A Minimal Synthetic Cleavage Module Enables Selective Degradation of Pathogenic r(CUG) Repeats in Cells
Jingsong Shan, Sandra Kovachka, Kisu Sung, Matthew David Disney
This study develops a synthetic cleavage module that selectively degrades pathogenic r(CUG) repeats in cells, reducing DM1-associated pathology without DNA damage.
bioRxiv · Nov 6, 2026 · PreprintBiochemistry