Building on this foundation, single AAV vectors engineered to target muscle (MyoAAV-UA) carrying a compact dCasMINI-VPR activator was used to activate endogenous full-length utrophin across multiple DMD models including patient-derived iPSC-myotubes and in non-human primates which led to increased strength without causing toxicity [157]. The gene discussed is UTRN; the disease is Duchenne muscular dystrophy.