Sarepta’s RNA therapy reaches the nucleus in myotonic dystrophy models. Can it restore muscle function?
Sarepta has reported up to 80% knockdown of DMPK RNA in both the nucleus and cytoplasm of patient-derived muscle cells, alongside correction of abnormal RNA splicing in preclinical models. The findings address a central delivery challenge in myotonic dystrophy type 1, while an ongoing Phase 1/2 trial will determine whether the mechanism translates into patient