Donald Barry Kohn MD

Pediatrics•Los Angeles, CA

Professor, Pediatrics, UCLA School of Medicine

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4650 W Sunset BlvdMS 62Los Angeles, CA 90027
Phone+1 323-669-4559
Fax+1 323-660-1904

Education & Training

National Institutes of Health Clinical CenterFellowship, Allergy and Immunology, 1986 - 1988
University of Wisconsin Hospitals and ClinicsResidency, Pediatrics, 1982 - 1985
University of Wisconsin School of Medicine & Public HealthClass of 1982

Certifications & Licensure

CA State Medical License 1987 - 2025
WI State Medical License 1983 - 2017
American Board of Pediatrics Pediatrics

Awards, Honors, & Recognition

Super Doctor SuperDoctors.com

Clinical Trials

MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID Start of enrollment: 2008 Nov 01
Completed
Phase 2
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector Start of enrollment: 2010 Apr 01
Completed
Phase 1, Phase 2
Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene Start of enrollment: 2013 Aug 02
Completed
Phase 1, Phase 2
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD) Start of enrollment: 2013 Aug 21
Completed
Phase 2, Phase 3
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease Start of enrollment: 2015 Oct 29
COMPLETED
PHASE1, PHASE2
Stem Cell Gene Therapy for Sickle Cell Disease Start of enrollment: 2014 Dec 01
Active, not recruiting
Phase 1, Phase 2
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID Start of enrollment: 2016 Dec 16
Completed
Phase 1, Phase 2
A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I Start of enrollment: 2019 Aug 30
Completed
Phase 1, Phase 2
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Publications & Presentations

PubMed

Response to: Safety and efficacy considerations of HSC-based gene therapy for RAG1-deficient SCID.
Eugenio Montini, Luigi Naldini, Claire Booth, Donald B Kohn, Alessandro Aiuti
Molecular Therapy. 2025-04-14
Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy.
David A Williams, Donald B Kohn, Adrian J Thrasher
Molecular Therapy. 2025-04-01
18 citations
Generation of allogeneic CAR-NKT cells from hematopoietic stem and progenitor cells using a clinically guided culture method.
Yan-Ruide Li, Yang Zhou, Jiaji Yu, Yu Jeong Kim, Miao Li
Nature Biotechnology. 2025-03-01

Journal Articles

T Cell Dynamics and Response of the Microbiota After Gene Therapy to Treat X-linked Severe Combined Immunodeficiency
Luigi D Notarangelo, Judith Kelsen, Ronald G Collman, Donald B Kohn, BioMed Central
IND-Enabling Studies for a Clinical Trial to Genetically Program a Persistent Cancer-Targeted Immune System
Kenneth Cornetta, Antoni Ribas, Gay M Crooks, Donald B Kohn, Clinical Cancer Research

Lectures

Friday Scientific Workshop on Novel Curative Options: Gene-Editing and Gene Therapy for Hemoglobinopathies with a Focus on Sickle Cell Disease
61st Annual American Society of Hematology Meeting - Orlando, FL - 12/7/2019
Completing the Genome Editing Arc in Hematology
60th American Society of Hematology Annual Meeting - 12/4/2018

Press Mentions

Gene Therapy Restores Immune Function and Extends Lives of Children with Rare Immune DisorderApril 25th, 2025
Breathing New Life: Inhalable Gene Therapy for Cystic FibrosisMarch 3rd, 2025
UCLA Scientists Awarded $21.8 Million in CIRM Grants to Advance Novel Stem-Cell Based TherapiesJanuary 30th, 2025
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Grant Support

