Donald Kohn, MD

Dr. Donald Kohn has served as an attending physician in the pediatric bone marrow transplant program for more than 30 years. His research focuses on the development of new methods to treat genetic diseases of blood cells by gene modification of hematopoietic stem cells. Kohn's group was the first (and still the only one) to perform a clinical trial with gene transfer to umbilical cord blood CD34+ cells for a genetic disorder (ADA-SCID in 1993) and the first in the U.S. to initiate a clinical trial of gene therapy for pediatric AIDS using bone marrow stem cells.

Kohn's laboratory is currently focused on two main areas: 1) the development and clinical evaluation of improved methods for gene therapy of genetic diseases of blood cells targeting hematopoietic stem cells, especially ADA-deficient SCID and sickle cell disease, and 2) pre-clinical studies focused on the use of lentiviral vectors for gene addition to stem cells and site-specific nucleases (ZFNs, CRISPRs) for gene correction by homologous recombination.

His lab is currently performing clinical trials using lentiviral vectors for ADA-deficient SCID, Sickle Cell Disease and X-linked Chronic Granulomatous Disease. In addition, Kohn's group is participating as one of three U.S. sites for an NIAID-funded clinical trial of gene therapy for X-SCID.

Masiuk KE, Brown D, Laborada J, Hollis RP, Urbinati F, Kohn DB. Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells. Mol Ther. 2017 Jun 26. pii: S1525-0016(17)30259-9. doi: 10.1016/j.ymthe.2017.05.023. [Epub ahead of print]

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. J Clin Invest. 2017 May 1;127(5):1689-1699. doi: 10.1172/JCI90367. Epub 2017 Mar 27.

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. Beta-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest. 2013 Jul 1. pii: 67930. doi: 10.1172/JCI67930. [Epub ahead of print]

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. Mol Ther. 2016 Sep;24(9):1561-9. doi: 10.1038/mt.2016.148. Epub 2016 Jul 29.

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. Mol Ther. 2013 May;21(5):1044-54. doi: 10.1038/mt.2013.8. Epub 2013 Feb 5.