Fyodor Urnov

Fyodor Urnov

Professor of Molecular Therapeutics*
*and Director for Technology and Translation at the IGI

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Research Interests

Development and advancement to the clinic of CRISPR-Cas-based genome and epigenome editing approaches for Mendelian, neurodegenerative, neuroinflammatory, and infectious disease.

Current Projects

Developing and advancing to the clinic CRISPR-Cas-based therapeutics for:

Inborn errors of immunity (supported by Danaher Corporation as part of the Danaher-IGI Beacon for CRISPR Cures)

Huntington's disease (supported by the Alliance for Therapies for Neuroscience / Roche and by the Laboratory for Genomics Research)

Adult-onset leukoencephalopathy (supported by the Weill Institute)

Virus-induced leukoencelopathy (supported by the CIRM)

ARTEMIS-deficient severe combined immune deficiency (supported by the CIRM)

Severe viral upper respiratory infections (supported by the DOE)

 

Selected Publications

1.         Kliegman M, Zaghlula M, Abrahamson S, et al. A roadmap for affordable genetic medicines. Nature. Published online July 17, 2024. doi:10.1038/s41586-024-07800-7

2.         Mackall CL, Bollard CM, Goodman N, et al. Enhancing pediatric access to cell and gene therapies. Nat Med. 2024;30(7):1836-1846. doi:10.1038/s41591-024-03035-1

3.         Ecker DJ, Aiello CD, Arron JR, et al. Opportunities and challenges for innovative and equitable healthcare. Nat Rev Drug Discov. Published online February 26, 2024. doi:10.1038/d41573-024-00032-4

4.         Wegmann S, DeVos SL, Zeitler B, et al. Persistent repression of tau in the brain using engineered zinc finger protein transcription factors. Sci Adv. 2021;7(12):eabe1611. doi:10.1126/sciadv.abe1611

5.         Urnov FD. Imagine CRISPR cures. Mol Ther. 2021;29(11):3103-3106. doi:10.1016/j.ymthe.2021.10.019

6.         Zeitler B, Froelich S, Marlen K, et al. Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease. Nat Med. 2019;25(7):1131-1142. doi:10.1038/s41591-019-0478-3

7.         Psatha N, Reik A, Phelps S, et al. Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major. Mol Ther Methods Clin Dev. 2018;10:313-326. doi:10.1016/j.omtm.2018.08.003

8.         Urnov FD. Genome Editing B.C. (Before CRISPR): Lasting Lessons from the “Old Testament.” CRISPR J. 2018;1(1):34-46. doi:10.1089/crispr.2018.29007.fyu

Last Updated 2024-09-23