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

Full Directory Information

Research Interests

Development and advancement to the clinic of novel approaches to treat human disease using CRISPR-based genome and epigenome editing.

Fundamental mechanisms of CRISPR-based enzyme action in clinically relevant primary human cells.

Fine-resolution structure-function of the human epigenome.

Current Projects

1. Genome editing in hematopoietic stem cells as a treatment for sickle cell disease.

2. Epigenome editing as a treatment for radiation sickness.

3. Epigenome editing as a treatment for disorders of the nervous system.

4. Mechanisms and principles of engineered nuclease function in mammalian cells.

Selected Publications

Zeitler B, Froelich S, Marlen K, Shivak DA, Yu Q, Li D, Pearl JR, Miller JC, Zhang L, Paschon DE, Hinkley SJ, Ankoudinova I, Lam S, Guschin D, Kopan L, Cherone JM, Nguyen HB, Qiao G, Ataei Y, Mendel MC, Amora R, Surosky R, Laganiere  J, Vu BJ, Narayanan A, Sedaghat Y, Tillack K, Thiede C, Gärtner A, Kwak S, Bard J, Mrzljak L, Park L, Heikkinen T, Lehtimäki KK, Svedberg MM, Häggkvist J, Tari L, Tóth M, Varrone A, Halldin C, Kudwa AE, Ramboz S, Day M, Kondapalli J, Surmeier DJ, Urnov FD, Gregory PD, Rebar EJ, Muñoz-Sanjuán I, Zhang HS. Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease. Nat Med. 2019 Jul;25(7):1131-1142 PR

Lo Sardo V, Chubukov P, Ferguson W, Kumar A, Teng EL, Duran M, Zhang L, Cost G, Engler AJ, Urnov F, Topol EJ, Torkamani A, Baldwin KK. Unveiling the Role of the Most Impactful Cardiovascular Risk Locus through Haplotype Editing. Cell 2018 175(7): 1796‑1810.

Urnov FD, Ronald PC, Carroll D. A call for science‑based review of the European court's decision on gene‑edited crops. Nat Biotechnol. 2018 Sep 6;36(9):800‑802.

Psatha N, Reik A, Phelps S, Zhou Y, Dalas D, Yannaki E, Levasseur DN, Urnov FD, Holmes MC, Papayannopoulou T. Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β‑Thalassemia Major. Mol Ther Methods Clin Dev. 2018 Aug 14;10:313‑326

Urnov FD. Genome Editing BC (Before CRISPR): Lasting Lessons From the “Old Testament.” The CRISPR Journal 2018 1: 34‑46. PR

De Ravin SS, Reik A, Liu PQ, Li L, Wu X, Su L, Raley C, Theobald N, Choi U, Song AH, Chan A, Pearl JR, Paschon DE, Lee J, Newcombe H, Koontz S, Sweeney C, Shivak DA, Zarember KA, Peshwa MV, Gregory PD, Urnov FD, Malech HL. Targeted gene addition in human CD34(+) hematopoietic cells for correction of X‑linked chronic granulomatous disease. Nat Biotechnol. 2016 Apr;34(4):424‑9. PR

Lillico SG, Proudfoot C, King TJ, Tan W, Zhang L, Mardjuki R, Paschon DE, Rebar EJ, Urnov FD, Mileham AJ, McLaren DG, Whitelaw CB. Mammalian interspecies substitution of immune modulatory alleles by genome editing. Sci Rep. 2016 Feb 22;6:21645. PR

Vierstra J, Reik A, Chang KH, Stehling‑Sun S, Zhou Y, Hinkley SJ, Paschon DE, Zhang L, Psatha N, Bendana YR, O'Neil CM, Song AH, Mich AK, Liu PQ, Lee G, Bauer DE, Holmes MC, Orkin SH, Papayannopoulou T, Stamatoyannopoulos G, Rebar EJ, Gregory PD, Urnov FD, Stamatoyannopoulos JA. Functional footprinting of regulatory DNA. Nat Methods. 2015 Oct;12(10):927‑30. PR

Lanphier E, Urnov F, Haecker SE, Werner M, Smolenski J. Don't edit the human germ line. Nature. 2015 Mar 26;519(7544):410‑1

Jiang J, Jing Y, Cost GJ, Chiang JC, Kolpa HJ, Cotton AM, Carone DM, Carone BR, Shivak DA, Guschin DY, Pearl JR, Rebar EJ, Byron M, Gregory PD, Brown CJ, Urnov FD, Hall LL, Lawrence JB (2013) Translating dosage compensation to trisomy 21. Nature July 17, 2013. PR

Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD. (2010) Genome editing with engineered zinc finger nucleases. Nat Rev Genet. 11(9):636‑46. PR

DeKelver RC, Choi VM, Moehle EA, Paschon DE, Hockemeyer D, Meijsing SH, Sancak Y, Cui X, Miller JC, Tam P, Bartsevich V, Meng X, Gopalan SM, Rock JM, Zhang L, Davis GD, Rebar EJ, Jaenisch R, Cheeseman IM,, Yamamoto KR, Sabatini DM, Gregory PD, Urnov FD (2010). Functional Genomics, Proteomics, and Regulatory DNA Analysis in Isogenic Settings Using Zinc Finger Nuclease‑Driven Transgenesis Into a Safe Harbor Locus in the Human Genome. Genome Research 20(8):1133‑42. PR

Shukla, V. K., Doyon, Y, Miller, J.C., Dekelver, R. C., Moehle, E. A., Worden, S., Mitchell, J., Arnold, S., Gopalan, S., Meng, X., Choi, V., Rock, J. M., Wu, Y‑Y., Katibah, G. E., Zhifang, G., McCaskill, D., Simpson, M.A., Blakeslee, B., Greenwalt, S.A., Bultler, H.J., Hinkley, S.J., Zhang, L., Rebar, E.J., Gregory, P. D., and Urnov, F.D. (2009) Precise Genome Modification in the Crop Species Zea mays Using Zinc‑Finger Nucleases Nature 459: 437‑41. PR

Moehle EA, Rock JM, Lee YL, Jouvenot Y, DeKelver RC, Gregory PD, Urnov FD, and Holmes MC. (2007) Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases. PNAS 104: 3055‑3060. PR

Urnov FD, Miller JC, Lee YL, Beausejour CM, Rock JM, Augustus S, Jamieson AC, Porteus MH, Gregory PD, Holmes MC. (2005). Highly efficient endogenous human gene correction using designed zinc‑finger nucleases. Nature 435: 646‑651. PR

Last Updated 2019-10-01