Publications

A case of progressive multifocal leukoencephalopathy in an adult patient with ADA-SCID. Shin H, Broderick L, Azevedo J, Mulligan C, Hird K, Goodwill V, Parkin C, Mortensen S, Traveller M, Rayes A, Bohnsack J, Kohn DB, Tarrant TK, Hershfield M, Lyons JJ. J Hum Immun. 2026 Jul 21;2(5):e20250093. doi: 10.70962/jhi.20250093. eCollection 2026 Sep 7. PMID: 42483435

Long-term stability of posttranscriptional genetic silencing of BCL11A using a shmiR vector in sickle cell disease. Esrick EB, Lehmann L, Federico A, Vincon H, Liu B, Daley H, Dansereau C, DeOliveira SN, Everett J, Kao PC, Moore TB, Morris E, Negre H, Shaw KL, Roach GD, Ritz J, Roche AM, Silva O, Grant PE, Bushman FD, Kohn DB, Jain A, London WB, Justus DG, Armant M, Manis J, Williams DA. Blood. 2026 Aug 20;148(8):957-966. doi: 10.1182/blood.2026033871. PMID: 42233421

Search for the grail: chemotherapy-free stem cell gene therapy. Kohn DB. Blood. 2026 Aug 20;148(8):924-925. doi: 10.1182/blood.2026034570. PMID: 42623101

Clinical outcomes of lentiviral vector gene therapy for sickle cell disease. Prueksapraopong C, Fernandes A, Campo Fernandez B, Roy S, Hollis RP, Habtemariam B, Pellin D, Ceoldo G, Lin TY, Dang TT, Cornetta K, Romero Z, Blazar BR, Shah AJ, Moore TB, Sehl M, Schiller GJ, Kohn DB. Blood Adv. 2026 Jul 28;10(14):4991-5002. doi: 10.1182/bloodadvances.2026019869. PMID: 42008008

Synthesis errors in oligonucleotides are propagated into the genome by homology-directed repair. Wyman SK, Romero Z, Heo SJ, Navarrete M, Krishnappa N, Kohn DB, Martin DI, Walters MC, Boffelli D. bioRxiv [Preprint]. 2025 Jul 11:2025.07.08.663753. doi: 10.1101/2025.07.08.663753. Update in: Genes (Basel). 2026 Jun 24;17(7):729. doi: 10.3390/genes17070729. PMID: 40672186 Free PMC article. Preprint

Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Alexander JL, Dávila Saldaña BJ, Brazauskas R, Dammalapati SG, Griffith LM, Shah AJ, Shimano KA, Ochs HD, Bleesing JJ, Ebens CL, Kapadia M, Bauchat A, Kapoor N, Oved JH, Eissa H, Lust H, Keller MD, Haines H, Chandrakasan S, Talano JA, Rayes A, Madden L, Shereck E, Miller HK, Satter LF, Martinez C, Rozmus J, Bednarski JJ, Yu LC, Chellapandian D, Aquino VM, Knutsen A, Chong H, Chopek A, Gillio AP, Joshi A, Rangarajan H, Moore TB, Andolina JR, DeSantes KB, Vander Lugt M, Prockop SE, Shyr DC, Sullivan KE, Parikh S, Weinacht KG, Torgerson TR, Marsh R, Dvorak CC, Chan AY, Haddad E, Heimall JR, Pulsipher MA, Leiding JW, Kohn DB, Puck JM, Notarangelo LD, Rawlings DJ, Cowan MJ, Petrovic A, Pai SY, Burroughs LM. Blood Adv. 2026 Mar 10;10(5):1783-1798. doi: 10.1182/bloodadvances.2025017662. PMID: 41346295 Free PMC article

