Article

Self-inactivating AAV-CRISPR at different ages enables sustained amelioration of Huntington’s disease deficits in BAC226Q mice

2025-06-25

Abstract excerpt

Huntington’s disease (HD) is a monogenic autosomal dominant neurodegenerative disorder caused by a CAG repeat expansion in the first exon of the HTT gene, yielding a gain-of-toxic-function mutant Huntingtin protein mHTT. CRISPR/Cas9 is a potentially powerful therapeutic tool for treating HD by eliminating mutant HTT (m HTT ) gene. We developed a specific SaCas9 guide RNA to target human m HTT , and a self-inac...

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Literature Corpus work: 3f2c3fff-565f-56fc-bb1e-8b52628b4479
DOI: 10.1101/2025.06.24.661435

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