Article

RNA-Targeting CRISPR/Cas13d System Eliminates Disease-Related Phenotypes in Pre-clinical Models of Huntington’s Disease

2022-01-24

Abstract excerpt

Huntington’s disease (HD) is a fatal, dominantly inherited neurodegenerative disorder caused by a CAG trinucleotide expansion in exon 1 of the huntingtin ( HTT ) gene. Although the pathogenesis of HD remains complex, the CAG-expanded (CAG EX ) HTT mRNA and protein ultimately causes disease through a toxic gain-of-function mechanism. As the reduction of pathogenic mutant HTT mRNA is beneficial as a treatment, w...

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Literature Corpus work: fc1867e2-4ef5-5941-9124-4e9dfb9d6959
DOI: 10.1101/2022.01.23.477417

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