| Year |
Citation |
Score |
| 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 |
0.412 |
|
| 2022 |
Smith RH, Bloomer H, Fink D, Keyvanfar K, Nasimuzzaman M, Sancheznieto F, Dutta R, Guenther Bui K, Alvarado LJ, Bauer TR, Hickstein D, Russell D, Malik P, Van Der Loo J, Highfill SL, et al. Preclinical Evaluation of Foamy Virus Vector-Mediated Gene Addition in Human Hematopoietic Stem/Progenitor Cells for Correction of LAD-1. Human Gene Therapy. PMID 36094106 DOI: 10.1089/hum.2022.065 |
0.354 |
|
| 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 |
0.448 |
|
| 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, ... ... Malik P, 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 |
0.462 |
|
| 2019 |
Hetzel M, Lopez-Rodriguez E, Mucci A, Nguyen AHH, Suzuki T, Shima K, Buchegger T, Dettmer S, Rodt T, Bankstahl JP, Malik P, Knudsen L, Schambach A, Hansen G, Trapnell BC, et al. Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis. Haematologica. PMID 31289207 DOI: 10.3324/Haematol.2018.214866 |
0.373 |
|
| 2019 |
Jayavaradhan R, Pillis DM, Goodman M, Zhang F, Zhang Y, Andreassen PR, Malik P. CRISPR-Cas9 fusion to dominant-negative 53BP1 enhances HDR and inhibits NHEJ specifically at Cas9 target sites. Nature Communications. 10: 2866. PMID 31253785 DOI: 10.1038/S41467-019-10735-7 |
0.322 |
|
| 2019 |
Nasimuzzaman M, Arumugam PI, Mullins ES, James JM, VandenHeuvel K, Narciso MG, Shaw MA, McGraw S, Aronow BJ, Malik P. Elimination of the fibrinogen integrin αβ-binding motif improves renal pathology in mice with sickle cell anemia. Blood Advances. 3: 1519-1532. PMID 31076408 DOI: 10.1182/Bloodadvances.2019032342 |
0.357 |
|
| 2018 |
Jayavaradhan R, Malik P. Genetic Therapies for Sickle Cell Disease. Pediatric Clinics of North America. 65: 465-480. PMID 29803277 DOI: 10.1016/J.Pcl.2018.01.008 |
0.408 |
|
| 2018 |
Jayavaradhan R, Pillis D, Malik P. A Versatile Tool for the Quantification of CRISPR/Cas9-Induced Genome Editing Events in Human Hematopoietic Cell Lines and Hematopoietic Stem/Progenitor Cells. Journal of Molecular Biology. PMID 29751014 DOI: 10.1016/J.Jmb.2018.05.005 |
0.333 |
|
| 2018 |
Nasimuzzaman M, van der Loo JCM, Malik P. Production and Purification of Baculovirus for Gene Therapy Application. Journal of Visualized Experiments : Jove. PMID 29683451 DOI: 10.3791/57019 |
0.324 |
|
| 2018 |
Malik P, Grimley M, Quinn CT, Shova A, Courtney L, Lutzko C, Kalfa TA, Niss O, Mehta PA, Chandra S, Grassman E, Van der Loo JC, Witting S, Nordling D, Shreshta A, et al. Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype Blood. 132: 1021-1021. DOI: 10.1182/Blood-2018-99-119591 |
0.359 |
|
| 2018 |
McGann PT, Niss O, Dong M, Marahatta A, Mizuno T, Kalinyak K, Kalfa TA, Malik P, Quinn CT, Ware RE, Vinks AA. Clinical and Laboratory Benefits of Early Initiation of Hydroxyurea with Pharmacokinetic Guided Dosing for Young Children with Sickle Cell Anemia Blood. 132: 507-507. DOI: 10.1182/Blood-2018-99-112909 |
0.302 |
|
| 2018 |
Nasimuzzaman M, Arumugam PI, Mullins ES, James JM, VandenHeuvel K, Narciso M, Shaw MA, McGraw S, Aronow BJ, Flick MJ, Malik P. Diminished Renal Pathology in a Mouse Model of Sickle Cell Anemia in Which Fibrinogen Binding to Mac-1 Is Inhibited Blood. 132: 267-267. DOI: 10.1182/Blood-2018-99-112641 |
0.354 |
|
| 2017 |
Goodman MA, Arumugam PI, Pillis DM, Loberg A, Nasimuzzaman M, Lynn D, van der Loo JCM, Dexheimer PJ, Keddache M, Bauer TR, Hickstein DD, Russell DW, Malik P. Foamy Virus Vector Carries a Strong Insulator in Its Long Terminal Repeat Which Reduces Its Genotoxic Potential. Journal of Virology. PMID 29046446 DOI: 10.1128/Jvi.01639-17 |
