Fig. 4: Delandistrogene moxeparvovec micro-dystrophin expression at 12 weeks after infusion in a subset of patients.
From: AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
a, Delandistrogene moxeparvovec micro-dystrophin expression at week 12 as measured by western blot, percent normal (n = 17) and placebo (n = 14) groups in patients who had a muscle biopsy. Baseline data were not available as muscle biopsies were performed only at week 12. Each patient had two samples of biopsies taken, and all samples were analyzed. b, Representative western blots for delandistrogene moxeparvovec micro-dystrophin (left) and loading controls (right) from week 12 biopsies. Lane 1: DMD pool (negative control); Lanes 2–3: samples from placebo-treated patients; Lanes 4–5: samples from delandistrogene moxeparvovec–treated patients; Lanes 6–10: recombinant micro-dystrophin protein standard curve (21.85, 43.70, 87.39, 174.79 and 349.58 fmol mg−1). The faint upper lower molecular weight bands are non-specific. The 137-kDa band denotes the presence of delandistrogene moxeparvovec micro-dystrophin and was quantified. Each patient had two samples of biopsies taken, and all samples were analyzed.