Outcomes following matched sibling donor transplantation for severe combined immunodeficiency: a report from the PIDTC. Rayes A, Logan BR, Liu X, Dara J, Buckley RH, Oved JH, Kapoor N, Kapadia M, Chandra S, Martinez CA, Bunin NJ, Chandrakasan S, Chen K, Bednarski JJ, Haines HL, Eissa H, Talano JM, Keller MD, Ebens CL, Chaudhury S, Shereck EB, Aquino VM, Knutsen AP, Alexander JL, Gillio AP, Chellapandian D, Shah AJ, Miller HK, Vander Lugt MT, Seroogy CM, Dorsey MJ, Mousallem T, Parrott RE, O’Reilly RJ, Aguayo-Hiraldo PI, Prockop SE, Dávila Saldaña BJ, Thakar MS, Burroughs LM, Torgerson TR, Leiding JW, Marsh RA, Griffith LM, Pulsipher MA, Kohn DB, Notarangelo L, Cowan MJ, Puck JM, Cuvelier GDE, Heimall J, Haddad E, Pai SY, Dvorak CC. Blood Adv. 2026 Feb 10;10(3):887-900. doi: 10.1182/bloodadvances.2025016812. PMID: 41289158 Free PMC article

Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study. Brumm VL, Kidd SA, Logan BR, Chunara F, Heimall J, Griffith LM, Kohn DB, Sanchez L, Bednarski JJ, Martinez C, Lugt MV, Kapoor N, Wright N, Spitzer B, Oved JH, Chandra S, Chellapandian D, Ebens CL, Petrovic A, Rayes A, Haines HL, Lust H, Schofield HT, Christopher L, Harris LL, Satter LF, Burroughs L, Dvorak CC, Haddad E, Leiding JW, Marsh RA, Notarangelo LD, Pai SY, Pulsipher MA, Puck JM, Cowan MJ, Shah AJ. J Hum Immun. 2025 Dec 17;2(1):e20250163. doi: 10.70962/jhi.20250163. eCollection 2026 Jan 5. PMID: 41608119

Clinical spectrum of Wiskott-Aldrich syndrome carriers: Self-reported survey of 193 carriers. Chandrakasan S, Westbrook A, Griffith LM, Hagin D, Iyengar S, Mangurian C, Scalchunes C, Sullivan KE, Zablocki A, Bakshi N, Sinha C, Burroughs LM, Chan AY, Dvorak CC, Haddad E, Heimall J, Kohn DB, Leiding JW, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Marsh RA, Torgerson T, Cowan MJ, Ochs HD, Parikh S. Clin Immunol. 2026 Feb;283:110658. doi: 10.1016/j.clim.2025.110658. Epub 2025 Dec 17. PMID: 41419161 Free PMC article

Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells. Segura EER, Hart K, Campo Fernandez B, Brown D, Tam K, Gutierrez Garcia A, Seigneurbieux E, Li K, Mulumba C, Blakely E, Masiuk K, Sinha R, Sharma D, Everett J, Hogenauer M, Cromer MK, Bushman F, MacKenzie TC, Kohn DB. Cell Rep Med. 2025 Oct 21;6(10):102362. doi: 10.1016/j.xcrm.2025.102362. Epub 2025 Sep 17. PMID: 40967220 Free PMC article

Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency. Booth C, Masiuk K, Vazouras K, Fernandes A, Xu-Bayford J, Campo Fernandez B, Roy S, Curio-Penny B, Arnold J, Terrazas D, Reid J, Gilmour KC, Adams S, Alvarez Mediavilla E, Mhaldien L, O’Toole G, Ahmed R, Garabedian E, Malech H, De Ravin SS, Moore TB, De Oliveira S, Pellin D, Lin TY, Dang TT, Cornetta K, Hershfield MS, Hara H, Thrasher AJ, Gaspar HB, Kohn DB. N Engl J Med. 2025 Oct 16;393(15):1486-1497. doi: 10.1056/NEJMoa2502754. PMID: 41092330

Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report. Arnold DE, Leiding JW, Logan B, Marsh RA, Griffith LM, Grunebaum E, Murguía-Favela L, Mallhi K, Chellapandian D, Deal CL, Lim SS, Prasad V, Heimall J, Chandrakasan S, Chen K, Yu LC, Seroogy CM, Gillio A, Bednarski JJ, Kapoor N, Moore TB, Cuvelier GDE, Touzot F, Rayes A, Ebens CL, Schaefer E, Bauchat A, Chopek A, Burroughs L, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson T, Malech HL, Kang EM, Parikh S. Transplant Cell Ther. 2026 Jan;32(1):89.e1-89.e10. doi: 10.1016/j.jtct.2025.05.020. Epub 2025 Oct 2. PMID: 41046057

Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia. Luthers CR, Mittelhauser A, Colamartino A, Wu X, Cirigliano S, Long JD, Sanchez JM, Romero Z, Kohn DB. Mol Ther Methods Clin Dev. 2025 Aug 12;33(3):101555. doi: 10.1016/j.omtm.2025.101555. eCollection 2025 Sep 11. PMID: 40917692 Free PMC article

Human cancer-targeted immunity via transgenic hematopoietic stem cell progeny. Nowicki TS, Deen NNA, Peters CW, Comin-Anduix B, Medina E, Puig-Saus C, Carretero IB, Kaplan-Lefko P, Macabali MH, Garcilazo IP, Chen D, Pang J, Berent-Maoz B, Haile S, Rodriguez J, Kawakami M, Kidd CK, Champhekar A, Carlucci G, Vega-Crespo A, Chmielowski B, Singh A, Federman N, Schiller GM, Larson SJ, Allen-Auerbach M, Klomhaus AM, Zack J, Baltimore D, Yang L, Kohn DB, Witte ON, Ribas A. Nat Commun. 2025 Jul 1;16(1):5599. doi: 10.1038/s41467-025-60816-z. PMID: 40593578 Free PMC article. Clinical Trial

Quantifying the mutational landscape of retroviral and lentiviral vectors in gene therapy patients. Hart KL, Crisostomo RV, Mittelhauser A, Zhan L, Kononov N, Bradford K, Kohn DB. Mol Ther. 2025 May 30:S1525-0016(25)00404-6. doi: 10.1016/j.ymthe.2025.05.034. Online ahead of print. PMID: 40450520 Free article

Response to: Safety and efficacy considerations of HSC-based gene therapy for RAG1-deficient SCID. Montini E, Naldini L, Booth C, Kohn DB, Aiuti A. Mol Ther. 2025 May 7;33(5):1871. doi: 10.1016/j.ymthe.2025.03.049. Epub 2025 Apr 15. PMID: 40233756

Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy. Williams DA, Kohn DB, Thrasher AJ. Mol Ther. 2025 May 7;33(5):2141-2153. doi: 10.1016/j.ymthe.2025.03.058. Epub 2025 Apr 1. PMID: 40176348 Review

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1. Booth C, Sevilla J, Almarza E, Kuo CY, Zubicaray J, Terrazas D, O’Toole G, Chitty-Lopez M, Choi G, Nicoletti E, Long-Boyle J, Fernandes A, Chetty K, De Oliveira S, Banuelos C, Xu-Bayford J, Bastone AL, John-Neek P, Jackson C, Moore TB, Gilmour K, Schambach A, Rothe M, Kasbekar S, Rao GR, Patel K, Shah G, Thrasher AJ, Bueren JA, Schwartz JD, Kohn DB. N Engl J Med. 2025 May 1;392(17):1698-1709. doi: 10.1056/NEJMoa2407376. PMID: 40305711 Clinical Trial

Balancing efficacy and safety in lentiviral vector-mediated hematopoietic stem cell gene therapy. Montini E, Naldini L, Booth C, Kohn DB, Aiuti A. Mol Ther. 2025 Jan 8;33(1):6-8. doi: 10.1016/j.ymthe.2024.12.028. Epub 2024 Dec 26. PMID: 39729983

Myelodysplasia after Lentiviral Gene Therapy. Kohn DB, Booth C, Naldini L. N Engl J Med. 2024 Dec 19;391(24):2382. doi: 10.1056/NEJMc2414069. PMID: 39693551

Stage-specific CAR-mediated signaling generates naïve-like, TCR-null CAR T cells from induced pluripotent stem cells. Yoo SP, Yuan X, Engstrom C, Chang P, Li S, Lathrop L, Lagosh J, Seet C, Kohn DB, Crooks GM. bioRxiv [Preprint]. 2024 Nov 28:2024.11.25.624041. doi: 10.1101/2024.11.25.624041. PMID: 39651198 Free PMC article. Preprint