0.417 |
|
| 2017 |
Kalra VK, Zhang S, Malik P, Tahara SM. Placenta growth factor mediated gene regulation in sickle cell disease. Blood Reviews. PMID 28823762 DOI: 10.1016/J.Blre.2017.08.008 |
0.342 |
|
| 2017 |
Strong H, Mitchell MJ, Goldstein-Leever A, Shook L, Malik P, Crosby LE. Erratum to: Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease. Advances in Therapy. PMID 28770518 DOI: 10.1007/S12325-017-0599-3 |
0.365 |
|
| 2017 |
Strong H, Mitchell MJ, Goldstein-Leever A, Shook L, Malik P, Crosby LE. Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease. Advances in Therapy. PMID 28717861 DOI: 10.1007/S12325-017-0587-7 |
0.366 |
|
| 2016 |
Chandrakasan S, Jayavaradhan R, Ernst J, Shrestha A, Loberg A, Dexheimer P, Jordan M, Pang Q, Aronow B, Malik P. KIT Blockade Is Sufficient for Donor Hematopoietic Stem Cell Engraftment in Fanconi Anemia Mice. Blood. PMID 27998889 DOI: 10.1182/Blood-2015-12-689083 |
0.331 |
|
| 2016 |
Nasimuzzaman M, Lynn D, van der Loo JC, Malik P. Purification of baculovirus vectors using heparin affinity chromatography. Molecular Therapy. Methods & Clinical Development. 3: 16071. PMID 27933303 DOI: 10.1038/Mtm.2016.71 |
0.351 |
|
| 2016 |
Rai P, Malik P. Gene therapy for hemoglobin disorders - a mini-review. Journal of Rare Diseases Research & Treatment. 1: 25-31. PMID 27891535 |
0.312 |
|
| 2016 |
Nasimuzzaman M, Lynn D, Ernst R, Beuerlein M, Smith RH, Shrestha A, Cross S, Link K, Lutzko C, Nordling D, Russell DW, Larochelle A, Malik P, Van der Loo JC. Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency. Molecular Therapy. Methods & Clinical Development. 3: 16004. PMID 27722179 DOI: 10.1038/Mtm.2016.4 |
0.374 |
|
| 2016 |
Goodman MA, Malik P. The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges. Therapeutic Advances in Hematology. 7: 302-315. PMID 27695619 DOI: 10.1177/2040620716653729 |
0.381 |
|
| 2016 |
Tiwari S, Hontz A, Terrell C, Arumugam P, Carmo M, Risma K, Jordan M, Malik P. HIGH LEVEL OF PERFORIN EXPRESSION IS REQUIRED FOR EFFECTIVE CORRECTION OF HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS. Human Gene Therapy. PMID 27471778 DOI: 10.1089/Hum.2016.065 |
0.438 |
|
| 2016 |
Salomonis N, Dexheimer PJ, Omberg L, Schroll R, Bush S, Huo J, Schriml L, Ho Sui S, Keddache M, Mayhew C, Shanmukhappa SK, Wells J, Daily K, Hubler S, Wang Y, ... ... Malik P, et al. Integrated Genomic Analysis of Diverse Induced Pluripotent Stem Cell from the Progenitor Cell Biology Consortium. Stem Cell Reports. PMID 27293150 DOI: 10.1016/J.Stemcr.2016.05.006 |
0.371 |
|
| 2016 |
Malik P. Gene Therapy for Hemoglobinopathies: Tremendous Successes and Remaining Caveats. Molecular Therapy : the Journal of the American Society of Gene Therapy. 24: 668-70. PMID 27081721 DOI: 10.1038/Mt.2016.57 |
0.374 |
|
| 2016 |
Christakopoulos GE, Chonat S, Valiente-Alandi I, Konstantinidis DG, Schleper A, Bakeer N, Loberg A, Blaxall BC, Zheng Y, James J, Malik P, Kalfa TA. Reactive Oxygen Species Produced by NADPH Oxidase Contribute to Cardiac Pathology in a Mouse Model of Sickle Cell Disease Blood. 128: 853-853. DOI: 10.1182/Blood.V128.22.853.853 |
0.305 |
|
| 2016 |
Niss O, Taylor MD, Fleck R, Alsaied T, Towbin J, Malik P, Quinn CT. Diffuse Myocardial Fibrosis Is a Common Feature of Sickle Cell Anemia That Is Associated with Diastolic Dysfunction and Restrictive Cardiac Physiology Blood. 128: 8-8. DOI: 10.1182/Blood.V128.22.8.8 |
