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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. Nat Med. 2019 Jul;25(7):1131-1142. doi: 10.1038/s41591-019-0478-3. Epub 2019 Jul 1. Nat Med. 2019. PMID: 31263285
Characterization of neurophysiological and behavioral changes, MRI brain volumetry and 1H MRS in zQ175 knock-in mouse model of Huntington's disease.
Heikkinen T, Lehtimäki K, Vartiainen N, Puoliväli J, Hendricks SJ, Glaser JR, Bradaia A, Wadel K, Touller C, Kontkanen O, Yrjänheikki JM, Buisson B, Howland D, Beaumont V, Munoz-Sanjuan I, Park LC. Heikkinen T, et al. PLoS One. 2012;7(12):e50717. doi: 10.1371/journal.pone.0050717. Epub 2012 Dec 20. PLoS One. 2012. PMID: 23284644 Free PMC article.
Therapeutic Strategies for Huntington's Disease.
Mrzljak L, Munoz-Sanjuan I. Mrzljak L, et al. Curr Top Behav Neurosci. 2015;22:161-201. doi: 10.1007/7854_2013_250. Curr Top Behav Neurosci. 2015. PMID: 24277342 Review.
The PDE1/5 Inhibitor SCH-51866 Does Not Modify Disease Progression in the R6/2 Mouse Model of Huntington's Disease.
Beaumont V, Park L, Rassoulpour A, Dijkman U, Heikkinen T, Lehtimaki K, Kontkanen O, Al Nackkash R, Bates GP, Gleyzes M, Steidl E, Ramboz S, Murphy C, Beconi MG, Dominguez C, Munoz-Sanjuan I. Beaumont V, et al. PLoS Curr. 2014 Feb 13;6:ecurrents.hd.3304e87e460b4bb0dc519a29f4deccca. doi: 10.1371/currents.hd.3304e87e460b4bb0dc519a29f4deccca. PLoS Curr. 2014. PMID: 24558637 Free PMC article.
Genetic deletion of transglutaminase 2 does not rescue the phenotypic deficits observed in R6/2 and zQ175 mouse models of Huntington's disease.
Menalled LB, Kudwa AE, Oakeshott S, Farrar A, Paterson N, Filippov I, Miller S, Kwan M, Olsen M, Beltran J, Torello J, Fitzpatrick J, Mushlin R, Cox K, McConnell K, Mazzella M, He D, Osborne GF, Al-Nackkash R, Bates GP, Tuunanen P, Lehtimaki K, Brunner D, Ghavami A, Ramboz S, Park L, Macdonald D, Munoz-Sanjuan I, Howland D. Menalled LB, et al. PLoS One. 2014 Jun 23;9(6):e99520. doi: 10.1371/journal.pone.0099520. eCollection 2014. PLoS One. 2014. PMID: 24955833 Free PMC article.
102 results