-Vector: AAV8-Payload: Micro-dystrophin (hMD1)-Promoter: Spc5.11 (skeletal and cardiac muscle)-Delivery and Dose: IV single dose; 1 × 1013 vg/kg (n = 2) or 3 × 1013 vg/kg (n = 3); sirolimus + steroids prophylaxis-Immune prophylaxis mitigates myositis risk. The gene discussed is DMD; the disease is myositis disease.