Donald Kohn
Institution Name:
University of California, Los Angeles
Total Awards:
9
Award Value:
$42,719,481
Awards as PI
Publications
- J Clin Invest 2013 beta-globin gene transfer to human bone marrow for sickle cell disease. (PubMed: 23863630)
- Cytotherapy 2017 Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease. (PubMed: 28733131)
- Stem Cells 2018 Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair. (PubMed: 30372555)
- Cell Rep 2018 Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome. (PubMed: 29847792)
- Mol Ther 2019 Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates. (PubMed: 31178391)
- Mol Ther 2018 Characterization of Gene Alterations following Editing of the beta-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells. (PubMed: 29221806)
- Nat Med 2020 Lentiviral gene therapy for X-linked chronic granulomatous disease. (PubMed: 31988463)