0.306 |
|
| 2016 |
Quinn CT, Saraf SL, Gordeuk VR, Fitzhugh CD, Creary SE, Bodas P, George A, Raj A, Nero AC, Terrell C, McCord L, Niss O, Dosani R, Taylor MD, Devarajan P, ... Malik P, et al. A Multi-Center, Phase-2 Trial of Losartan for the Nephropathy of Sickle Cell Anemia Blood. 128: 265-265. DOI: 10.1182/Blood.V128.22.265.265 |
0.305 |
|
| 2016 |
Goodman M, Arumugam P, Pillis D, Lynn D, Van der Loo JC, Keddache M, Bauer TR, Hickstein DD, Russell DW, Malik P. Foamy Virus Backbone Has Insulator Properties Which Remarkably Reduce Its Genotoxicity Potential Blood. 128: 1002-1002. DOI: 10.1182/Blood.V128.22.1002.1002 |
0.458 |
|
| 2016 |
Smith RH, Nasimuzzaman M, Dutta R, Uzel G, Bauer TR, Holland SM, Russell DW, Hickstein DD, Malik P, Loo JCMvd, Larochelle A. 285. Foamy Viral Vector Expressing Human CD18 Results in High Levels of Transduction and Multilineage Engraftment with CD18+ LAD-1 Cells in NSG Mice Molecular Therapy. 24: S114-S115. DOI: 10.1016/S1525-0016(16)33094-5 |
0.352 |
|
| 2015 |
Eiymo Mwa Mpollo MS, Brandt EB, Shanmukhappa SK, Arumugam PI, Tiwari S, Loberg A, Pillis D, Rizvi T, Lindsey M, Jonck B, Carmeliet P, Kalra VK, Le Cras TD, Ratner N, Wills-Karp M, ... ... Malik P, et al. Placenta growth factor augments airway hyperresponsiveness via leukotrienes and IL-13. The Journal of Clinical Investigation. PMID 26690703 DOI: 10.1172/Jci77250 |
0.3 |
|
| 2015 |
Arumugam PI, Mullins ES, Shanmukhappa SK, Monia BP, Loberg A, Shaw MA, Rizvi T, Wansapura J, Degen JL, Malik P. Genetic diminution of circulating prothrombin ameliorates multiorgan pathologies in sickle cell disease mice. Blood. 126: 1844-55. PMID 26286849 DOI: 10.1182/Blood-2015-01-625707 |
0.341 |
|
| 2015 |
Gonsalves CS, Li C, Mwa Mpollo MS, Pullarkat V, Malik P, Tahara SM, Kalra VK. Erythropoietin mediated expression of Placenta Growth Factor is regulated via activation of hypoxia inducible factor -1α and post-transcriptionally by mir214 in Sickle Cell Disease. The Biochemical Journal. PMID 25876995 DOI: 10.1042/Bj20141138 |
0.319 |
|
| 2015 |
Carmo M, Risma KA, Arumugam P, Tiwari S, Hontz AE, Montiel-Equihua CA, Alonso-Ferrero ME, Blundell MP, Schambach A, Baum C, Malik P, Thrasher AJ, Jordan MB, Gaspar HB. Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency. Molecular Therapy : the Journal of the American Society of Gene Therapy. 23: 737-45. PMID 25523759 DOI: 10.1038/Mt.2014.242 |
0.424 |
|
| 2015 |
Eiymo Mwa Mpollo M, Brandt E, Shanmukhappa SK, Arumugam P, Tiwari S, Loberg A, Rizvi T, Lindsey M, Jonckx B, Carmeliet P, Kalra V, Lecras T, Ratner N, wills-Karp M, Khurana hershey G, ... Malik P, et al. Placenta Growth Factor Links the IL-13 Response and the Leukotriene Pathway to Augment Airway Hyper-Responsiveness Blood. 126: 977-977. DOI: 10.1182/Blood.V126.23.977.977 |
0.302 |
|
| 2015 |
Doering CB, Denning G, Shields JE, Swaney W, Reeves L, Malik P, Spencer HT. Preclinical Development and Validation of a Hematopoietic Stem Cell-Based Lentivector Treatment for Hemophilia a Blood. 126: 4421-4421. DOI: 10.1182/Blood.V126.23.4421.4421 |
0.436 |
|
| 2015 |
Lachmann N, Happle C, Suzuki T, Hetzel M, Link KA, Mucci A, Arumugam P, Bankstahl J, Dilloo D, Malik P, Lutzko C, Schambach A, Trapnell BC, Hansen G, Moritz T. Innovative Hematopoietic Gene-Therapy Concepts for Hereditary Pulmonary Alveolar Proteinosis Utilizing Hematopoietic Stem Cell Derived Macrophages Blood. 126: 4417-4417. DOI: 10.1182/Blood.V126.23.4417.4417 |
