Ex vivo CRISPR-Cas9 correction of LAMA2 mutations in mesoangioblasts from adults with LAMA2 muscular dystrophy

Open-label trial in congenital muscular dystrophy type 1A (MDC1A) (2 cohorts), sponsored by Maastricht University. Primary endpoint: Effect of LAMA2 mutations on skeletal muscle LAMA protein quality and quantity and in vitro analysis of genetically corrected mesoangioblasts. Started Jun 2021; status: completed.