Donald Kohn

Donald Barry Kohn

Professor, MIMG, University of California Los Angeles

Professor, Molecular and Medical Pharmacology, University of California Los Angeles

Professor, Pediatrics, University of California Los Angeles

Publications

  1. Thakar MS, Logan BR, Puck JM, Dunn EA, Buckley RH, Cowan MJ, O'Reilly RJ, Kapoor N, Satter LF, Pai SY, Heimall J, Chandra S, Ebens CL, Chellapandian D, Williams O, Burroughs LM, Saldana BD, Rayes A, Madden LM, Chandrakasan S, Bednarski JJ, DeSantes KB, Cuvelier GDE, Teira P, Gillio AP, Eissa H, Knutsen AP, Goldman FD, Aquino VM, Shereck EB, Moore TB, Caywood EH, Lugt MTV, Rozmus J, Broglie L, Yu LC, Shah AJ, Andolina JR, Liu X, Parrott RE, Dara J, Prockop S, Martinez CA, Kapadia M, Jyonouchi SC, Sullivan KE, Bleesing JJ, Chaudhury S, Petrovic A, Keller MD, Quigg TC, Parikh S, Shenoy S, Seroogy C, Rubin T, Decaluwe H, Routes JM, Torgerson TR, Leiding JW, Pulsipher MA, Kohn DB, Griffith LM, Haddad E, Dvorak CC, Notarangelo LD. Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium.. Lancet (London, England), 2023.
  2. McAuley GE, Yiu G, Chang PC, Newby GA, Campo-Fernandez B, Fitz-Gibbon ST, Wu X, Kang SL, Garibay A, Butler J, Christian V, Wong RL, Everette KA, Azzun A, Gelfer H, Seet CS, Narendran A, Murguia-Favela L, Romero Z, Wright N, Liu DR, Crooks GM, Kohn DB. Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing.. Cell, 2023.
  3. Segura EER, Ayoub PG, Hart KL, Kohn DB. Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials.. Viruses, 2023.
  4. Ayoub PG, Kohn DB. β-Thalassemia: all about that base, no cutting.. Blood, 2023.
  5. Wong RL, Sackey S, Brown D, Senadheera S, Masiuk K, Quintos JP, Colindres N, Riggan L, Morgan RA, Malech HL, Hollis RP, Kohn DB. Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression.. Blood, 2023.
  6. 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, 2023.
  7. Grunebaum E, Booth C, Cuvelier GDE, Loves R, Aiuti A, Kohn DB. Updated Management Guidelines for Adenosine Deaminase Deficiency.. The journal of allergy and clinical immunology. In practice, 2023.
  8. Eapen M, Brazauskas R, Williams DA, Walters MC, St Martin A, Jacobs BL, Antin JH, Bona K, Chaudhury S, Coleman-Cowger VH, DiFronzo NL, Esrick EB, Field JJ, Fitzhugh CD, Kanter J, Kapoor N, Kohn DB, Krishnamurti L, London WB, Pulsipher MA, Talib S, Thompson AA, Waller EK, Wun T, Horowitz MM. Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease.. Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2023.
  9. Cornetta K, Yao J, House K, Duffy L, Adusumilli PS, Beyer R, Booth C, Brenner M, Curran K, Grilley B, Heslop H, Hinrichs CS, Kaplan RN, Kiem HP, Kochenderfer J, Kohn DB, Mailankody S, Norberg SM, O'Cearbhaill RE, Pappas J, Park J, Ramos C, Ribas A, Rivière I, Rosenberg SA, Sauter C, Shah NN, Slovin SF, Thrasher A, Williams DA, Lin TY. Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials.. Molecular therapy : the journal of the American Society of Gene Therapy, 2022.
  10. Cornetta K, Lin TY, Pellin D, Kohn DB. Meeting FDA Guidance recommendations for replication-competent virus and insertional oncogenesis testing.. Molecular therapy. Methods & clinical development, 2022.
  11. Dvorak CC, Haddad E, Heimall J, Dunn E, Buckley RH, Kohn DB, Cowan MJ, Pai SY, Griffith LM, Cuvelier GDE, Eissa H, Shah AJ, O'Reilly RJ, Pulsipher MA, Wright NAM, Abraham RS, Satter LF, Notarangelo LD, Puck JM. The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions.. The Journal of allergy and clinical immunology, 2022.
  12. Dvorak CC, Haddad E, Heimall J, Dunn E, Cowan MJ, Pai SY, Kapoor N, Satter LF, Buckley RH, O'Reilly RJ, Chandra S, Bednarski JJ, Williams O, Rayes A, Moore TB, Ebens CL, Davila Saldana BJ, Petrovic A, Chellapandian D, Cuvelier GDE, Vander Lugt MT, Caywood EH, Chandrakasan S, Eissa H, Goldman FD, Shereck E, Aquino VM, Desantes KB, Madden LM, Miller HK, Yu L, Broglie L, Gillio A, Shah AJ, Knutsen AP, Andolina JP, Joshi AY, Szabolcs P, Kapadia M, Martinez CA, Parrot RE, Sullivan KE, Prockop SE, Abraham RS, Thakar MS, Leiding JW, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Puck JM. The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions.. The Journal of allergy and clinical immunology, 2022.
  13. White SL, Lee TD, Toy T, Carroll JE, Polsky L, Campo Fernandez B, Davila A, Kohn DB, Chang VY. Evaluation of clonal hematopoiesis in pediatric ADA-SCID gene therapy participants.. Blood advances, 2022.
