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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
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240 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
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  0.44
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  0.64
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  0.6
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  0.88
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  0.48
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  0.64
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  0.64
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  0.44
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  0.64
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  0.64
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  0.64
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  0.64
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  0.6
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  0.64
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  0.88
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  0.64
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  0.64
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  0.88
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  0.88
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  0.64
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  0.64
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.32
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  0.48
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  0.88
2016 Pinello L, Canver MC, Hoban MD, Orkin SH, Kohn DB, Bauer DE, Yuan GC. Analyzing CRISPR genome-editing experiments with CRISPResso. Nature Biotechnology. 34: 695-697. PMID 27404874 DOI: 10.1038/nbt.3583  0.36
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  0.88
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  0.88
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  0.88
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  0.88
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  0.64
2015 Komarow HD, Sokolic R, Hershfield MS, Kohn DB, Young M, Metcalfe DD, Candotti F. Impulse oscillometry identifies peripheral airway dysfunction in children with adenosine deaminase deficiency. Orphanet Journal of Rare Diseases. 10: 159. PMID 26682746 DOI: 10.1186/s13023-015-0365-z  0.88
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  0.88
2015 Kohn DB. Gene therapy outpaces haplo for SCID-X1. Blood. 125: 3521-2. PMID 26045591 DOI: 10.1182/blood-2015-04-641720  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.32
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  0.88
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  0.44
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  0.88
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  0.88
2014 Kwan A, Abraham RS, Currier R, Brower A, Andruszewski K, Abbott JK, Baker M, Ballow M, Bartoshesky LE, Bonilla FA, Brokopp C, Brooks E, Caggana M, Celestin J, Church JA, ... ... Kohn DB, et al. Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States. Jama. 312: 729-38. PMID 25138334 DOI: 10.1001/jama.2014.9132  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
2014 Cannon PM, Kohn DB, Kiem HP. HIV eradication--from Berlin to Boston. Nature Biotechnology. 32: 315-6. PMID 24714473 DOI: 10.1038/nbt.2868  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
2014 Kohn DB. Gene Therapy for Primary Immune Deficiencies Stiehm's Immune Deficiencies. 1043-1058. DOI: 10.1016/B978-0-12-405546-9.00059-5  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.48
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
2011 Shaw KL, Kohn DB. A tale of two SCIDs. Science Translational Medicine. 3: 97ps36. PMID 21865536 DOI: 10.1126/scitranslmed.3002594  0.88
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  0.88
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  0.88
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  0.88
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 β-Cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 1389-1396. PMID 28178449 DOI: 10.1038/mt.2010.63  0.48
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
2009 Shah AJ, Kapoor N, Cooper RM, Crooks GM, Lenarsky C, Abdel-Azim H, Wu SQ, Wilson K, Weinberg KI, Parkman R, Kohn DB. Pre- and post-natal treatment of hemophagocytic lymphohistiocytosis. Pediatric Blood & Cancer. 52: 139-42. PMID 18819128 DOI: 10.1002/pbc.21778  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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: 10.1038/mt.2008.93  0.88
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.hg1307s14  0.88
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  0.88
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  0.88
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  0.88
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  0.88
2008 Kohn DB. Gene therapy for childhood immunological diseases. Bone Marrow Transplantation. 41: 199-205. PMID 17994122 DOI: 10.1038/sj.bmt.1705895  0.88
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  0.88
2007 Rossi JJ, June CH, Kohn DB. Genetic therapies against HIV. Nature Biotechnology. 25: 1444-54. PMID 18066041 DOI: 10.1038/nbt1367  0.88
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  0.88
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  0.88
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  0.88
2007 Kohn DB. Lentiviral vectors ready for prime-time. Nature Biotechnology. 25: 65-6. PMID 17211402 DOI: 10.1038/nbt0107-65  0.88
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  0.88
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  0.88
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  0.88
