Antisense oligonucleotide-mediated disruption of HTT caspase-6 cleavage site ameliorates the phenotype of YAC128 Huntington disease mice

Antisense oligonucleotide-mediated disruption of HTT caspase-6 cleavage site ameliorates the phenotype of YAC128 Huntington disease mice

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...

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Bibliographic Details
Main Authors: Elsa C. Kuijper, Maurice Overzier, Ernst Suidgeest, Oleh Dzyubachyk, Cécile Maguin, Jean-Baptiste Pérot, Julien Flament, Yavuz Ariyurek, Hailiang Mei, Ronald A.M. Buijsen, Louise van der Weerd, Willeke van Roon-Mom
Format: Article
Language:English
Published: Elsevier 2024-01-01
Series:Neurobiology of Disease
Subjects:
Huntington disease
YAC128 mice
Antisense oligonucleotides
Caspase-6
RNA splicing
RNA sequence analysis
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996123003844

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http://www.sciencedirect.com/science/article/pii/S0969996123003844

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