0.389 |
|
| 2015 |
Nasimuzzaman M, Lynn D, Ernst R, Beuerlein M, Cross S, Link K, Lutzko C, Nordling D, Russell D, Malik P, Van der Loo JC. 461. Scale-Up and Manufacturing of High-Titer Foamy Virus Vector Containing Human CD18 for the Treatment of Leukocyte Adhesion Deficiency Molecular Therapy. 23: S183. DOI: 10.1016/S1525-0016(16)34070-9 |
0.41 |
|
| 2015 |
Tiwari S, Hontz A, Terrell C, Arumugam P, Carmo M, Gaspar BH, Risma K, Jordan M, Malik P. 237. Genetic Therapy for Perforin Deficiency Associated Hemophagocytic Lymphohistiocytosis Requires High Level Expression of the Perforin Gene for Adequate Correction Molecular Therapy. 23: S93. DOI: 10.1016/S1525-0016(16)33842-4 |
0.429 |
|
| 2014 |
Kiem HP, Arumugam PI, Burtner CR, Fox CF, Beard BC, Dexheimer P, Adair JE, Malik P. Pigtailed macaques as a model to study long-term safety of lentivirus vector-mediated gene therapy for hemoglobinopathies. Molecular Therapy. Methods & Clinical Development. 1: 14055. PMID 26052523 DOI: 10.1038/Mtm.2014.55 |
0.419 |
|
| 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, ... ... Malik P, 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 |
0.493 |
|
| 2014 |
Suzuki T, Arumugam P, Sakagami T, Lachmann N, Chalk C, Sallese A, Abe S, Trapnell C, Carey B, Moritz T, Malik P, Lutzko C, Wood RE, Trapnell BC. Pulmonary macrophage transplantation therapy. Nature. 514: 450-4. PMID 25274301 DOI: 10.1038/Nature13807 |
0.353 |
|
| 2014 |
Chandrakasan S, Malik P. Gene therapy for hemoglobinopathies: the state of the field and the future. Hematology/Oncology Clinics of North America. 28: 199-216. PMID 24589262 DOI: 10.1016/J.Hoc.2013.12.003 |
0.382 |
|
| 2013 |
Zhou X, Florian MC, Arumugam P, Chen X, Cancelas JA, Lang R, Malik P, Geiger H, Zheng Y. RhoA GTPase controls cytokinesis and programmed necrosis of hematopoietic progenitors. The Journal of Experimental Medicine. 210: 2371-85. PMID 24101377 DOI: 10.1084/Jem.20122348 |
0.343 |
|
| 2013 |
Trobridge GD, Beard BC, Wu RA, Ironside C, Malik P, Kiem H. Correction: Stem Cell Selection In Vivo Using Foamy Vectors Cures Canine Pyruvate Kinase Deficiency Plos One. 8. DOI: 10.1371/Annotation/90F278B2-F474-42Ec-8645-435F7006018C |
0.323 |
|
| 2013 |
Arumugam P, Mullins ES, Shaw MA, Shanmukhappa K, Loberg A, Rizvi T, Wansapura J, Witte D, Malik P, Degen JL. Diminished Multi-Organ Pathologies and Inflammation Associated With Sickle Cell Disease In Mice With Genetically Limited Prothrombin Levels Blood. 122: 729-729. DOI: 10.1182/Blood.V122.21.729.729 |
0.359 |
|
| 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, ... ... Malik P, 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 |
0.567 |
|
| 2013 |
Kiem H, Arumugam P, Christopher B, Adair JE, Beard BC, Fox C, Malik P. Safety Of a Gamma Globin Expressing Lentivirus Vector In a Non-Human Primate Model For Gene Therapy Of Sickle Cell Disease Blood. 122: 2896-2896. DOI: 10.1182/Blood.V122.21.2896.2896 |
0.462 |
|
| 2013 |
Roy S, Konstantinidis DG, Rizvi T, Chang K, Sundaram N, Wourms M, Anastacia L, Song L, Burke K, Inagami T, Ratner N, Witte D, Kalfa TA, Cancelas JA, Shanmukhappa SK, ... Malik P, et al. Increased Oxidative Stress In Sickle Cell Disease Activates The Renin-Angiotensin-TGF-β Pathway To Mediate Sickle Nephropathy Blood. 122: 2211-2211. DOI: 10.1182/Blood.V122.21.2211.2211 |
0.307 |
|
| 2013 |
