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Moving toward a gene therapy for Huntington's disease.
Glorioso JC, Cohen JB, Carlisle DL, Munoz-Sanjuan I, Friedlander RM. Glorioso JC, et al. Gene Ther. 2015 Dec;22(12):931-3. doi: 10.1038/gt.2015.102. Gene Ther. 2015. PMID: 26633828 No abstract available.
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.
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.
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. Sci Transl Med. 2014 Dec 24;6(268):268ra178. doi: 10.1126/scitranslmed.3010523. Sci Transl Med. 2014. PMID: 25540325
Human glia can both induce and rescue aspects of disease phenotype in Huntington disease.
Benraiss A, Wang S, Herrlinger S, Li X, Chandler-Militello D, Mauceri J, Burm HB, Toner M, Osipovitch M, Jim Xu Q, Ding F, Wang F, Kang N, Kang J, Curtin PC, Brunner D, Windrem MS, Munoz-Sanjuan I, Nedergaard M, Goldman SA. Benraiss A, et al. Nat Commun. 2016 Jun 7;7:11758. doi: 10.1038/ncomms11758. Nat Commun. 2016. PMID: 27273432 Free PMC article.
102 results