Article

Computational modeling and preclinical validation support targeting somatic instability for Huntington’s disease treatment

2026-01-07

Abstract excerpt

<h4>ABSTRACT</h4> Huntington’s disease (HD) is caused by an expanded CAG trinucleotide repeat within the huntingtin (HTT) gene. Genetic modifiers of disease onset and progression in HD implicate somatic instability (SI) of the expanded CAG repeat as a key pathogenic driver, with MSH3 emerging as a leading therapeutic target. Reducing SI, particularly in the most affected neuronal cell type, medium spiny neurons (...

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Literature Corpus work: e9db69ca-7aed-5065-885f-60ce43678c0e
DOI: 10.64898/2026.01.06.697909

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