Yang M, Loberg A, Rizvi T, Wourms M, Song L, Burke K, Ratner N, Carmeliet P, LeCras T, Wills-Karp M, Malik P. Airway Hyper-Responsiveness Associated With Sickle Cell Disease Is Mediated By Placenta Growth Factor Blood. 122: 185-185. DOI: 10.1182/Blood.V122.21.185.185 |
0.326 |
|
| 2012 |
Chen X, Skutt-Kakaria K, Davison J, Ou YL, Choi E, Malik P, Loeb K, Wood B, Georges G, Torok-Storb B, Paddison PJ. G9a/GLP-dependent histone H3K9me2 patterning during human hematopoietic stem cell lineage commitment. Genes & Development. 26: 2499-511. PMID 23105005 DOI: 10.1101/Gad.200329.112 |
0.34 |
|
| 2012 |
Trobridge GD, Beard BC, Wu RA, Ironside C, Malik P, Kiem HP. Stem cell selection in vivo using foamy vectors cures canine pyruvate kinase deficiency. Plos One. 7: e45173. PMID 23028826 DOI: 10.1371/Journal.Pone.0045173 |
0.404 |
|
| 2012 |
Ballas SK, Kesen MR, Goldberg MF, Lutty GA, Dampier C, Osunkwo I, Wang WC, Hoppe C, Hagar W, Darbari DS, Malik P. Beyond the definitions of the phenotypic complications of sickle cell disease: an update on management. Thescientificworldjournal. 2012: 949535. PMID 22924029 DOI: 10.1100/2012/949535 |
0.323 |
|
| 2012 |
van der Loo JC, Swaney WP, Grassman E, Terwilliger A, Higashimoto T, Schambach A, Hacein-Bey-Abina S, Nordling DL, Cavazzana-Calvo M, Thrasher AJ, Williams DA, Reeves L, Malik P. Critical variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency. Gene Therapy. 19: 872-6. PMID 22551777 DOI: 10.1038/Gt.2012.37 |
0.404 |
|
| 2012 |
van der Loo JC, Swaney WP, Grassman E, Terwilliger A, Higashimoto T, Schambach A, Baum C, Thrasher AJ, Williams DA, Nordling DL, Reeves L, Malik P. Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection. Gene Therapy. 19: 246-54. PMID 21753795 DOI: 10.1038/Gt.2011.102 |
0.358 |
|
| 2012 |
Jordan M, Carmo M, Tiwari S, Arumugam P, Risma K, Gaspar B, Malik P. Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH): Fixing a Criticial ‘Circuit Breaker’ in the Immune System. Blood. 120: 3158-3158. DOI: 10.1182/Blood.V120.21.3158.3158 |
0.432 |
|
| 2012 |
Carmo M, Arumugam P, Tiwari S, Thrasher A, Risma K, Malik P, Gaspar B, Jordan M. Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH): Fixing a Critical ‘Circuit Breaker’ in the Immune System Biology of Blood and Marrow Transplantation. 18: S201. DOI: 10.1016/J.Bbmt.2011.12.524 |
0.314 |
|
| 2011 |
Modlich U, Sürth J, Zychlinski D, Meyer J, Brendel C, Grez M, Arumugam P, Malik P, Grassman E, Schambach A, Baum C. Use of the in Vitro Immortalization Assay to Quantify the Impact of Integration Spectrum and Vector Design on Insertional Mutagenesis Blood. 118: 3123-3123. DOI: 10.1182/Blood.V118.21.3123.3123 |
0.43 |
|
| 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, ... ... Malik P, 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 |
0.595 |
|
| 2011 |
Mpollo MEM, Malik P. Sickle Cell Disease Is Associated with Reduced Adenosine Deaminase Catalytic Activity, Resulting in Altered Adenosine Metabolism Blood. 118: 1078-1078. DOI: 10.1182/Blood.V118.21.1078.1078 |
0.347 |
|
| 2010 |
Arumugam P, Malik P. Genetic therapy for beta-thalassemia: from the bench to the bedside. Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program. 2010: 445-50. PMID 21239833 DOI: 10.1182/Asheducation-2010.1.445 |
0.445 |
|
| 2010 |
Perumbeti A, Malik P. Therapy for beta-globinopathies: a brief review and determinants for successful and safe correction. Annals of the New York Academy of Sciences. 1202: 36-44. PMID 20712770 DOI: 10.1111/J.1749-6632.2010.05584.X |
0.361 |
|
| 2010 |
