Article
Antisense oligonucleotide-mediated disruption of HTT caspase-6 cleavage site ameliorates the phenotype of YAC128 Huntington disease mice.
Neurobiology of disease - 1 Jan 2024
Kuijper Elsa C, Overzier Maurice, Suidgeest Ernst, Dzyubachyk Oleh, Maguin Cécile, Pérot Jean-Baptiste, Flament Julien, Ariyurek Yavuz, Mei Hailiang, Buijsen Ronald A M, van der Weerd Louise, van Roon-Mom Willeke
Abstract excerpt
In Huntington disease, cellular toxicity is particularly caused by toxic protein fragments generated from the mutant huntingtin (HTT) protein. By modifying the HTT protein, we aim to reduce proteolytic cleavage and ameliorate the consequences of mutant HTT without lowering total HTT levels. To that end, we use an antisense oligonucleotide (AON) that targets HTT pre-mRNA and induces partial skipping of exon 12,...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.