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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: kwak s. Nat Med. 2019 Jul;25(7):1131-1142. doi: 10.1038/s41591-019-0478-3. Epub 2019 Jul 1. Nat Med. 2019. PMID: 31263285
Caspase-6 activity in a BACHD mouse modulates steady-state levels of mutant huntingtin protein but is not necessary for production of a 586 amino acid proteolytic fragment.
Gafni J, Papanikolaou T, Degiacomo F, Holcomb J, Chen S, Menalled L, Kudwa A, Fitzpatrick J, Miller S, Ramboz S, Tuunanen PI, Lehtimäki KK, Yang XW, Park L, Kwak S, Howland D, Park H, Ellerby LM. Gafni J, et al. Among authors: kwak s. J Neurosci. 2012 May 30;32(22):7454-65. doi: 10.1523/JNEUROSCI.6379-11.2012. J Neurosci. 2012. PMID: 22649225 Free PMC article.
Targeting ATM ameliorates mutant Huntingtin toxicity in cell and animal models of Huntington's disease.
Lu XH, Mattis VB, Wang N, Al-Ramahi I, van den Berg N, Fratantoni SA, Waldvogel H, Greiner E, Osmand A, Elzein K, Xiao J, Dijkstra S, de Pril R, Vinters HV, Faull R, Signer E, Kwak S, Marugan JJ, Botas J, Fischer DF, Svendsen CN, Munoz-Sanjuan I, Yang XW. Lu XH, et al. Among authors: kwak s. Sci Transl Med. 2014 Dec 24;6(268):268ra178. doi: 10.1126/scitranslmed.3010523. Sci Transl Med. 2014. PMID: 25540325
Characterization of HTT inclusion size, location, and timing in the zQ175 mouse model of Huntington's disease: an in vivo high-content imaging study.
Carty N, Berson N, Tillack K, Thiede C, Scholz D, Kottig K, Sedaghat Y, Gabrysiak C, Yohrling G, von der Kammer H, Ebneth A, Mack V, Munoz-Sanjuan I, Kwak S. Carty N, et al. Among authors: kwak s. PLoS One. 2015 Apr 10;10(4):e0123527. doi: 10.1371/journal.pone.0123527. eCollection 2015. PLoS One. 2015. PMID: 25859666 Free PMC article.
Integrated genomics and proteomics define huntingtin CAG length-dependent networks in mice.
Langfelder P, Cantle JP, Chatzopoulou D, Wang N, Gao F, Al-Ramahi I, Lu XH, Ramos EM, El-Zein K, Zhao Y, Deverasetty S, Tebbe A, Schaab C, Lavery DJ, Howland D, Kwak S, Botas J, Aaronson JS, Rosinski J, Coppola G, Horvath S, Yang XW. Langfelder P, et al. Among authors: kwak s. Nat Neurosci. 2016 Apr;19(4):623-33. doi: 10.1038/nn.4256. Epub 2016 Feb 22. Nat Neurosci. 2016. PMID: 26900923 Free PMC article.
Large-scale phenome analysis defines a behavioral signature for Huntington's disease genotype in mice.
Alexandrov V, Brunner D, Menalled LB, Kudwa A, Watson-Johnson J, Mazzella M, Russell I, Ruiz MC, Torello J, Sabath E, Sanchez A, Gomez M, Filipov I, Cox K, Kwan M, Ghavami A, Ramboz S, Lager B, Wheeler VC, Aaronson J, Rosinski J, Gusella JF, MacDonald ME, Howland D, Kwak S. Alexandrov V, et al. Among authors: kwak s. Nat Biotechnol. 2016 Aug;34(8):838-44. doi: 10.1038/nbt.3587. Epub 2016 Jul 4. Nat Biotechnol. 2016. PMID: 27376585
1,932 results