells by retrovirus-mediated gene transfer. Proceedings of the National Academy of Sciences of the United States of America. 83: 6563-6567. PMID 3489233 DOI: 10.1073/Pnas.83.17.6563  0.485
1986 Anderson WF, Kantoff P, Eglitis M, McLachlin J, Karson E, Zwiebel J, Nienhuis A, Karlsson S, Blaese RM, Kohn D. Gene transfer and expression in nonhuman primates using retroviral vectors Cold Spring Harbor Symposia On Quantitative Biology. 51: 1073-1081. PMID 3034494 DOI: 10.1101/SQB.1986.051.01.125  0.556
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