Perumbeti A, Malik P. Genetic correction of sickle cell anemia and beta-thalassemia: progress and new perspective. Thescientificworldjournal. 10: 644-54. PMID 20419277 DOI: 10.1100/Tsw.2010.67 |
0.368 |
|
| 2010 |
Patel N, Sundaram N, Yang M, Madigan C, Kalra VK, Malik P. Placenta growth factor (PlGF), a novel inducer of plasminogen activator inhibitor-1 (PAI-1) in sickle cell disease (SCD). The Journal of Biological Chemistry. 285: 16713-22. PMID 20351105 DOI: 10.1074/Jbc.M110.101691 |
0.339 |
|
| 2010 |
Sundaram N, Tailor A, Mendelsohn L, Wansapura J, Wang X, Higashimoto T, Pauciulo MW, Gottliebson W, Kalra VK, Nichols WC, Kato GJ, Malik P. High levels of placenta growth factor in sickle cell disease promote pulmonary hypertension. Blood. 116: 109-12. PMID 20335221 DOI: 10.1182/Blood-2009-09-244830 |
0.311 |
|
| 2009 |
Arumugam PI, Higashimoto T, Urbinati F, Modlich U, Nestheide S, Xia P, Fox C, Corsinotti A, Baum C, Malik P. Genotoxic Potential of Lineage-specific Lentivirus Vectors Carrying the β-Globin Locus Control Region. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1929-1937. PMID 28178459 DOI: 10.1038/mt.2009.183 |
0.699 |
|
| 2009 |
Wang D, Zhang W, Kalfa TA, Grabowski G, Davies S, Malik P, Pan D. Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome. Proceedings of the National Academy of Sciences of the United States of America. 106: 19958-63. PMID 19903883 DOI: 10.1073/Pnas.0908528106 |
0.396 |
|
| 2009 |
Arumugam PI, Urbinati F, Velu CS, Higashimoto T, Grimes HL, Malik P. The 3' region of the chicken hypersensitive site-4 insulator has properties similar to its core and is required for full insulator activity. Plos One. 4: e6995. PMID 19746166 DOI: 10.1371/Journal.Pone.0006995 |
0.701 |
|
| 2009 |
Arumugam PI, Higashimoto T, Urbinati F, Modlich U, Nestheide S, Xia P, Fox C, Corsinotti A, Baum C, Malik P. Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1929-37. PMID 19707188 DOI: 10.1038/Mt.2009.183 |
0.719 |
|
| 2009 |
Perumbeti A, Higashimoto T, Urbinati F, Franco R, Meiselman HJ, Witte D, Malik P. A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction. Blood. 114: 1174-85. PMID 19474450 DOI: 10.1182/Blood-2009-01-201863 |
0.71 |
|
| 2009 |
Urbinati F, Arumugam P, Higashimoto T, Perumbeti A, Mitts K, Xia P, Malik P. Mechanism of reduction in titers from lentivirus vectors carrying large inserts in the 3'LTR. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1527-36. PMID 19384292 DOI: 10.1038/Mt.2009.89 |
0.71 |
|
| 2009 |
Patel N, Gonsalves CS, Yang M, Malik P, Kalra VK. Placenta growth factor induces 5-lipoxygenase-activating protein to increase leukotriene formation in sickle cell disease. Blood. 113: 1129-38. PMID 18945963 DOI: 10.1182/Blood-2008-07-169821 |
0.316 |
|
| 2009 |
Arumugam P, Urbinati F, Velu CS, Higashimoto T, Grimes HL, Malik P. A Novel Combination of Chicken Hypersensitive Site-4 Insulator Elements Improves Titers and Restores Full Insulator Activity. Blood. 114: 3566-3566. DOI: 10.1182/Blood.V114.22.3566.3566 |
0.7 |
|
| 2008 |
Williams JP, Wu J, Johansson G, Rizvi TA, Miller SC, Geiger H, Malik P, Li W, Mukouyama YS, Cancelas JA, Ratner N. Nf1 mutation expands an EGFR-dependent peripheral nerve progenitor that confers neurofibroma tumorigenic potential. Cell Stem Cell. 3: 658-69. PMID 19041782 DOI: 10.1016/J.Stem.2008.10.003 |
0.306 |
|
| 2008 |
Trobridge GD, Beard BC, Gooch C, Wohlfahrt M, Olsen P, Fletcher J, Malik P, Kiem HP. Efficient transduction of pigtailed macaque hematopoietic repopulating cells with HIV-based lentiviral vectors. Blood. 111: 5537-43. PMID 18388180 DOI: 10.1182/Blood-2007-09-115022 |
