In vivo CRISPR base editing for treatment of Huntington's disease
S. Shirguppe, M. Gapinske, D. Swami, N. Gosstola, P. Acharya, A. Miskalis, D. Joulani, M. G. Szkwarek, A. Bhattacharjee, G. Elias, M. Stilger, J. Winter, W. S. Woods, D. Anand, C. K. W. Lim, , P. Perez-Pinera
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.
bioRxiv · Jul 5, 2024Bioengineering