Cell·· 23 hours agoScore69
In vivo genome-editing therapy for Duchenne muscular dystrophy: Preclinical studies and first-in-human trial.
In brief
Single-dose AAV9 delivery of hfCas12Max for in vivo genome editing reported one treatment-related death at the 1.0 × 10^14 vg/kg dose in the open-label dose-escalation Duchenne muscular dystrophy trial NCT06594094.
Source: Cell · doi.org