Transduction Of Hematopoietic Stem Cells For Enhanced Immunotherapy Of MelanomaNational Cancer Institute2010–2012
Training In Developmental HematologyNational Heart, Lung, And Blood Institute2011
In Vivo ADA Gene Delivery For The Treatment Of SCIDNational Institute Of Allergy And Infectious Diseases2009–2011
Gene Therapy Using Hematopoietic Stem CellsNational Heart, Lung, And Blood Institute2009–2011
In Vivo ADA Gene Delivery For The Treatment Of SCIDNational Center For Research Resources2009–2011
Mnd-Ada Transduced CD34 Cells For Ada-ScidFood And Drug Administration2005–2011
Clinical Trial: Transduction Of CD34+ Cells From The Umbilical Cord Blood Of InfNational Center For Research Resources2009
Clinical Trial: Long-Term Follow-Up For Studies Of Gene Transfer (Hiv And OthersNational Center For Research Resources2009
Lentiviral Vector For Gene Transfer To Hematopoietic Stem CellsNational Center For Research Resources2007–2009
In Vivo ADA Gene Delivery For The Treatment Of SCIDNational Institute Of Allergy And Infectious Diseases2007–2008
Long-Term Follow-Up For Studies Of Gene Transfer (Hiv And Others)National Center For Research Resources2007–2008
Conference--American Society Of Gene TherapyNational Heart, Lung, And Blood Institute2004–2008
Transduction Of CD34+ Cells From The Umbilical Cord Blood Of Infants Or The BNational Center For Research Resources2006–2007
Gene Therapy For Ada-Deficient SCIDNational Heart, Lung, And Blood Institute2004–2007
Gene Therapy Using Hematopoietic Stem CellsNational Heart, Lung, And Blood Institute2003–2007
Clinical Trial Planning Grant: Lentiviral Vector For HIV-1National Institute Of Allergy And Infectious Diseases2006
Long-Term Follow-Up For Studies Of Gene TransferNational Center For Research Resources2005–2006
Lentiviral Vector Transfer To Hematopoietic Stem CellsNational Center For Research Resources2003–2006
Treatment Of Severe Combined Immune Deficiency Disease Due To ADA DeficiencyNational Center For Research Resources2005
Mnd-Ada Transduced CD34 Cells For Ada-ScidFood And Drug Administration2005
Lentiviral Vector Transfer To Hematopoietic Stem CellsNational Institute Of Allergy And Infectious Diseases2002–2005
Core A-- Administrative CoreNational Heart, Lung, And Blood Institute2004
Treatment Of Severe Combined Immune Deficiency DiseaseNational Center For Research Resources2004
Transfer Of Revm10 And FX Genes Into Bone MarrowNational Center For Research Resources2004
Transduction Of CD34+ Cells From The Bone Marrow Of HIV-1 Infected ChildrenNational Center For Research Resources1998–2004
Gene Expression In Beta-Cells By Lentiviral VectorsNational Institute Of Diabetes And Digestive And Kidney Diseases2002–2003
Core--Gene Transduction And Gene TherapyNational Heart, Lung, And Blood Institute2000–2002
Retrovirus And Lentivirus For Stem Cell TransductionNational Institute Of Allergy And Infectious Diseases1999–2002
In Vitro And In Vivo Models Of Human ErythropoiesisNational Heart, Lung, And Blood Institute1998–2002
Transduction Of CD34 Peripheral Blood Progenital Cell From HIV Infected PersonsNational Center For Research Resources1998–2002
Mechanisms Of Gene SilencingNational Cancer Institute1996–2002
Core--Vector DevelopmentNational Cancer Institute1996–2002
Gene Therapy For Hurlers DiseaseNational Institute Of Diabetes And Digestive And Kidney Diseases1998–2001
Hematopoietic Stem Cells For Gene Therapy With REV M10National Institute Of Allergy And Infectious Diseases1998–2001
Modified Retroviral Vectors For HSC Gene TherapyNational Institute Of Diabetes And Digestive And Kidney Diseases1999–2000
Treatment Of Severe Combined Immunodeficiency Disease Due To ADA DeficiencyNational Center For Research Resources1997–1999
In Vivo Model For Human Stem CellsNational Heart, Lung, And Blood Institute1996–1999
Core--Gene Therapy/Molecular Biology LaboratoryNational Heart, Lung, And Blood Institute1996–1999
Retroviral Transfer Of CDNA For Glucocerebrosidase Into Stem CellsNational Center For Research Resources1996–1999
Retroviral Transduction Of Anti-Hiv Ribozyme Into Hematopoietic Stem CellsNational Institute Of Allergy And Infectious Diseases1996–1998
Hematopoietic Gene Transfer--Pediatric AIDSNational Institute Of Allergy And Infectious Diseases1996–1997
Modified Retroviral Vectors For Stable Gene ExpressionNational Institute Of Diabetes And Digestive And Kidney Diseases1994–1996
In Vivo Model Of Human Stem Cell Gene TherapyNational Institute Of Diabetes And Digestive And Kidney Diseases1994–1995
Glucocerebrosidase Gene Expression--Hematopoietic CellsNational Institute Of Diabetes And Digestive And Kidney Diseases1994
Glucocerebrosidase Gene Expression-Hematopoietic CellsNational Institute Of Diabetes And Digestive And Kidney Diseases1991–1993
Retroviral Vector-Mediated Transfer Of Nef GeneNational Institute Of Allergy And Infectious Diseases1989–1991