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Items: 5

1.

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.

Nat Med. 2019 Jul;25(7):1131-1142. doi: 10.1038/s41591-019-0478-3. Epub 2019 Jul 1.

PMID:
31263285
2.

Antagonistic regulation of mRNA expression and splicing by CELF and MBNL proteins.

Wang ET, Ward AJ, Cherone JM, Giudice J, Wang TT, Treacy DJ, Lambert NJ, Freese P, Saxena T, Cooper TA, Burge CB.

Genome Res. 2015 Jun;25(6):858-71. doi: 10.1101/gr.184390.114. Epub 2015 Apr 16.

3.

Genetic engineering of human pluripotent cells using TALE nucleases.

Hockemeyer D, Wang H, Kiani S, Lai CS, Gao Q, Cassady JP, Cost GJ, Zhang L, Santiago Y, Miller JC, Zeitler B, Cherone JM, Meng X, Hinkley SJ, Rebar EJ, Gregory PD, Urnov FD, Jaenisch R.

Nat Biotechnol. 2011 Jul 7;29(8):731-4. doi: 10.1038/nbt.1927.

4.

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.

Proc Natl Acad Sci U S A. 2011 Apr 26;108(17):7052-7. doi: 10.1073/pnas.1102030108. Epub 2011 Apr 6.

5.

Rapid and efficient clathrin-mediated endocytosis revealed in genome-edited mammalian cells.

Doyon JB, Zeitler B, Cheng J, Cheng AT, Cherone JM, Santiago Y, Lee AH, Vo TD, Doyon Y, Miller JC, Paschon DE, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Drubin DG.

Nat Cell Biol. 2011 Mar;13(3):331-7. doi: 10.1038/ncb2175. Epub 2011 Feb 6.

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