Sep 2026
Genomic splice-donor disruption rescues systemic disease in progeria
These findings establish pathogenic splice signals as genomic targets for sustained suppression of toxic isoforms while retaining canonical gene output and establish pathogenic splice signals as genomic targets for sustained suppression of toxic isoforms while retaining canonical gene output.
Wei Jin, Jing-Chun Ma, Hao Yuan et al.
· bioRxiv · 0 citations