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个人简介
The research in the Kohn lab focuses on the development of new methods to treated genetic diseases of blood cells by gene modification of hematopoietic stem cells. Dr. 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 clinical trials of gene therapy for pediatric HIV/AIDS using bone marrow stem cells. Current projects include (1) performing clinical trials for ADA-deficient SCID, XSCID, Sickle Cell Disease and X-linked Chronic Granulomatous Disease, (2) developing new lentiviral vectors for regulated gene expression, (3) advancing techniques for site-specific gene editing for sickle cell disease and primary immune deficiency diseases, and (4) improving clinical hematopoietic stem cell processing. Our research group spans the bench-to-bedside spectrum and provides an excellent training environment for physician/scientists.
研究兴趣
论文共 671 篇作者统计合作学者相似作者
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Janel R. Long-Boyle,Donald B. Kohn,Ami J. Shah, Sueli Marques Spencer,Julian Sevilla,Claire Booth,Jose Luis Lopez Lorenzo,Eileen Nicoletti, Arpita Shah, Meredith Reatz, Joana Matos,Jonathan D. Schwartz
PEDIATRIC BLOOD & CANCERno. 1 (2024)
Stephanie Si Lim, Christin Deal,Kyle Ishikawa,Athanasios Tsalatsanis,Elisa Ochfeld, Aleks Petrovic,Kanwaldeep Mallhi,Avni Joshi,Kristin Shimano,Christopher Dvorak,Elie Haddad,Donald B. Kohn,
Clinical Immunology (2024): 110111
Christin Deal, Stephanie Si Lim,Kyle Ishikawa, Athanasios Tsalatsanis,Elisa Ochfeld, Aleks Petrovic,Kanwaldeep Mallhi,Avni Joshi, Kristin Shimano,Christopher Dvorak,Elie Haddad,Donald B. Kohn,
crossref(2023)
Gene therapyno. 10-11 (2023): 753-755
Molecular Genetics and Metabolismno. 2 (2023): 26-26
crossref(2023)
Bloodno. 10 (2023): 1098-1099
MOLECULAR THERAPYno. 4 (2023): 744-744
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