  14. Collon K, Bell JA, Gallo MC, Chang SW, Bougioukli S, Sugiyama O, Tassey J, Hollis R, Heckmann N, Oakes DA, Longjohn DB, Evseenko D, Kohn DB, Lieberman JR. Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential.. Gene therapy, 2022.
  15. White SL, Hart K, Kohn DB. Diverse Approaches to Gene Therapy of Sickle Cell Disease.. Annual review of medicine, 2022.
  16. Cuvelier GDE, Logan BR, Prockop SE, Buckley RH, Kuo CY, Griffith LM, Liu X, Yip A, Hershfield MS, Ayoub PG, Moore TB, Dorsey MJ, O'Reilly RJ, Kapoor N, Pai SY, Kapadia M, Ebens CL, Forbes Satter LR, Burroughs LM, Petrovic A, Chellapandian D, Heimall J, Shyr DC, Rayes A, Bednarski JJ, Chandra S, Chandrakasan S, Gillio AP, Madden L, Quigg TC, Caywood EH, Dávila Saldaña BJ, DeSantes K, Eissa H, Goldman FD, Rozmus J, Shah AJ, Vander Lugt MT, Thakar MS, Parrott RE, Martinez C, Leiding JW, Torgerson TR, Pulsipher MA, Notarangelo LD, Cowan MJ, Dvorak CC, Haddad E, Puck JM, Kohn DB. Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.. Blood, 2022.
  17. Labrosse R, Boufaied I, Bourdin B, Gona S, Randolph HE, Logan BR, Bourbonnais S, Berthe C, Chan W, Buckley RH, Parrott RE, Cuvelier GDE, Kapoor N, Chandra S, Dávila Saldaña BJ, Eissa H, Goldman FD, Heimall J, O'Reilly R, Chaudhury S, Kolb EA, Shenoy S, Griffith LM, Pulsipher M, Kohn DB, Notarangelo LD, Pai SY, Cowan MJ, Dvorak CC, Haddad É, Puck JM, Barreiro LB, Decaluwe H. Aberrant T-cell exhaustion in severe combined immunodeficiency survivors with poor T-cell reconstitution after transplantation.. The Journal of allergy and clinical immunology, 2022.
  18. Sosa RA, Mone T, Naini BV, Kohn DB, Reed EF, Wheeler K, Campo-Fernandez B, Davila A, Chaffin DJ, DiNorcia J, Kaldas FM, Cohen A, Lum EL, Veale JL, Kogut NM. Apheresis of Deceased Donors as a New Source of Mobilized Peripheral Blood Hematopoietic Stem Cells for Transplant Tolerance.. Transplantation, 2022.
  19. Collon K, Gallo M, Bell J, Chang S, Rodman J, Sugiyama O, Kohn DB, Lieberman JR. Improving Lentiviral Transduction of Human Adipose-Derived Mesenchymal Stem Cells.. Human gene therapy, 2022.
  20. Campagna GA, Chew L, Pettenkofer M, Nicoletti E, Schwartz JD, Choi G, Fernandes AO, Terrazas D, Kohn DB, Tsui I. Findings on Optical Coherence Tomography in Malignant Infantile Osteopetrosis.. Ophthalmic surgery, lasers & imaging retina, 2022.
  21. Magis W, DeWitt MA, Wyman SK, Vu JT, Heo SJ, Shao SJ, Hennig F, Romero ZG, Campo-Fernandez B, Said S, McNeill MS, Rettig GR, Sun Y, Wang Y, Behlke MA, Kohn DB, Boffelli D, Walters MC, Corn JE, Martin DIK. High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation.. iScience, 2022.
  22. Arnold DE, Chellapandian D, Parikh S, Mallhi K, Marsh RA, Heimall JR, Grossman D, Chitty-Lopez M, Murguia-Favela L, Gennery AR, Boulad F, Arbuckle E, Cowan MJ, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson T, Kang EM, Malech HL, Leiding JW. Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy.. Journal of clinical immunology, 2022.
  23. Ibarrondo FJ, Hofmann C, Ali A, Ayoub P, Kohn DB, Yang OO. Previous Infection Combined with Vaccination Produces Neutralizing Antibodies with Potency against SARS-CoV-2 Variants.. mBio, 2021.
  24. Han J, Tam K, Tam C, Hollis RP, Kohn DB. Improved lentiviral vector titers from a multi-gene knockout packaging line.. Molecular therapy oncolytics, 2021.
  25. Li YR, Zhou Y, Kim YJ, Zhu Y, Ma F, Yu J, Wang YC, Chen X, Li Z, Zeng S, Wang X, Lee D, Ku J, Tsao T, Hardoy C, Huang J, Cheng D, Montel-Hagen A, Seet CS, Crooks GM, Larson SM, Sasine JP, Wang X, Pellegrini M, Ribas A, Kohn DB, Witte O, Wang P, Yang L. Development of allogeneic HSC-engineered iNKT cells for off-the-shelf cancer immunotherapy.. Cell reports. Medicine, 2021.
  26. Reinhardt B, Habib O, Shaw KL, Garabedian E, Carbonaro-Sarracino DA, Terrazas D, Fernandez BC, De Oliveira S, Moore TB, Ikeda AK, Engel BC, Podsakoff GM, Hollis RP, Fernandes A, Jackson C, Shupien S, Mishra S, Davila A, Mottahedeh J, Vitomirov A, Meng W, Rosenfeld AM, Roche AM, Hokama P, Reddy S, Everett J, Wang X, Luning Prak ET, Cornetta K, Hershfield MS, Sokolic R, De Ravin SS, Malech HL, Bushman FD, Candotti F, Kohn DB. Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.. Blood, 2021.
