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LRRK2 mutations cause mitochondrial DNA damage in iPSC-derived neural cells from Parkinson's disease patients: reversal by gene correction.
Sanders LH, Laganière J, Cooper O, Mak SK, Vu BJ, Huang YA, Paschon DE, Vangipuram M, Sundararajan R, Urnov FD, Langston JW, Gregory PD, Zhang HS, Greenamyre JT, Isacson O, Schüle B. Sanders LH, et al. Among authors: urnov fd. Neurobiol Dis. 2014 Feb;62:381-6. doi: 10.1016/j.nbd.2013.10.013. Epub 2013 Oct 19. Neurobiol Dis. 2014. PMID: 24148854 Free PMC article.
Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations.
Soldner F, Laganière J, Cheng AW, Hockemeyer D, Gao Q, Alagappan R, Khurana V, Golbe LI, Myers RH, Lindquist S, Zhang L, Guschin D, Fong LK, Vu BJ, Meng X, Urnov FD, Rebar EJ, Gregory PD, Zhang HS, Jaenisch R. Soldner F, et al. Among authors: urnov fd. Cell. 2011 Jul 22;146(2):318-31. doi: 10.1016/j.cell.2011.06.019. Epub 2011 Jul 14. Cell. 2011. PMID: 21757228 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.
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.
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.
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.
DeKelver RC, Choi VM, Moehle EA, Paschon DE, Hockemeyer D, Meijsing SH, Sancak Y, Cui X, Steine EJ, Miller JC, Tam P, Bartsevich VV, Meng X, Rupniewski I, Gopalan SM, Sun HC, Pitz KJ, Rock JM, Zhang L, Davis GD, Rebar EJ, Cheeseman IM, Yamamoto KR, Sabatini DM, Jaenisch R, Gregory PD, Urnov FD. DeKelver RC, et al. Among authors: urnov fd. Genome Res. 2010 Aug;20(8):1133-42. doi: 10.1101/gr.106773.110. Epub 2010 May 27. Genome Res. 2010. PMID: 20508142 Free PMC article.
99 results