Publications

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. What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity. 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.

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.

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.

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.

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.

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

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.

Improved 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.

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.

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.

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.

Improved 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.