Donald B. Kohn, MD
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Donald B. Kohn, MD

  • Pediatric Hematologic Malignancy


Dr. Kohn earned his B.S in Biology and M.S. in Microbiology from the University of Illinois, Champaign-Urbana, and an M.D. from the University of Wisconsin Medical School. He performed Pediatric Residency in the Department of pediatrics at the University of Wisconsin Hospitals and then a Fellowship in Pediatric Immunology in the Metabolism Branch of the National Cancer Institute at the National Institutes of Health, Bethesda MD. He began research on gene therapy for blood cell diseases during his fellowship, a research area he has continued to the present.

He was an Attending Physician in the Bone Marrow Transplantation at another Los Angeles area Children's Hospital for twenty years, where he established his research program in gene therapy for immune deficiencies and hemoglobin disorders. He relocated to UCLA in 2009, where his clinical practice is limited to in-patient service on the Pediatric Hematopoietic Stem Cell Transplantation unit at Mattel Children's Hospital in the Ronald Reagan Medical Center in Westwood. He directs a large research program performing bench-to-bedside research on gene modification of blood forming stem cells and has brought this research to multiple clinical trials. He is actively involved in training of undergraduate, graduate, medical students, as well as PhD and MD fellows.

Dr. Kohn grew up in the Chicago area, as did his wife Sheryl Handler, M.D. a pediatric ophthalmologist in private practice in Encino CA. When not working (which is most of the time) he enjoys skiing and being with their children, Lisa Kohn, M.D. a UCLA Immunology/Allergy Fellow and Scott Kohn, a programmer for the NBA2k video game.

He is a founding member of the American Society of Gene and Cell Therapy (President in 2004), where he has been involved in numerous committees. He is also a member of the American Society of Hematology, the Clinical Immunology Society (President in 2014), the International Society of Cell and Gene Therapy and has served on multiple data safety monitoring boards for gene therapy clinical trials.




Medical Board Certification

Pediatrics, American Board of Pediatrics, 1988


Allergy & Immunology, National Cancer Institute, 1987


Pediatrics, University of Wisconsin Hospitals and Clinics, 1985


Pediatrics, University of Wisconsin Hospitals and Clinics, 1983


MD, University of Wisconsin Medical School, 1982


  • Voted Super DoctorsSuper doctors, Southern California, 2024
  • Super Doctors® Southern California,  2022 - 2024
  • Lifetime Achievement Award, the Pediatric Blood & Marrow Transplant Consortium, 2018
  • University of Wisconsin Medical Alumni Association, Medical Alumni Citation Award, 2011
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Hospital Affiliations


Stem Cell Cure for "Bubble Baby" Disease (SCID), Pioneered by UCLA's Don Kohn | Nov. 2014



  • Gene Therapy Using Hematopoietic Stem Cells
  • Primary Immune Deficiency Diseases
  • Sickle Cell Disease
  • Immunotherapy for Leukemia and Cancer


Link to Dr. Kohn's PubMed publications.


  1. Shaw KL, Garabedian E, Mishra S, et al and Kohn DB. Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency. J Clin Invest. 127(5):1689-1699, 2017. PMID: 28346229
  2. Morgan RA, Gray, D, Lomova A, and Kohn DB. Hematopoietic stem cell gene therapy – progress made and lessons learned. (Perspective). Cell Stem Cell, 21(5):574-590, 2017. Review. PMID: 29100011
  3. Kuo CY, Long JD, Campo-Fernandez B, de Oliviera S, Cooper AR, Romero Z, Hoban MD, Joglekar AV, Lill G, Kaufman ML, Fitz-Gibbon S, Wang X, Hollis RP, and Kohn DB. Targeted Gene Insertion for the Treatment of X-Linked Hyper-IgM Syndrome. Cell Reports, 23(9):2606-2616, 2018.
  4. Kohn DB, Hershfield MS, Puck JM, Aiuti A, Blincoe A, Gaspar HB, Notarangelo LD and Grunebaum E. Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency. J Allergy and Clin Immunol, S0091-6749(18)31268-5, 2018. PMID: 30194989
  5. Lomova A, Clark DN, Miyahira EY, Campo-Fernandez B, Flores-Bjurstrom C, Kaufman ML, Fitz-Gibbon S, Wang X, Brown D, DeWitt MA, Corn JE, Hollis RP, Romero Z, and Kohn DB. Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair. Stem Cells. 37:284-294, 2018. PMID: 30372555
  6. Masiuk KE, Laborada J, Roncarolo MG, Hollis RP and Kohn DB. Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome. Cell Stem Cells, 24(2):309-317, 2019. PMID: 30639036
  7. Romero Z, Miggelbrink A, Lomova A, Kuo CY, Campo B, Hoban MD, Masiuk KE, Clark DN, Long J, Miyahira E, Brown D, Hollis RP, and Kohn DB. Editing the sickle cell disease mutation in primary human hematopoietic stem cells: comparison of endonucleases and homologous donor templates. Mol Ther. 27:1389-1406, 2019. PMID: 31178391
  8. 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. Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements. Mol Ther. pii: S1525-0016(19)30449-6, 2019. PMID: 31628051
  9. Kohn DB, Booth C, Kang EM et al. Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med. 26:200-206, 2020. PMID: 31988463
    Kohn DB and Kuo CY. Overview of the Current Status of Gene Therapy for Primary Immune Deficiencies (PID). JACI, In Press.


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  • Prime Health Services
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  • Super Doctors® Southern California,  2022 - 2024
  • Lifetime Achievement Award, the Pediatric Blood & Marrow Transplant Consortium, 2018
  • University of Wisconsin Medical Alumni Association, Medical Alumni Citation Award, 2011
  • USC Mellon Award for Faculty to Faculty Mentoring, 2008
  • Doris Duke Distinguished Clinical Scientist Award, 2000-2007
  • Elizabeth Glaser Scientist Award, Pediatric AIDS Foundation, 2001