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Page 1
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: paschon de. 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.
An engineered zinc finger protein activator of the endogenous glial cell line-derived neurotrophic factor gene provides functional neuroprotection in a rat model of Parkinson's disease.
Laganiere J, Kells AP, Lai JT, Guschin D, Paschon DE, Meng X, Fong LK, Yu Q, Rebar EJ, Gregory PD, Bankiewicz KS, Forsayeth J, Zhang HS. Laganiere J, et al. Among authors: paschon de. J Neurosci. 2010 Dec 8;30(49):16469-74. doi: 10.1523/JNEUROSCI.2440-10.2010. J Neurosci. 2010. PMID: 21147986 Free PMC article.
Targeted correction and restored function of the CFTR gene in cystic fibrosis induced pluripotent stem cells.
Crane AM, Kramer P, Bui JH, Chung WJ, Li XS, Gonzalez-Garay ML, Hawkins F, Liao W, Mora D, Choi S, Wang J, Sun HC, Paschon DE, Guschin DY, Gregory PD, Kotton DN, Holmes MC, Sorscher EJ, Davis BR. Crane AM, et al. Among authors: paschon de. Stem Cell Reports. 2015 Apr 14;4(4):569-77. doi: 10.1016/j.stemcr.2015.02.005. Epub 2015 Mar 12. Stem Cell Reports. 2015. PMID: 25772471 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: paschon de. 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.
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: paschon de. 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.
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: paschon de. Nat Biotechnol. 2011 Feb;29(2):143-8. doi: 10.1038/nbt.1755. Epub 2010 Dec 22. Nat Biotechnol. 2011. PMID: 21179091
Improved specificity of TALE-based genome editing using an expanded RVD repertoire.
Miller JC, Zhang L, Xia DF, Campo JJ, Ankoudinova IV, Guschin DY, Babiarz JE, Meng X, Hinkley SJ, Lam SC, Paschon DE, Vincent AI, Dulay GP, Barlow KA, Shivak DA, Leung E, Kim JD, Amora R, Urnov FD, Gregory PD, Rebar EJ. Miller JC, et al. Among authors: paschon de. Nat Methods. 2015 May;12(5):465-71. doi: 10.1038/nmeth.3330. Epub 2015 Mar 23. Nat Methods. 2015. PMID: 25799440
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease.
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. Zeitler B, et al. Among authors: paschon de. Nat Med. 2019 Jul;25(7):1131-1142. doi: 10.1038/s41591-019-0478-3. Epub 2019 Jul 1. Nat Med. 2019. PMID: 31263285
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: paschon de. 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.
Efficient targeted gene disruption in the soma and germ line of the frog Xenopus tropicalis using engineered zinc-finger nucleases.
Young JJ, Cherone JM, Doyon Y, Ankoudinova I, Faraji FM, Lee AH, Ngo C, Guschin DY, Paschon DE, Miller JC, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Harland RM, Zeitler B. Young JJ, et al. Among authors: paschon de. Proc Natl Acad Sci U S A. 2011 Apr 26;108(17):7052-7. doi: 10.1073/pnas.1102030108. Epub 2011 Apr 6. Proc Natl Acad Sci U S A. 2011. PMID: 21471457 Free PMC article.
26 results