  27. Ihn H, Kang H, Iglesias B, Sugiyama O, Tang A, Hollis R, Bougioukli S, Skorka T, Park S, Longjohn D, Oakes DA, Kohn DB, Lieberman JR. Regional Gene Therapy with Transduced Human Cells: The Influence of "Cell Dose" on Bone Repair.. Tissue engineering. Part A, 2021.
  28. Abou-El-Enein M, Angelis A, Appelbaum FR, Andrews NC, Bates SE, Bierman AS, Brenner MK, Cavazzana M, Caligiuri MA, Clevers H, Cooke E, Daley GQ, Dzau VJ, Ellis LM, Fineberg HV, Goldstein LSB, Gottschalk S, Hamburg MA, Ingber DE, Kohn DB, Krainer AR, Maus MV, Marks P, Mummery CL, Pettigrew RI, Rutter JL, Teichmann SA, Terzic A, Urnov FD, Williams DA, Wolchok JD, Lawler M, Turtle CJ, Bauer G, Ioannidis JPA. Evidence generation and reproducibility in cell and gene therapy research: A call to action.. Molecular therapy. Methods & clinical development, 2021.
  29. Baloh CH, Borkar SA, Chang KF, Yao J, Hershfield MS, Parikh SH, Kohn DB, Goodenow MM, Sleasman JW, Yin L. Normal IgH Repertoire Diversity in an Infant with ADA Deficiency After Gene Therapy.. Journal of clinical immunology, 2021.
  30. Kang HP, Ihn H, Robertson DM, Chen X, Sugiyama O, Tang A, Hollis R, Skorka T, Longjohn D, Oakes D, Shah R, Kohn D, Jakus AE, Lieberman JR. Regional gene therapy for bone healing using a 3D printed scaffold in a rat femoral defect model.. Journal of biomedical materials research. Part A, 2021.
  31. Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.. The New England journal of medicine, 2021.
  32. Gray DH, Villegas I, Long J, Santos J, Keir A, Abele A, Kuo CY, Kohn DB. Optimizing Integration and Expression of Transgenic Bruton's Tyrosine Kinase for CRISPR-Cas9-Mediated Gene Editing of X-Linked Agammaglobulinemia.. The CRISPR journal, 2021.
  33. Garcia G, Sharma A, Ramaiah A, Sen C, Purkayastha A, Kohn DB, Parcells MS, Beck S, Kim H, Bakowski MA, Kirkpatrick MG, Riva L, Wolff KC, Han B, Yuen C, Ulmert D, Purbey PK, Scumpia P, Beutler N, Rogers TF, Chatterjee AK, Gabriel G, Bartenschlager R, Gomperts B, Svendsen CN, Betz UAK, Damoiseaux RD, Arumugaswami V. Antiviral drug screen identifies DNA-damage response inhibitor as potent blocker of SARS-CoV-2 replication.. Cell reports, 2021.
  34. Kohn LA, Kohn DB. Gene Therapies for Primary Immune Deficiencies.. Frontiers in immunology, 2021.
  35. Carbonaro-Sarracino DA, Chun K, Clark DN, Kaufman ML, Jin X, Wang X, Kohn DB. Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID.. Molecular therapy. Methods & clinical development, 2021.
  36. Dorsey M, Wright NAM, Chaimowitz NS, Dávila Saldaña BJ, Miller H, Keller MD, Thakar MS, Shah AJ, Abu-Arja R, Andolina J, Aquino V, Barnum JL, Bednarski JJ, Bhatia M, Bonilla FA, Butte MJ, Bunin NJ, Burroughs LM, Chandra S, Chaudhury S, Chen K, Chong H, Cuvelier G, Dalal J, DeFelice ML, DeSantes KB, Forbes LR, Gillio A, Goldman F, Joshi AY, Kapoor N, Knutsen AP, Kobrynski L, Lieberman JA, Leiding JW, Oshrine B, Patel KP, Prockop S, Quigg TC, Quinones R, Schultz KR, Seroogy C, Shyr D, Siegel S, Smith AR, Torgerson TR, Vander Lugt MT, Yu LC, Cowan MJ, Buckley RH, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Logan B, Notarangelo LD, Pai SY, Puck J, Pulsipher MA, Heimall J. Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers.. Journal of clinical immunology, 2021.
  37. 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.. Human gene therapy, 2021.
  38. Benitez EK, Lomova Kaufman A, Cervantes L, Clark DN, Ayoub PG, Senadheera S, Osborne K, Sanchez JM, Crisostomo RV, Wang X, Reuven N, Shaul Y, Hollis RP, Romero Z, Kohn DB. Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells.. Frontiers in genome editing, 2020.
  39. 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, 2020.
  40. Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GDE, Thakar MS, Chen K, Teira P, Shenoy S, Phelan R, Forbes LR, Martinez C, Chellapandian D, Dávila Saldaña BJ, Shah AJ, Weinacht KG, Joshi A, Boulad F, Quigg TC, Dvorak CC, Grossman D, Torgerson T, Graham P, Prasad V, Knutsen A, Chong H, Miller H, de la Morena MT, DeSantes K, Cowan MJ, Notarangelo LD, Kohn DB, Stenger E, Pai SY, Routes JM, Puck JM, Kapoor N, Pulsipher MA, Malech HL, Parikh S, Kang EM, submitted on behalf of the Primary Immune Deficiency Treatment Consortium. Correction: Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT.. Journal of clinical immunology, 2020.
  41. 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, Ke Z, Zou C, Lee CF, Jonas SJ, Ban Q, Weiss PS, Kohn DB, Chen K, Chiou SH, Tseng HR. Supramolecular nanosubstrate-mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies.. Science advances, 2020.
