Allele-specific silencing of a dominant SETX mutation in familial amyotrophic lateral sclerosis type 4
Audrey M. Winkelsas, Athena Marie Apfel, Brian Johnson, George G. Harmison, Kimberly K. Diaz Perez, Dongjun Li, Vivian G. Cheung, Christopher Grunseich
The paper demonstrates that allele-specific siRNA can effectively silence a dominant SETX mutation in ALS4, restoring R-loop levels in patient cells.
Human Genetics and Genomics Advances · Apr 8, 2025Peer-reviewedGenetics