0.419 |
|
| 2008 |
Arumugam P, Urbinati F, Velu CS, Grimes HL, Malik P. The 3′ End of the Chicken Hypersensitive Site-4 Insulator Has Properties Similar to the 5′ Insulator Core and Is Necessary in Conjunction with the Core for Full Insulator Activity Blood. 112: 817-817. DOI: 10.1182/Blood.V112.11.817.817 |
0.66 |
|
| 2008 |
Grassman E, Higashimoto T, Rohrbach J, Hall D, Williams DA, Reeves L, Malik P. Pulmozyme® Treatment of Vectors Produced by Transient Transfection Reduces Residual Plasmid DNA on Human CD34+ Hematopoietic Progenitor Cells without Loss of Transduction or Engraftment Efficiency Blood. 112: 4626-4626. DOI: 10.1182/Blood.V112.11.4626.4626 |
0.415 |
|
| 2008 |
Van der Loo JC, Swaney W, Nordling D, Schambach A, Baum C, Williams DA, Reeves L, Malik P. Production of High Titer cGMP-Grade SIN Gamma-Retroviral Vectors by Transfection in a Closed System Bioreactor Blood. 112: 3539-3539. DOI: 10.1182/Blood.V112.11.3539.3539 |
0.383 |
|
| 2008 |
Urbinati F, Arumugam P, Higashimoto T, Perumbeti A, Xia P, Malik P. Mechanism of Reduction in Titers from Lentivirus Vectors Carrying Chromatin Insulator Elements in the 3′ LTR Blood. 112: 2359-2359. DOI: 10.1182/Blood.V112.11.2359.2359 |
0.718 |
|
| 2008 |
Wang D, Zhang W, Kalfa TA, Malik P, Pan D. High Level Production of a Lysosomal Enzyme from HSC-Derived Erythroid Cells for Therapeutic Correction of Hurler Syndrome in Mice. Blood. 112: 2355-2355. DOI: 10.1182/Blood.V112.11.2355.2355 |
0.441 |
|
| 2008 |
Perumbeti A, Higashimoto T, Urbinati F, Lauderback K, Loberg A, Estevez-Pagani G, Meiselman H, Franco R, Witte D, Joiner CH, Malik P. Correction of Sickle Cell Anemia with γ-Globin Gene Delivered by Lentivirus Vector in the Setting of Myeloablative or Reduced Intensity Conditioning, and Establishing Critical Determinants for Successful Gene Therapy for Sickle Cell Disease. Blood. 112: 2354-2354. DOI: 10.1182/Blood.V112.11.2354.2354 |
0.702 |
|
| 2008 |
Kalra VK, Patel N, Gonsalves C, Yang M, Malik P. Placenta Growth Factor Induces 5-Lipoxygenase-Activating Protein Via Hypoxia-Inducible Factor-1α and Contributes to Increased Leukotrienes in Sickle Cell Disease. Blood. 112: 1447-1447. DOI: 10.1182/Blood.V112.11.1447.1447 |
0.333 |
|
| 2008 |
Arumugam P, Urbinati F, Higashimoto T, Perumbeti A, Malik P. Lentivirus vectors for gene therapy for hemoglobinopathies Blood Cells, Molecules, and Diseases. 40: 275-276. DOI: 10.1016/J.Bcmd.2007.10.059 |
0.681 |
|
| 2008 |
Arumugam P, Xia P, Urbinati F, Malik P. Elements of cHS4 insulator necessary for maximum chromatin barrier activity Blood Cells, Molecules, and Diseases. 40: 252. DOI: 10.1016/J.Bcmd.2007.10.015 |
0.637 |
|
| 2007 |
Arumugam PI, Scholes J, Perelman N, Xia P, Yee JK, Malik P. Improved Human β-globin Expression from Self-inactivating Lentiviral Vectors Carrying the Chicken Hypersensitive Site-4 (cHS4) Insulator Element. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 1863-1871. PMID 28182916 DOI: 10.1038/sj.mt.6300259 |
0.353 |
|
| 2007 |
Arumugam PI, Scholes J, Perelman N, Xia P, Yee JK, Malik P. Improved human beta-globin expression from self-inactivating lentiviral vectors carrying the chicken hypersensitive site-4 (cHS4) insulator element. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 1863-71. PMID 17622240 DOI: 10.1038/sj.mt.6300259 |
0.361 |
|
| 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 |
0.71 |
|