  42. Dorsey MJ, Wright NAM, Chaimowitz NS, Dávila Saldaña BJ, Miller H, Keller MD, Thakar MS, Shah AJ, Abu-Arja R, Andolina J, Aquino V, Barnum JL, Bednarski JJ, Bhatia M, Bonilla FA, Butte MJ, Bunin NJ, Chandra S, Chaudhury S, Chen K, Chong H, Cuvelier GDE, Dalal J, DeFelice ML, DeSantes KB, Forbes LR, Gillio A, Goldman F, Joshi AY, Kapoor N, Knutsen AP, Kobrynski L, Lieberman JA, Leiding JW, Oshrine B, Patel KP, Prockop S, Quigg TC, Quinones R, Schultz KR, Seroogy C, Shyr D, Siegel S, Smith AR, Torgerson TR, Vander Lugt MT, Yu LC, Cowan MJ, Buckley RH, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Logan B, Notarangelo LD, Pai SY, Puck J, Pulsipher MA, Heimall J. Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers.. Journal of clinical immunology, 2020.
  43. Kuo CY, Garabedian E, Puck J, Cowan MJ, Sullivan KE, Buckley RH, Cunningham-Rundles C, Marsh R, Candotti F, Kohn DB. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry.. Journal of clinical immunology, 2020.
  44. Kuo CY, Kohn DB. Overview of the current status of gene therapy for primary immune deficiencies (PIDs).. The Journal of allergy and clinical immunology, 2020.
  45. Bradford KL, Pearl M, Kohn DB, Weng P, Yadin O, Bowles V, De Oliveira SN, Moore TB. AT1R Activating Autoantibodies in Hematopoietic Stem Cell Transplantation.. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2020.
  46. 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 Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy.. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2020.
  47. 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, Lahoud MH, Radford KJ. 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, 2020.
  48. Bosticardo M, Pala F, Calzoni E, Delmonte OM, Dobbs K, Gardner CL, Sacchetti N, Kawai T, Garabedian EK, Draper D, Bergerson JRE, DeRavin SS, Freeman AF, Güngör T, Hartog N, Holland SM, Kohn DB, Malech HL, Markert ML, Weinacht KG, Villa A, Seet CS, Montel-Hagen A, Crooks GM, Notarangelo LD. Artificial thymic organoids represent a reliable tool to study T-cell differentiation in patients with severe T-cell lymphopenia.. Blood advances, 2020.
  49. Burroughs LM, Petrovic A, Brazauskas R, Liu X, Griffith LM, Ochs HD, Bleesing JJ, Edwards S, Dvorak CC, Chaudhury S, Prockop SE, Quinones R, Goldman FD, Quigg TC, Chandrakasan S, Smith AR, Parikh S, Dávila Saldaña BJ, Thakar MS, Phelan R, Shenoy S, Forbes LR, Martinez C, Chellapandian D, Shereck E, Miller HK, Kapoor N, Barnum JL, Chong H, Shyr DC, Chen K, Abu-Arja R, Shah AJ, Weinacht KG, Moore TB, Joshi A, DeSantes KB, Gillio AP, Cuvelier GDE, Keller MD, Rozmus J, Torgerson T, Pulsipher MA, Haddad E, Sullivan KE, Logan BR, Kohn DB, Puck JM, Notarangelo LD, Pai SY, Rawlings DJ, Cowan MJ. Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report.. Blood, 2020.
  50. 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, 2020.
  51. Chan AY, Leiding JW, Liu X, Logan BR, Burroughs LM, Allenspach EJ, Skoda-Smith S, Uzel G, Notarangelo LD, Slatter M, Gennery AR, Smith AR, Pai SY, Jordan MB, Marsh RA, Cowan MJ, Dvorak CC, Craddock JA, Prockop SE, Chandrakasan S, Kapoor N, Buckley RH, Parikh S, Chellapandian D, Oshrine BR, Bednarski JJ, Cooper MA, Shenoy S, Davila Saldana BJ, Forbes LR, Martinez C, Haddad E, Shyr DC, Chen K, Sullivan KE, Heimall J, Wright N, Bhatia M, Cuvelier GDE, Goldman FD, Meyts I, Miller HK, Seidel MG, Vander Lugt MT, Bacchetta R, Weinacht KG, Andolina JR, Caywood E, Chong H, de la Morena MT, Aquino VM, Shereck E, Walter JE, Dorsey MJ, Seroogy CM, Griffith LM, Kohn DB, Puck JM, Pulsipher MA, Torgerson TR. Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey.. Frontiers in immunology, 2020.
  52. Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ, Net4CGD consortium. Lentiviral gene therapy for X-linked chronic granulomatous disease.. Nature medicine, 2020.
  53. 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, Krishnamurti L, Little J, Makani J, Malik P, McGann PT, Minniti C, Morris CR, Odame I, Oneal PA, Setse R, Sharma P, Shenoy S. End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings.. Blood advances, 2019.
  54. 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 In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice.. Molecular therapy. Methods & clinical development, 2019.
  55. Morgan RA, Unti MJ, Aleshe B, Brown D, Osborne KS, Koziol C, Ayoub PG, 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, 2019.
  56. 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, McLaughlin J, Cheng D, Hollis RP, Campo-Fernandez B, Urbinati F, Wei L, Pang L, Rezek V, Berent-Maoz B, Macabali MH, Gjertson D, Wang X, Galic Z, Kitchen SG, An DS, Hu-Lieskovan S, Kaplan-Lefko PJ, De Oliveira SN, Seet CS, Larson SM, Forman SJ, Heath JR, Zack JA, Crooks GM, Radu CG, Ribas A, Kohn DB, Witte ON, Yang L. Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer.. Cell stem cell, 2019.
