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Office
4650 W Sunset Blvd
MS 62
Los Angeles, CA 90027Phone+1 323-669-4559Fax+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
- Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector Start of enrollment: 2010 Apr 01
- 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
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Publications & Presentations
PubMed
- Making Sense of Adenosine Deaminase Variants and Their Clinical Implications.Eyal Grunebaum, Robyn Loves, Donald B Kohn
The Journal of Allergy and Clinical Immunology. 2024-11-15 - High symptom burden in female X-linked chronic granulomatous disease carriers.Mary Ann Miranda, Athanasios Tsalatsanis, Jessica R Trotter, Danielle E Arnold, Jacqueline D Squire
Clinical Immunology. 2024-11-01 - 4 citationsEnhancing pediatric access to cell and gene therapies.Crystal L Mackall, Catherine M Bollard, Nancy Goodman, Casey Carr, Rebecca Gardner
Nature Medicine. 2024-07-01
Journal Articles
- T Cell Dynamics and Response of the Microbiota After Gene Therapy to Treat X-linked Severe Combined ImmunodeficiencyLuigi 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 SystemKenneth 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 Disease61st Annual American Society of Hematology Meeting - Orlando, FL - 12/7/2019
- Completing the Genome Editing Arc in Hematology60th American Society of Hematology Annual Meeting - 12/4/2018
Press Mentions
- ASH: Gene Therapy for Rare Genetic Disorder, Molecular Insights into Leukemia Growth and MoreDecember 2nd, 2024
- 'A Real Regulatory Dilemma Here': What We Heard This WeekNovember 24th, 2024
- Novel Gene Therapy Trial for Sickle Cell Disease LaunchesNovember 22nd, 2024
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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
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