Making sense of adenosine deaminase variants and their clinical implications. Grunebaum E, Loves R, Kohn DB. J Allergy Clin Immunol. 2025 Jan;155(1):92-93. doi: 10.1016/j.jaci.2024.11.010. Epub 2024 Nov 16. PMID: 39551441

DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34 + cell clonogenic potential. Luthers CR, Ha SM, Mittelhauser A, Morselli M, Long JD, Kuo CY, Romero Z, Kohn DB. Mol Ther Methods Clin Dev. 2024 Sep 12;32(4):101334. doi: 10.1016/j.omtm.2024.101334. eCollection 2024 Dec 12. PMID: 39381161 Free PMC article

Lentiviral vectors for precise expression to treat X-linked lymphoproliferative disease. Ayoub PG, Gensheimer J, Lathrop L, Juett C, Quintos J, Tam K, Reid J, Ma F, Tam C, McAuley GE, Brown D, Wu X, Zhang R, Bradford K, Hollis RP, Crooks GM, Kohn DB. Mol Ther Methods Clin Dev. 2024 Aug 20;32(4):101323. doi: 10.1016/j.omtm.2024.101323. eCollection 2024 Dec 12. PMID: 39309261 Free PMC article

Enhancing pediatric access to cell and gene therapies. Mackall CL, Bollard CM, Goodman N, Carr C, Gardner R, Rouce R, Sotillo E, Stoner R, Urnov FD, Wayne AS, Park J, Kohn DB. Nat Med. 2024 Jul;30(7):1836-1846. doi: 10.1038/s41591-024-03035-1. Epub 2024 Jun 17. PMID: 38886624 Review

Generation of allogeneic CAR-NKT cells from hematopoietic stem and progenitor cells using a clinically guided culture method.  Lusis AJ, Zhou JJ, Seet CS, Kohn DB, Wang P, Zhou XJ, Pellegrini M, Puliafito BR, Larson SM, Yang L.Nat Biotechnol. 2024 May 14. doi: 10.1038/s41587-024-02226-y. Online ahead of print.PMID: 38744947

Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A  Primary Immune Deficiency Treatment Consortium study. Parta M, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM, Leiding JW.J Allergy Clin Immunol. 2024 May;153(5):1423-1431.e2. doi: 10.1016/j.jaci.2024.01.013. Epub 2024 Jan 28.PMID: 38290608

A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease. Hart KL, Liu B, Brown D, Campo-Fernandez B, Tam K, Orr K, Hollis RP, Brendel C, Williams DA, Kohn DB. Mol Ther Methods Clin Dev. 2024 Apr 24;32(2):101254. doi: 10.1016/j.omtm.2024.101254. eCollection 2024 Jun 13.PMID: 3874589

Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment. Keller MD, Hanley PJ, Chi YY, Aguayo-Hiraldo P, Dvorak CC, Verneris MR, Kohn DB, Pai SY, Dávila Saldaña BJ.Nat Commun. 2024 Apr 18;15(1):3258. doi: 10.1038/s41467-024-47057-2.PMID: 38637498

Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia. Torgerson TR, Leiding JW, Pai SY, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Cowan MJ, Puck J, Dvorak CC, Haddad E.Clin Immunol. 2024 Apr;261:109942. doi: 10.1016/j.clim.2024.109942. Epub 2024 Feb 15.PMID: 38367737

What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity  Mudde ACA, Kuo CY, Kohn DB, Booth C. J Allergy Clin Immunol Pract. 2024 Jan 19:S2213-2198(24)00071-0. doi: 10.1016/j.jaip.2024.01.019. Epub ahead of print. PMID: 38246560

Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study. Bleesing JH, Prockop S, Kohn DB, Bednarski JJ, Leiding J, Marsh RA, Torgerson T, Notarangelo LD, Pai SY, Pulsipher MA, Puck JM, Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Heimall J. J Allergy Clin Immunol. 2024 Jan;153(1):287-296. doi: 10.1016/j.jaci.2023.09.027. Epub 2023 Oct 2.PMID: 37793572

Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease. Touzot F, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Kang EM, Malech HL, Segre JA, Bryant CE, Holland SM, Falcone EL.J Allergy Clin Immunol. 2023 Dec;152(6):1619-1633.e11. doi: 10.1016/j.jaci.2023.07.022. Epub 2023 Sep 1.PMID: 37659505

Biodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair  Bell JA, Collon K, Mayfield C, Gallo MC, Chang SW, Sugiyama O, Tang AH, Hollis R, Chopra S, Kohn DB, Lieberman JR. Biodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair. Gene Ther. 2023 Dec;30(12):826-834. doi: 10.1038/s41434-023-00415-z. Epub 2023 Aug 11. PMID: 37568039

Gene therapy for adenosine deaminase severe combined immune deficiency-An unexpected journey of four decades  Kohn DB. Gene therapy for adenosine deaminase severe combined immune deficiency-An unexpected journey of four decades. Immunol Rev. 2023 Nov 30. doi: 10.1111/imr.13293. Epub ahead of print. PMID: 38033164

Successes and challenges in clinical gene therapy  Kohn DB, Chen YY, Spencer MJ. Successes and challenges in clinical gene therapy. Gene Ther. 2023 Nov;30(10-11):738-746. doi: 10.1038/s41434-023-00390-5. Epub 2023 Nov 8. PMID: 37935854; PMCID: PMC10678346

Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease Recapitulates Endogenous CYBB Regulation and Expression. Wong RL, Sackey S, Brown D, Senadheera S, Masiuk K, Quintos JP, Colindres N, Riggan L, Morgan RA, Malech HL, Hollis RP, Kohn DB. Blood. 2023. 14:1007-1022. PMID: 36332160

Busulfan and subsequent malignancy: An evidence-based risk assessment  Long-Boyle JR, Kohn DB, Shah AJ, Spencer SM, Sevilla J, Booth C, López Lorenzo JL, Nicoletti E, Shah A, Reatz M, Matos J, Schwartz JD. Busulfan and subsequent malignancy: An evidence-based risk assessment. Pediatr Blood Cancer. 2024 Jan;71(1):e30738. doi: 10.1002/pbc.30738. Epub 2023 Oct 19. PMID: 37856098

Fluorinated Silane-Modified Filtroporation Devices Enable Gene Knockout in Human Hematopoietic Stem and Progenitor Cells  Frost IM, Mendoza AM, Chiou TT, Kim P, Aizenberg J, Kohn DB, De Oliveira SN, Weiss PS, Jonas SJ. Fluorinated Silane-Modified Filtroporation Devices Enable Gene Knockout in Human Hematopoietic Stem and Progenitor Cells. ACS Appl Mater Interfaces. 2023 Sep 6;15(35):41299-41309. doi: 10.1021/acsami.3c07045. Epub 2023 Aug 24. PMID: 37616579; PMCID: PMC10485797

Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials  Segura EER, Ayoub PG, Hart KL, Kohn DB. Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials. Viruses. 2023 Mar 9;15(3):713. doi: 10.3390/v15030713. PMID: 36992422; PMCID: PMC10054523

Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing. 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. Cell. 2023. 186:1398-1416.e23. PMID: 36944331

Evaluation of clonal hematopoiesis in pediatric ADA-SCID gene therapy participants. White SL, Lee TD, Toy T, Carroll JE, Polsky L, Campo Fernandez B, Davila A, Kohn DB, Chang VY. Blood Adv. 2022 Nov 8;6(21):5732-5736. doi: 10.1182/bloodadvances.2022007803. PMID: 35914227 Free PMC article

Diverse Approaches to Gene Therapy of Sickle Cell Disease. White SL, Hart K, Kohn DB. Annu Rev Med. 2022 Sep 6. doi: 10.1146/annurev-med-042921-021707. Online ahead of print. PMID: 36067800 Review

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC. 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. Blood. 2022 Aug 18;140(7):685-705. doi: 10.1182/blood.2022016196. PMID: 35671392 Clinical Trial

High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation. 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. iScience. 2022 May 10;25(6):104374. doi: 10.1016/j.isci.2022.104374. eCollection 2022 Jun 17. PMID: 35633935 Free PMC article

mproved lentiviral vector titers from a multi-gene knockout packaging line. Han J, Tam K, Tam C, Hollis RP, Kohn DB. Mol Ther Oncolytics. 2021 Nov 20;23:582-592. doi: 10.1016/j.omto.2021.11.012. eCollection 2021 Dec 17. PMID: 34938858 Free PMC article

