Donald B. Kohn

University of California, Los Angeles — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

90h-index28.6kcitations656works3.92yr avg
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Research Topics

Virus-based gene therapy research(343), CAR-T cell therapy research(183), CRISPR and Genetic Engineering(157), RNA Interference and Gene Delivery(127), Immunodeficiency and Autoimmune Disorders(97)

Publications655 total

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue
Advanced Functional Materials·2026Open Access
Outcomes Following Hematopoietic Cell Transplant in CD3δ SCID Patients in Canada
Journal of Human Immunity·2025Open Access
Clinical spectrum of Wiskott-Aldrich syndrome carriers: Self-reported survey of 193 carriers
Clinical Immunology·2025Open Access
Prime Editing for p47 <sup>phox</sup> -Deficient Chronic Granulomatous Disease
New England Journal of Medicine·2025· 1 cited
Hematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report
Blood Advances·2025Open Access
Outcomes following matched sibling donor transplantation for severe combined immunodeficiency: a report from the PIDTC
Blood Advances·2025Open Access
TriLeukeVax: A CD80/IL-15/IL-15Rα Expressing Autologous AML Cell Vaccine Elicits Robust Anti-Leukemic Cytolytic Activity
2025
Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study
Journal of Human Immunity·2025Open Access
Synthetic oligonucleotides contain synthesis errors that are propagated into the genome by editing through homology-directed repair
Blood·2025Open Access
First in human prime edited autologous hematopoietic stem cell therapy for the treatment of p47phox CGD: Initial results of PRIME-0101
Blood·2025Open Access
Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a
Blood·2025
Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial
Blood·2025
Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1
Obstetrical & Gynecological Survey·2025
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency
New England Journal of Medicine·2025· 5 cited
Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report
Transplantation and Cellular Therapy·2025Open Access
Investigation of Double-Stranded DNA Donors and CRISPR-Cas9 RNP for Universal Correction of Mutations Causing Cystic Fibrosis in Human Airway Cells
2025Open Access
Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells
Cell Reports Medicine·2025Open Access
Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia
Molecular Therapy — Methods & Clinical Development·2025· 1 citedOpen Access
Synthesis errors in oligonucleotides are propagated into the genome by homology-directed repair
2025· 1 citedOpen Access
Human cancer-targeted immunity via transgenic hematopoietic stem cell progeny
Nature Communications·2025· 1 citedOpen Access
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Frequent Co-authors

Gay M. Crooks(9), Neena Kapoor(8), Jan A. Nolta(8), Sung‐Yun Pai(7), Jennifer M. Puck(7), Morton J. Cowan(7), Theodore B. Moore(6), Luigi D. Notarangelo(6), Rebecca H. Buckley(5), Christopher C. Dvorak(5), William T. Shearer(5), Kenneth I. Weinberg(5), Brent R. Logan(4), Linda M. Griffith(4), Alfred P. Gillio(4), James A. Connelly(4), Matthew H. Porteus(4), Thomas A. Fleisher(4), Richard J. O’Reilly(4), Joseph A. Church(4)