Article

Suppression of Huntington’s Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding

2024-11-04

Abstract excerpt

Huntington’s disease (HD) arises from a CAG expansion in the huntingtin ( HTT ) gene beyond a critical threshold. A major thrust of current HD therapeutic development is lowering levels of mutant HTT mRNA (m HTT ) and protein (mHTT) with the aim of reducing the toxicity of these product(s). Human genetic data also support a key role for somatic instability (SI) in HTT ’s CAG repeat – whereby it lengthens with...

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Literature Corpus work: 11ec9c4a-a313-5291-ad8e-6f32a843ec60
DOI: 10.1101/2024.11.04.619693

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