Targeting Huntingtin Expression in Patients with Huntington’s Disease
聚谷氨酰胺束
神经退行性变
作者
Sarah J. Tabrizi,Blair R. Leavitt,G. Bernhard Landwehrmeyer,Edward J. Wild,Carsten Saft,Roger A. Barker,Nick F. Blair,D Craufurd,Josef Priller,Hugh Rickards,Anne Elizabeth Rosser,Holly B. Kordasiewicz,Christian Czech,Eric E. Swayze,Daniel A. Norris,Tiffany L. Baumann,Irene Gerlach,Scott Schobel,Erika Paz,Anne Smith,C. Frank Bennett,Roger M. Lane
Abstract Background Huntington’s disease is an autosomal-dominant neurodegenerative disease caused by CAG trinucleotide repeat expansion in HTT, resulting in a mutant huntingtin protein. IONIS-HTTR...
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