Article

Unbiased human genomic characterization of polyglutamine disorder genes to guide biological understanding and therapeutic strategies

2025-02-18

Abstract excerpt

<h4>Background</h4> Polyglutamine (polyQ) disorders, such as Huntington disease (HD) and several spinocerebellar ataxias, are severe neurological disorders caused by glutamine codon repeat expansions. These conditions lack effective treatments, with therapeutic research focused on pathogenic gene knockdown. <h4>Objectives</h4> We aimed to profile these genes using diverse human genomic data to inform therapeutic...

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Literature Corpus work: b2a7834b-fe26-5137-a06d-8bb0bb41e55b
DOI: 10.1101/2025.02.17.25322412

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