  57. Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GDE, Thakar MS, Chen K, Teira P, Shenoy S, Phelan R, Forbes LR, Chellapandian D, Dávila Saldaña BJ, Shah AJ, Weinacht KG, Joshi A, Boulad F, Quigg TC, Dvorak CC, Grossman D, Torgerson T, Graham P, Prasad V, Knutsen A, Chong H, Miller H, de la Morena MT, DeSantes K, Cowan MJ, Notarangelo LD, Kohn DB, Stenger E, Pai SY, Routes JM, Puck JM, Kapoor N, Pulsipher MA, Malech HL, Parikh S, Kang EM, submitted on behalf of the Primary Immune Deficiency Treatment Consortium. Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT.. Journal of clinical immunology, 2019.
  58. 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, Wang X, Kurmangaliyev YZ, Hollis RP, Kohn DB. 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, 2019.
  59. 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, 2019.
  60. Hazim RA, Karumbayaram S, Jiang M, Dimashkie A, Lopes VS, Li D, Burgess BL, Vijayaraj P, Alva-Ornelas JA, Zack JA, Kohn DB, Gomperts BN, Pyle AD, Lowry WE, Williams DS. Correction to: Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization.. Stem cell research & therapy, 2019.
  61. Soni S, Kohn DB. Chemistry, manufacturing and controls for gene modified hematopoietic stem cells.. Cytotherapy, 2019.
  62. Amatuni GS, Currier RJ, Church JA, Bishop T, Grimbacher E, Nguyen AA, Agarwal-Hashmi R, Aznar CP, Butte MJ, Cowan MJ, Dorsey MJ, Dvorak CC, Kapoor N, Kohn DB, Markert ML, Moore TB, Naides SJ, Sciortino S, Feuchtbaum L, Koupaei RA, Puck JM. Newborn Screening for Severe Combined Immunodeficiency and T-cell Lymphopenia in California, 2010-2017.. Pediatrics, 2019.
  63. 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, 2019.
  64. Kwon HS, Logan AC, Chhabra A, Pang WW, Czechowicz A, Tate K, Le A, Poyser J, Hollis R, Kelly BV, Kohn DB, Weissman IL, Prohaska SS, Shizuru JA. Anti-human CD117 antibody-mediated bone marrow niche clearance in nonhuman primates and humanized NSG mice.. Blood, 2019.
  65. Kohn DB. Gene therapy for blood diseases.. Current opinion in biotechnology, 2018.
  66. 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), 2018.
  67. 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, Goodwin JL, Bolon B, Seet CS, Montel-Hagen A, Crooks GM, Hollis RP, Campo-Fernandez B, Bischof D, Cornetta K, Gschweng EH, Adelson C, Nguyen A, Yang L, Witte ON, Baltimore D, Comin-Anduix B, Kohn DB, Wang X, Cabrera P, Kaplan-Lefko PJ, Berent-Maoz B, Ribas A. 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, 2018.
  68. 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, 2018.
  69. Urbinati F, Campo Fernandez B, Masiuk KE, Poletti V, Hollis RP, Koziol C, Kaufman ML, Brown D, Mavilio F, Kohn DB. Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.. Human gene therapy, 2018.
  70. Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD. T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency.. Genome medicine, 2018.
  71. Hu P, Bi Y, Ma H, Suwanmanee T, Zeithaml B, Fry NJ, Kohn DB, Kafri T. Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization.. Gene therapy, 2018.
  72. Kohn DB, Hershfield MS, Puck JM, Aiuti A, Blincoe A, Gaspar HB, Notarangelo LD, Grunebaum E. Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.. The Journal of allergy and clinical immunology, 2018.
  73. Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Logan B, Griffith LM, Kohn DB, Pai SY, Notarangelo L, Shearer W, Prockop S, Kapoor N, Heimall J, Chaudhury S, Shyr D, Chandra S, Cuvelier G, Moore T, Shenoy S, Goldman F, Smith AR, Sunkersett G, Vander Lugt M, Caywood E, Quigg T, Torgerson T, Chandrakasan S, Craddock J, Dávila Saldaña BJ, Gillio A, Shereck E, Aquino V, DeSantes K, Knutsen A, Thakar M, Yu L, Puck JM. The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018).. The Journal of allergy and clinical immunology, 2018.
  74. Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Prockop SE, Small TN, Chaisson J, Dvorak CC, Murnane M, Kapoor N, Abdel-Azim H, Hanson IC, Martinez C, Bleesing JJH, Chandra S, Smith AR, Cavanaugh ME, Jyonouchi S, Sullivan KE, Burroughs L, Skoda-Smith S, Haight AE, Tumlin AG, Quigg TC, Taylor C, Dávila Saldaña BJ, Keller MD, Seroogy CM, Desantes KB, Petrovic A, Leiding JW, Shyr DC, Decaluwe H, Teira P, Gillio AP, Knutsen AP, Moore TB, Kletzel M, Craddock JA, Aquino V, Davis JH, Yu LC, Cuvelier GDE, Bednarski JJ, Goldman FD, Kang EM, Shereck E, Porteus MH, Connelly JA, Fleisher TA, Malech HL, Shearer WT, Szabolcs P, Thakar MS, Vander Lugt MT, Heimall J, Yin Z, Pulsipher MA, Pai SY, Kohn DB, Puck JM, Cowan MJ, O'Reilly RJ, Notarangelo LD. SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery.. Blood, 2018.
  75. 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, 2018.
