August 1, 2024

mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo

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Resumen

Huntington's disease (HD) is an autosomal dominant neurodegenerative disease caused by CAG repeat expansion in the first exon of the huntingtin gene (

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Cite This Study

Allen et al. (Thu,) studied this question.

synapsesocial.com/papers/68e5dd8fb6db6435875730f0 https://doi.org/https://doi.org/10.1089/nat.2024.0027

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1Expanded GGGGCC Hexanucleotide Repeat in Noncoding Region of C9ORF72 Causes Chromosome 9p-Linked FTD and ALS2011 · 4,971 citations
2Double setback for ASO trials in Huntington disease2021 · 116 citations
3CAG Repeat Not Polyglutamine Length Determines Timing of Huntington’s Disease Onset2019 · 562 citations
4Fully 2‘-Modified Oligonucleotide Duplexes with Improved in Vitro Potency and Stability Compared to Unmodified Small Interfering RNA2005 · 442 citations
5Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington’s disease2023 · 54 citations