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Peer-Reviewed Publication
Biochem Pharmacol2025;242(Pt 4):117438.December 1, 2025Journal Article

Inhibiting Cytosine-Adenine-Guanine (CAG) repeat expansions as a therapeutic strategy for Huntington's disease.

Raymond J Winquist1, Bruce Church2
1Opprtna Therapeutics Inc, United States. Electronic address: ray_winquist@opprtna.com.
2Aitia, United States.
PubMed DOI

Abstract

Huntington's Disease (HD) became the first disease mapped to a single chromosome and associated with mutations in the huntingtin (HTT) gene, specifically expansions in the trinucleotide cytosine-adenine-guanine (CAG) within exon 1. The onset and progression of disease has been linked, inversely, to the inherited CAG repeat length in the mutated allele but also to an ongoing somatic expansion. Howe…

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