In vivo CRISPR base editing for treatment of Huntington's disease
Shraddha Shirguppe, Michael Gapinske, Devyani Swami, Kyrollos Shenouda, Angelo Miskalis, Nicholas C. Gosstola, Daniela Del Bosque Siller, Isabelle Guerra, Pankaj Acharya, Dana Joulani, Maddie G. Szkwarek, Ananthan Nambiar, Abhishek Bhattacharjee, Anagha S. Dangi, Norah Odle, , Pablo Pérez‐Piñera
This study uses CRISPR base editing to create HTT protein isoforms resistant to caspase-6 cleavage, reducing toxicity and neurodegeneration in a rodent HD model.
Nature Biomedical Engineering · Jul 29, 2026Peer-reviewedBioengineering