Article

In vivo CRISPR base editing for treatment of Huntington’s disease

2024-07-07

Abstract excerpt

<h4>ABSTRACT</h4> 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 g...

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Literature Corpus work: 6518c4d1-983d-55f9-acf6-e85d326fdd0c
DOI: 10.1101/2024.07.05.602282

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