Article

Treatment of Huntington’s disease with a pan-HTT-targeting CRISPR nuclease

2025-12-22

Abstract excerpt

<h4>ABSTRACT</h4> Huntington’s disease (HD) is an inherited neurodegenerative disorder caused by an expansion of a CAG trinucleotide repeat in the huntingtin (HTT) gene, which leads to a mutant protein that destroys neurons in the brain. Despite intense effort, there remains no approved disease-modifying therapy for HD. Here we develop a pan-HTT-targeting CRISPR-Cas9 system that, when delivered to the striatum of...

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Literature Corpus work: c71b4dfc-d500-5663-b829-9c6f3e27dc85
DOI: 10.64898/2025.12.20.695720

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