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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: vu bj. Nat Med. 2019 Jul;25(7):1131-1142. doi: 10.1038/s41591-019-0478-3. Epub 2019 Jul 1. Nat Med. 2019. PMID: 31263285
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: vu bj. 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: vu bj. 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.
Hexokinase 3 enhances myeloid cell survival via non-glycolytic functions.
Seiler K, Humbert M, Minder P, Mashimo I, Schläfli AM, Krauer D, Federzoni EA, Vu B, Moresco JJ, Yates JR 3rd, Sadowski MC, Radpour R, Kaufmann T, Sarry JE, Dengjel J, Tschan MP, Torbett BE. Seiler K, et al. Cell Death Dis. 2022 May 11;13(5):448. doi: 10.1038/s41419-022-04891-w. Cell Death Dis. 2022. PMID: 35538058 Free PMC article.