  76. Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Abdel-Azim H, Prockop SE, Shyr D, Decaluwe H, Hanson IC, Gillio A, Dávila Saldaña BJ, Eibel H, Hopkins G, Walter JE, Whangbo JS, Kohn DB, Puck JM, Cowan MJ, Griffith LM, Haddad E, O'Reilly RJ, Notarangelo LD, Pai SY. B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation.. Blood, 2018.
  77. Almarza Novoa E, Kasbekar S, Thrasher AJ, Kohn DB, Sevilla J, Nguyen T, Schwartz JD, Bueren JA. Leukocyte adhesion deficiency-I: A comprehensive review of all published cases.. The journal of allergy and clinical immunology. In practice, 2018.
  78. Dunbar CE, High KA, Joung JK, Kohn DB, Ozawa K, Sadelain M. Gene therapy comes of age.. Science (New York, N.Y.), 2018.
  79. 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, 2017.
  80. Morgan RA, Gray D, Lomova A, Kohn DB. Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned.. Cell stem cell, 2017.
  81. Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Pulsipher MA, Parikh S, Martinez C, Kapoor N, O'Reilly R, Boyer M, Pai SY, Goldman F, Burroughs L, Chandra S, Kletzel M, Thakar M, Connelly J, Cuvelier G, Davila Saldana BJ, Shereck E, Knutsen A, Sullivan KE, DeSantes K, Gillio A, Haddad E, Petrovic A, Quigg T, Smith AR, Stenger E, Yin Z, Shearer WT, Fleisher T, Buckley RH, Dvorak CC. Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study.. Blood, 2017.
  82. Hazim RA, Karumbayaram S, Jiang M, Dimashkie A, Lopes VS, Li D, Burgess BL, Vijayaraj P, Alva-Ornelas JA, Zack JA, Kohn DB, Gomperts BN, Pyle AD, Lowry WE, Williams DS. Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization.. Stem cell research & therapy, 2017.
  83. Kohn DB. Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy.. Hematology/oncology clinics of North America, 2017.
  84. Kohn DB. Gene therapy: WAS (not) just for kids.. Blood, 2017.
  85. Bradford KL, Moretti FA, Carbonaro-Sarracino DA, Gaspar HB, Kohn DB. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations.. Journal of clinical immunology, 2017.
  86. Bauer DE, Kohn DB. Gene Therapy.. Hematology/oncology clinics of North America, 2017.
  87. 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. Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease.. Cytotherapy, 2017.
  88. 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, 2017.
  89. 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, 2017.
  90. Cooper AR, Lill GR, Shaw K, Carbonaro-Sarracino DA, Davila A, Sokolic R, Candotti F, Pellegrini M, Kohn DB. Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients.. Blood, 2017.
  91. 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, Ikeda A, Terrazas D, Fu PY, Yu A, Fernandez BC, Cooper AR, Engel B, Podsakoff G, Balamurugan A, Anderson S, Muul L, Jagadeesh GJ, Kapoor N, Tse J, Moore TB, Purdy K, Rishi R, Mohan K, Skoda-Smith S, Buchbinder D, Abraham RS, Scharenberg A, Yang OO, Cornetta K, Gjertson D, Hershfield M, Sokolic R, Candotti F, Kohn DB. Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency.. The Journal of clinical investigation, 2017.
  92. Kohn DB, Kuo CY. New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing.. The Journal of allergy and clinical immunology, 2017.
  93. Kohn DB, Gaspar HB. How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID).. Journal of clinical immunology, 2017.
  94. 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, 2016.
  95. 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 JA, Kitchen SG, An DS, Yang L. Propagating Humanized BLT Mice for the Study of Human Immunology and Immunotherapy.. Stem cells and development, 2016.
  96. 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, 2016.
  97. Punwani D, Kawahara M, Yu J, Sanford U, Roy S, Patel K, Carbonaro DA, Karlen AD, Khan S, Cornetta K, Rothe M, Schambach A, Kohn DB, Malech HL, McIvor RS, Puck JM, Cowan MJ. Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency.. Human gene therapy, 2016.
  98. 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, Hollis RP, Kohn DB. CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells.. Molecular therapy : the journal of the American Society of Gene Therapy, 2016.
  99. Pinello L, Canver MC, Hoban MD, Orkin SH, Kohn DB, Bauer DE, Yuan GC. Analyzing CRISPR genome-editing experiments with CRISPResso.. Nature biotechnology, 2016.
  100. Kuo CY, Kohn DB. Gene Therapy for the Treatment of Primary Immune Deficiencies.. Current allergy and asthma reports, 2016.
  101. Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Shearer WT, Burroughs LM, Torgerson TR, Decaluwe H, Haddad E, workshop participants. Primary Immune Deficiency Treatment Consortium (PIDTC) update.. The Journal of allergy and clinical immunology, 2016.
  102. Kohn DB, Porteus MH, Scharenberg AM. Ethical and regulatory aspects of genome editing.. Blood, 2016.
  103. 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), 2016.
  104. 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, Pyle AD. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells.. Cell stem cell, 2016.
  105. 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, 2016.
  106. 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, 2016.
  107. 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, 2015.
  108. Kohn D, Wilson JM. A Journey in the Development of Gene Therapy for Inherited Disorders of the Bone Marrow.. Human gene therapy. Clinical development, 2015.
  109. 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, 2015.
  110. Kohn DB. Gene therapy outpaces haplo for SCID-X1.. Blood, 2015.
  111. 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), 2015.
  112. 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, Rosenthal E, Hassani M, Singh A, Porteus MH. Genome editing technologies: defining a path to clinic.. Molecular therapy : the journal of the American Society of Gene Therapy, 2015.
  113. 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, 2015.
