Open access
Jul 2026
In vivo CRISPR base editing for treatment of Huntington's disease.
CRISPR base editors are developed that generate proteolysis-resistant HTT isoforms by disrupting the splice acceptor of HTT exon 13, an exon that encodes critical proteolytic cleavage sites implicated in N-terminal fragment production.
Shraddha Shirguppe, Michael Gapinske, Devyani Swami et al.
· Nature Biomedical Engineerin... · 0 citations