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Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells.
Chang KH, Smith SE, Sullivan T, Chen K, Zhou Q, West JA, Liu M, Liu Y, Vieira BF, Sun C, Hong VP, Zhang M, Yang X, Reik A, Urnov FD, Rebar EJ, Holmes MC, Danos O, Jiang H, Tan S. Chang KH, et al. Among authors: urnov fd. Mol Ther Methods Clin Dev. 2017 Jan 11;4:137-148. doi: 10.1016/j.omtm.2016.12.009. eCollection 2017 Mar 17. Mol Ther Methods Clin Dev. 2017. PMID: 28344999 Free PMC article.
Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major.
Psatha N, Reik A, Phelps S, Zhou Y, Dalas D, Yannaki E, Levasseur DN, Urnov FD, Holmes MC, Papayannopoulou T. Psatha N, et al. Among authors: urnov fd. Mol Ther Methods Clin Dev. 2018 Aug 14;10:313-326. doi: 10.1016/j.omtm.2018.08.003. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30182035 Free PMC article.
Biotechnologies and therapeutics: chromatin as a target.
Reik A, Gregory PD, Urnov FD. Reik A, et al. Among authors: urnov fd. Curr Opin Genet Dev. 2002 Apr;12(2):233-42. doi: 10.1016/s0959-437x(02)00291-5. Curr Opin Genet Dev. 2002. PMID: 11893498 Review.
A TALE nuclease architecture for efficient genome editing.
Miller JC, Tan S, Qiao G, Barlow KA, Wang J, Xia DF, Meng X, Paschon DE, Leung E, Hinkley SJ, Dulay GP, Hua KL, Ankoudinova I, Cost GJ, Urnov FD, Zhang HS, Holmes MC, Zhang L, Gregory PD, Rebar EJ. Miller JC, et al. Among authors: urnov fd. Nat Biotechnol. 2011 Feb;29(2):143-8. doi: 10.1038/nbt.1755. Epub 2010 Dec 22. Nat Biotechnol. 2011. PMID: 21179091
Functional footprinting of regulatory DNA.
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. Vierstra J, et al. Among authors: urnov fd. Nat Methods. 2015 Oct;12(10):927-30. doi: 10.1038/nmeth.3554. Epub 2015 Aug 31. Nat Methods. 2015. PMID: 26322838 Free PMC article.
Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases.
Santiago Y, Chan E, Liu PQ, Orlando S, Zhang L, Urnov FD, Holmes MC, Guschin D, Waite A, Miller JC, Rebar EJ, Gregory PD, Klug A, Collingwood TN. Santiago Y, et al. Among authors: urnov fd. Proc Natl Acad Sci U S A. 2008 Apr 15;105(15):5809-14. doi: 10.1073/pnas.0800940105. Epub 2008 Mar 21. Proc Natl Acad Sci U S A. 2008. PMID: 18359850 Free PMC article.
Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease.
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. De Ravin SS, et al. Among authors: urnov fd. Nat Biotechnol. 2016 Apr;34(4):424-9. doi: 10.1038/nbt.3513. Epub 2016 Mar 7. Nat Biotechnol. 2016. PMID: 26950749 Free PMC article.
99 results