  114. 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, Paschon DE, Zhang L, Rebar EJ, Wilber A, Wang X, Gregory PD, Holmes MC, Reik A, Hollis RP, Kohn DB. Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.. Blood, 2015.
  115. 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, 2015.
  116. 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, 2015.
  117. 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, 2014.
  118. Kohn DB. Eliminating SCID row: new approaches to SCID.. Hematology. American Society of Hematology. Education Program, 2014.
  119. 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, 2014.
  120. 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, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.. The New England journal of medicine, 2014.
  121. 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, Powell D, Ribas A, Rosenberg S, Sadelain M, Till B, Patterson AP, Jambou RC, Rosenthal E, Gargiulo L, Montgomery M, Kohn DB. T-cell immunotherapy: looking forward.. Molecular therapy : the journal of the American Society of Gene Therapy, 2014.
  122. Kohn DB, Hollis RP. Envelope, please. And the award goes to….. Blood, 2014.
  123. 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, Comeau AM, Connelly JA, Cowan MJ, Cunningham-Rundles C, Dasu T, Dave N, De La Morena MT, Duffner U, Fong CT, Forbes L, Freedenberg D, Gelfand EW, Hale JE, Hanson IC, Hay BN, Hu D, Infante A, Johnson D, Kapoor N, Kay DM, Kohn DB, Lee R, Lehman H, Lin Z, Lorey F, Abdel-Mageed A, Manning A, McGhee S, Moore TB, Naides SJ, Notarangelo LD, Orange JS, Pai SY, Porteus M, Rodriguez R, Romberg N, Routes J, Ruehle M, Rubenstein A, Saavedra-Matiz CA, Scott G, Scott PM, Secord E, Seroogy C, Shearer WT, Siegel S, Silvers SK, Stiehm ER, Sugerman RW, Sullivan JL, Tanksley S, Tierce ML, Verbsky J, Vogel B, Walker R, Walkovich K, Walter JE, Wasserman RL, Watson MS, Weinberg GA, Weiner LB, Wood H, Yates AB, Puck JM, Bonagura VR. Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States.. JAMA, 2014.
  124. 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, Grizzle A, Pulsipher MA, Chan KW, Fuleihan RL, Haddad E, Loechelt B, Aquino VM, Gillio A, Davis J, Knutsen A, Smith AR, Moore TB, Schroeder ML, Goldman FD, Connelly JA, Porteus MH, Xiang Q, Shearer WT, Fleisher TA, Kohn DB, Puck JM, Notarangelo LD, Cowan MJ, O'Reilly RJ. Transplantation outcomes for severe combined immunodeficiency, 2000-2009.. The New England journal of medicine, 2014.
  125. 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, 2014.
  126. 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, 2014.
  127. 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), 2014.
  128. 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, Prograis L, Rosenthal E, Sterman DH, Vandenberghe LH, Zoloth L, Abedi M, Adair J, Adusumilli PS, Goins WF, Gray J, Monahan P, Popplewell L, Sena-Esteves M, Tannous B, Weber T, Wierda W, Gopal-Srivastava R, McDonald CL, Rosenblum D, Corrigan-Curay J. Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.. Human gene therapy, 2014.
  129. 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), 2014.
  130. 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, 2014.
  131. Cannon PM, Kohn DB, Kiem HP. HIV eradication--from Berlin to Boston.. Nature biotechnology, 2014.
  132. 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, Gjertson DW, Cochran AJ, Cornetta K, Wong DJ, Kaplan-Lefko P, Hamid O, Samlowski W, Cohen PA, Daniels GA, Mukherji B, Yang L, Zack JA, Kohn DB, Heath JR, Glaspy JA, Witte ON, Baltimore D, Economou JS, Ribas A. 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, 2014.
  133. Gschweng E, De Oliveira S, Kohn DB. Hematopoietic stem cells for cancer immunotherapy.. Immunological reviews, 2014.
  134. 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, 2013.
  135. 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, 2013.
  136. 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, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB. 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, 2013.
  137. 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, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Dvorak CC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Hovermale E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Logan BR, Long-Boyle JR, Malech HL, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC, workshop participants. Primary Immune Deficiency Treatment Consortium (PIDTC) report.. The Journal of allergy and clinical immunology, 2013.
  138. 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, 2013.
  139. 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, Phelps PE, Pera RA, Byrne JA. Generation and characterization of transgene-free human induced pluripotent stem cells and conversion to putative clinical-grade status.. Stem cell research & therapy, 2013.
  140. 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, 2013.
  141. 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, Goldman F, Kapoor N, Small T, Smith A, Chan KW, Cuvelier G, Heimall J, Knutsen A, Loechelt B, Moore T, Buckley RH. 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, 2013.
  142. 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, 2013.
  143. 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, Kempert P, Shupien S, Abdel-Azim H, Walters MC, Meiselman HJ, Wenby RB, Gruber T, Marder V, Coates TD, Kohn DB. β-globin gene transfer to human bone marrow for sickle cell disease.. The Journal of clinical investigation, 2013.
  144. 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, Montgomery M, Rosenthal E, Samulski RJ, Skarlatos SI, Sorrentino B, Wilson JM, Xie Y, Corrigan-Curay J. Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012.. Human gene therapy, 2013.
  145. 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. 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, 2013.
  146. 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, 2013.
  147. 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, 2013.
  148. 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, 2012.
  149. 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, 2012.
  150. 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, Crooks GM, Kapoor N, Shah A, Abdel-Azim H, Yu XJ, Smogorzewska M, Wayne AS, Rosenblatt HM, Davis CM, Hanson C, Rishi RG, Wang X, Gjertson D, Yang OO, Balamurugan A, Bauer G, Ireland JA, Engel BC, Podsakoff GM, Hershfield MS, Blaese RM, Parkman R, Kohn DB. Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.. Blood, 2012.