| 2007 |
Robert-Richard E, Richard E, Malik P, Ged C, de Verneuil H, Moreau-Gaudry F. Murine retroviral but not human cellular promoters induce in vivo erythroid-specific deregulation that can be partially prevented by insulators. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 173-82. PMID 17164789 DOI: 10.1038/sj.mt.6300030 |
0.328 |
|
| 2007 |
Urbinati F, Arumugam P, Higashimoto T, Xia P, Malik P. Mechanism of Reduction in Titers with Vectors Flanked by Chromatin Insulator Elements Blood. 110: 3734-3734. DOI: 10.1182/blood.v110.11.3734.3734 |
0.314 |
|
| 2007 |
Arumugam PI, Xia P, Urbinati F, Malik P. Elements of a Chromatin Insulator Necessary for Consistent, High-Level Globin Gene Expression Blood. 110: 3731-3731. DOI: 10.1182/Blood.V110.11.3731.3731 |
0.669 |
|
| 2006 |
Urbinati F, Madigan C, Malik P. Pathophysiology and therapy for haemoglobinopathies. Part II: thalassaemias. Expert Reviews in Molecular Medicine. 8: 1-26. PMID 16684395 DOI: 10.1017/S1462399406010805 |
0.657 |
|
| 2006 |
Urbinati F, Xia P, Perumbeti A, Xia M, Corsinotti A, Zarzuela A, Perelman N, Malik P. 467. Lentiviral Vector (LV) Non-Coding Elements That Impart High Titers and Expression to β-Globin (hβ) Cassettes Molecular Therapy. 13: S181. DOI: 10.1016/J.Ymthe.2006.08.536 |
0.675 |
|
| 2005 |
Malik P, Arumugam PI. Gene Therapy for beta-thalassemia. Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program. 45-50. PMID 16304358 DOI: 10.1182/asheducation-2005.1.45 |
0.315 |
|
| 2004 |
Puthenveetil G, Scholes J, Carbonell D, Qureshi N, Xia P, Zeng L, Li S, Yu Y, Hiti AL, Yee JK, Malik P. Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector. Blood. 104: 3445-53. PMID 15292064 DOI: 10.1182/Blood-2004-04-1427 |
0.352 |
|
| 2004 |
Puthenveetil G, Malik P. Gene therapy for hemoglobinopathies: are we there yet? Current Hematology Reports. 3: 298-305. PMID 15217560 |
0.305 |
|
| 2004 |
Richard E, Robert E, Cario-Andre M, Ged C, Malik P, Verneuil Hd, Moreau-Gaudry F. 701. A SIN lentiviral vector that allows in vivo drug-selection of hematopoietic stem cells yet retains erythroid-specific expression of the ferrochelatase gene and results in cure of murine protoporphyria Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.609 |
0.476 |
|
| 2001 |
Moreau-Gaudry F, Xia P, Jiang G, Perelman NP, Bauer G, Ellis J, Surinya KH, Mavilio F, Shen CK, Malik P. High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors Blood. 98: 2664-2672. PMID 11675336 DOI: 10.1182/blood.V98.9.2664 |
0.375 |
|
| 2001 |
Richard E, Mendez M, Mazurier F, Morel C, Costet P, Xia P, Fontanellas A, Geronimi F, Cario-André M, Taine L, Ged C, Malik P, De Verneuil H, Moreau-Gaudry F. Gene therapy of a mouse model of protoporphyria with a self-inactivating erythroid-specific lentiviral vector without preselection Molecular Therapy. 4: 331-338. PMID 11592836 DOI: 10.1006/mthe.2001.0467 |
0.329 |
|
| 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 |
0.462 |
|
| 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 |
0.442 |
|
| 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 |
0.504 |
|
| 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 |
0.524 |
|
| 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. |
0.466 |
|
| 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 |
0.514 |
|
| 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 |
0.546 |
|
| 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 |
0.558 |
|
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