2024
DOI: 10.1101/2024.07.05.602282
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In vivo CRISPR base editing for treatment of Huntington’s disease

Shraddha Shirguppe,
Michael Gapinske,
Devyani Swami
et al.

Abstract: Huntington’s disease (HD) is an inherited and ultimately fatal neurodegenerative disorder caused by an expanded polyglutamine-encoding CAG repeat within exon 1 of the huntingtin (HTT) gene, which produces a mutant protein that destroys striatal and cortical neurons. Importantly, a critical event in the pathogenesis of HD is the proteolytic cleavage of the mutant HTT protein by caspase-6, which generates fragments of the N-terminal domain of the protein that form highly toxic aggregates. Given the role that pro… Show more

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