  151. 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, 2012.
  152. 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, Montini E, Bushman F, Sorrentino BP, Carrondo M, Malech H, Gahrton G, Shapiro R, Wolff L, Rosenthal E, Jambou R, Zaia J, Kohn DB. 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, 2012.
  153. 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), 2012.
  154. 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, 2012.
  155. 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, 2011.
  156. 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, 2011.
  157. Shaw KL, Kohn DB. A tale of two SCIDs.. Science translational medicine, 2011.
  158. 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, 2011.
  159. 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, Candotti F. Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency.. Blood, 2011.
  160. 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, 2011.
  161. Horwitz EM, Horowitz MM, DiFronzo NL, Kohn DB, Heslop HE, Blood and Marrow Transplant Clinical Trials Network, State of the Science Cell and Gene Therapy Comm. Guidance for developing phase II cell therapy trial proposals for consideration by the Blood and Marrow Transplant Clinical Trials Network.. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2010.
  162. 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, 2010.
  163. 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, 2010.
  164. Shah AJ, Kohn DB. Neurocognitive function of patients with severe combined immunodeficiency.. Immunology and allergy clinics of North America, 2010.
  165. Kohn DB. Update on gene therapy for immunodeficiencies.. Clinical immunology (Orlando, Fla.), 2010.
  166. 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, Shearer WT, Workshop Participants. Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management.. The Journal of allergy and clinical immunology, 2009.
  167. 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, 2009.
  168. 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, 2009.
  169. Kohn DB, Candotti F. Gene therapy fulfilling its promise.. The New England journal of medicine, 2009.
  170. 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, 2009.
  171. 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, Shearer WT. Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs.. The Journal of allergy and clinical immunology, 2008.
  172. 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, 2008.
  173. 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, 2008.
  174. 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, Economou JS, Baltimore D, Mukherji B. 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), 2008.
  175. 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, 2008.
  176. 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, 2008.
  177. 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, 2008.
  178. 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, 2008.
  179. 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, 2008.
  180. Bonde J, Wirthlin L, Kohn DB, Nolta JA. Human hematopoietic cell culture, transduction, and analyses.. Current protocols in human genetics, 2008.
  181. Rossi JJ, June CH, Kohn DB. Genetic therapies against HIV.. Nature biotechnology, 2007.
  182. 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.), 2007.
  183. 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, 2007.
  184. Barwe SP, Maul RS, Christiansen JJ, Anilkumar G, Cooper CR, Kohn DB, Rajasekaran AK. Preferential association of prostate cancer cells expressing prostate specific membrane antigen to bone marrow matrix.. International journal of oncology, 2007.
  185. 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, 2007.
  186. 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, 2007.
  187. 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, 2007.
  188. Kohn DB. Lentiviral vectors ready for prime-time.. Nature biotechnology, 2007.
  189. 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, 2006.
  190. 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, Hershfield MS, Candotti F, Kohn DB. Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report.. Blood, 2006.
  191. Anilkumar G, Barwe SP, Christiansen JJ, Rajasekaran SA, Kohn DB, Rajasekaran AK. Association of prostate-specific membrane antigen with caveolin-1 and its caveolae-dependent internalization in microvascular endothelial cells: implications for targeting to tumor vasculature.. Microvascular research, 2006.
  192. 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, 2006.
  193. 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, 2006.
  194. 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, 2006.
  195. 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, 2006.
  196. 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, 2006.
  197. 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, 2005.
  198. 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, 2005.
  199. 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, 2005.
  200. 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, 2005.
  201. 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.), 2005.
  202. 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, 2005.
  203. 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, von Kalle C, Church JA, Kohn DB. 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, 2005.
  204. 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, 2005.
  205. 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, 2005.
  206. Logan AC, Nightingale SJ, Haas DL, Cho GJ, Pepper KA, Kohn DB. Factors influencing the titer and infectivity of lentiviral vectors.. Human gene therapy, 2004.
  207. 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, 2004.
  208. 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, 2004.
  209. 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, 2004.
  210. 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), 2004.
  211. 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, 2004.
  212. 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, 2004.
  213. Engel BC, Kohn DB, Podsakoff GM. Update on gene therapy of inherited immune deficiencies.. Current opinion in molecular therapeutics, 2003.
  214. 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, 2003.
  215. 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, 2003.
  216. 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, 2003.
  217. 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, Cavagnaro J, High K, Glorioso J, American Society of Gene Therapy (ASGT). 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, 2003.
  218. 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, 2003.
  219. 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, 2003.
  220. 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, 2003.
  221. Kohn DB, Gänsbacher B. Letter to the editors of Nature from the American Society of Gene Therapy (ASGT) and the European Society of Gene Therapy (ESGT).. The journal of gene medicine, 2003.
  222. Kohn DB, Sadelain M, Glorioso JC. Occurrence of leukaemia following gene therapy of X-linked SCID.. Nature reviews. Cancer, 2003.
  223. 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, Von Kalle C. Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing.. Annals of the New York Academy of Sciences, 2003.
  224. 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, 2003.
  225. 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, 2003.
  226. Engel BC, Kohn DB. Gene therapy for inborn and acquired immune deficiency disorders.. Acta haematologica, 2003.
  227. 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, 2003.
  228. Logan AC, Lutzko C, Kohn DB. Advances in lentiviral vector design for gene-modification of hematopoietic stem cells.. Current opinion in biotechnology, 2002.
  229. 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 : the journal of the American Society of Gene Therapy, 2002.
  230. 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), 2002.
  231. 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, 2002.