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency. 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. Blood. 2021 Oct 14;138(15):1304-1316. doi: 10.1182/blood.2020010260. PMID: 33974038 Free PMC article. Clinical Trial

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. 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. N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11. PMID: 33974366 Free PMC article. Clinical Trial

Optimizing Integration and Expression of Transgenic Bruton’s Tyrosine Kinase for CRISPR-Cas9-Mediated Gene Editing of X-Linked Agammaglobulinemia. Gray DH, Villegas I, Long J, Santos J, Keir A, Abele A, Kuo CY, Kohn DB. CRISPR J. 2021 Apr;4(2):191-206. doi: 10.1089/crispr.2020.0080. PMID: 33876953 Free PMC article

Gene Therapies for Primary Immune Deficiencies. Kohn LA, Kohn DB. Front Immunol. 2021 Feb 25;12:648951. doi: 10.3389/fimmu.2021.648951. eCollection 2021. PMID: 33717203 Free PMC article. Review

Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID. Carbonaro-Sarracino DA, Chun K, Clark DN, Kaufman ML, Jin X, Wang X, Kohn DB. Mol Ther Methods Clin Dev. 2021 Feb 15;20:765-778. doi: 10.1016/j.omtm.2021.02.007. eCollection 2021 Mar 12. PMID: 33738330 Free PMC article

β-Globin Lentiviral Vectors Have Reduced Titers due to Incomplete Vector RNA Genomes and Lowered Virion Production. Han J, Tam K, Ma F, Tam C, Aleshe B, Wang X, Quintos JP, Morselli M, Pellegrini M, Hollis RP, Kohn DB. Stem Cell Reports. 2021 Jan 12;16(1):198-211. doi: 10.1016/j.stemcr.2020.10.007. Epub 2020 Nov 12. PMID: 33186538 Free PMC article

Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells. 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. Front Genome Ed. 2020 Dec 10;2:601541. doi: 10.3389/fgeed.2020.601541. eCollection 2020. PMID: 34713224 Free PMC article

Overview of the current status of gene therapy for primary immune deficiencies (PIDs). Kuo CY, Kohn DB. J Allergy Clin Immunol. 2020 Aug;146(2):229-233. doi: 10.1016/j.jaci.2020.05.024. PMID: 32771134 Review

Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy. Bradford KL, Liu S, Krajinovic M, Ansari M, Garabedian E, Tse J, Wang X, Shaw KL, Gaspar HB, Candotti F, Kohn DB. Biol Blood Marrow Transplant. 2020 Oct;26(10):1819-1827. doi: 10.1016/j.bbmt.2020.07.004. Epub 2020 Jul 9. PMID: 32653625 Free PMC article

Lentiviral gene therapy for X-linked chronic granulomatous disease. 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. Nat Med. 2020 Feb;26(2):200-206. doi: 10.1038/s41591-019-0735-5. Epub 2020 Jan 27. PMID: 31988463 Free PMC article. Clinical Trial

mproved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements.  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. Mol Ther. 2020 Jan 8;28(1):328-340. doi: 10.1016/j.ymthe.2019.09.020. Epub 2019 Sep 28. PMID: 31628051 Free PMC article

Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice.  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. Mol Ther Methods Clin Dev. 2019 Nov 16;16:78-93. doi: 10.1016/j.omtm.2019.11.004. eCollection 2020 Mar 13. PMID: 31871959 Free PMC article

Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates.  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. Mol Ther. 2019 Aug 7;27(8):1389-1406. doi: 10.1016/j.ymthe.2019.05.014. Epub 2019 May 24. PMID: 31178391 Free PMC article

PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a β-Globin Lentiviral Vector. Masiuk KE, Zhang R, Osborne K, Hollis RP, Campo-Fernandez B, Kohn DB. Mol Ther Methods Clin Dev. 2019 Apr 4;13:390-398. doi: 10.1016/j.omtm.2019.03.005. eCollection 2019 Jun 14. PMID: 31024981 Free PMC article