GeneticsHuman Genetics and Genomics AdvancesPeer-reviewed
Allele-specific silencing of a dominant SETX mutation in familial amyotrophic lateral sclerosis type 4
Audrey M. Winkelsas, Athena Marie Apfel, Brian Johnson…·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.
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