Assessment of systemic delivery of rAAVrh74. MHCK7. micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial
JR Mendell, Z Sahenk, K Lehman, C Nease… - JAMA …, 2020 - jamanetwork.com
Importance Micro-dystrophin gene transfer shows promise for treating patients with
Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype
rh74 (rAAVrh74) and codon-optimized human micro-dystrophin driven by a skeletal and
cardiac muscle-specific promoter with enhanced cardiac expression (MHCK7). Objective To
identify the 1-year safety and tolerability of intravenous rAAVrh74. MHCK7. micro-dystrophin
in patients with DMD. Design, Setting, and Participants This open-label, phase 1/2a …
Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype
rh74 (rAAVrh74) and codon-optimized human micro-dystrophin driven by a skeletal and
cardiac muscle-specific promoter with enhanced cardiac expression (MHCK7). Objective To
identify the 1-year safety and tolerability of intravenous rAAVrh74. MHCK7. micro-dystrophin
in patients with DMD. Design, Setting, and Participants This open-label, phase 1/2a …
