Studylow evidence
In vivo genome-editing therapy for Duchenne muscular dystrophy: Preclinical studies and first-in-human trial.
Cell·October 26, 2026·PMID 42933389
Why it mattersDuchenne muscular dystrophy remains a progressive, fatal neuromuscular disease with limited therapies. This study provides initial human proof-of-concept for in vivo genome editing to restore dystrophin, while revealing a critical immunologic safety signal that may define the therapeutic window for systemic AAV-CRISPR approaches.
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