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  0.88
2007 Kohn DB. Gene therapy as salvage Blood. 110: 4.  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
2003 Burgos JS, Rosol M, Moats RA, Khankaldyyan V, Kohn DB, Nelson MD, Laug WE. Time course of bioluminescent signal in orthotopic and heterotopic brain tumors in nude mice. Biotechniques. 34: 1184-8. PMID 12813886 DOI: 10.2144/03346ST01  0.88
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  0.88
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  0.88
2003 Wang L, Haas D, Halene S, Kohn DB. Effects of the negative control region on expression from retroviral LTR. Molecular Therapy : the Journal of the American Society of Gene Therapy. 7: 438-40. PMID 12727105 DOI: 10.1016/S1525-0016(03)00035-2  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
2002 Kohn DB. Adenosine deaminase gene therapy protocol revisited Molecular Therapy. 5: 96-97. PMID 11829515 DOI: 10.1006/mthe.2002.0535  0.88
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  0.88
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  0.88
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  0.88
2001 Skelton D, Satake N, Kohn DB. The enhanced green fluorescent protein (eGFP) is minimally immunogenic in C57BL/6 mice [1] Gene Therapy. 8: 1813-1814. PMID 11803402 DOI: 10.1038/sj.gt.3301586  0.88
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  0.88
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  0.88
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  0.88
2001 Kohn DB. Progress in hematopoietic stem cell gene therapy Cancer Research, Therapy and Control. 11: 41-45.  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
2000 Smogorzewska EM, Brooks J, Annett G, Kapoor N, Crooks GM, Kohn DB, Parkman R, Weinberg KI. T cell depleted haploidentical bone marrow transplantation for the treatment of children with severe combined immunodeficiency Archivum Immunologiae Et Therapiae Experimentalis. 48: 111-118. PMID 10807052  0.88
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  0.88
2000 Kapoor N, Shah AJ, Hoffman J, Crooks GM, Kohn DB, Weinberg KI, Killen R, Wilson K. Cidofovir for treatment of adenoviral infections in pediatric hematopoietic stem cell transplant recipients Blood. 96: 189a.  0.88
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.  0.88
2000 Kohn DB, Weinberg KI, Parkman R. Gene therapy for T-cell immunodeficiencies Immunology and Allergy Clinics of North America. 20: 221-235.  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
1999 Engel BC, Kohn DB. Stem cell directed gene therapy Frontiers in Bioscience : a Journal and Virtual Library. 4: e26-33. PMID 10228093  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
1998 Weinberg KI, Kohn DB. Gene therapy for congenital lymphoid immunodeficiency diseases Seminars in Hematology. 35: 354-366. PMID 9801264  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
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  0.88
1998 Crooks GM, Case SS, Price MA, Naldini L, Kohn DB. Efficient transduction of CD34+CD38- cells with lentiviral vectors Experimental Hematology. 26: 814.  0.88
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  0.88
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  0.88
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  0.88
1997 Kohn DB, Parkman R. Gene therapy for newborns Faseb Journal. 11: 635-639. PMID 9240965  0.88
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  0.88
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  0.88
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  0.88
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  0.88
1997 Kohn DB. Gene therapy for haematopoietic and lymphoid disorders Clinical and Experimental Immunology, Supplement. 107: 54-57. PMID 9020937  0.88
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  0.88
1996 Kohn DB. Gene therapy for hematopoietic and immune disorders Bone Marrow Transplantation. 18: S55-S58. PMID 8971410  0.88
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  0.88
1996 Pao MA, Yu XJ, Kohn DB, Nolta JA. Clonal diversity of vector-marked human henutopoietk progenitors recovered from longterm engrafted immune deficient mice Experimental Hematology. 24: 1057.  0.88
1996 Kohn DB, Weinberg KI, Parkman R. Gene therapy for immune deficiency syndromes - The past, the present, the future Immunologist. 4: 199-202.  0.88
1996 Jadus MR, Kleinman M, Graf M, Pepper K, Kohn DB, Hiserodt J, Wepsic HT. Tumor cells bearing the membrane form of macrophage coloni stimulating factor (m-csf) are killed by macrophages both in vitro and jw vivo Faseb Journal. 10: A1342.  0.88
1996 Kohn DB, Bobbins P, Cooper R, Skelton D, Carbonaro D, Zhu L, Challita P. Qene expression, silencing and methylation in gene therapy Experimental Hematology. 24: 1054.  0.88
1996 Dao MA, Hashino K, Kato L, Kohn DB, Williams PA, Nolta JA. High efficiency transduction of human CD34' progenitors on fibronectin CH-296 verified by clonal integration analysis Experimental Hematology. 24: 1057.  0.88
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.  0.88
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  0.88
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  0.88
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  0.88
1989 Kohn DB, Anderson WF, Blaese RM. Gene therapy for genetic diseases. Cancer Investigation. 7: 179-92. PMID 2676086 DOI: 10.3109/07357908909038283  0.88
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  0.88
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  0.88
1988 Fearon ER, Kohn DB, Winkelstein JA, Vogelstein B, Blaese RM. Carrier detection in the Wiskott Aldrich syndrome. Blood. 72: 1735-9. PMID 3263154  0.88
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  0.88
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  0.88
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