Duchenne muscular dystrophy is a debilitating X-linked disease with limited treatment
options. We examined the possibility of moving forward with gene therapy, an approach that
demonstrates promise for treating Duchenne muscular dystrophy. Gene therapy is not
limited to replacement of defective genes but also includes strategies using surrogate genes
with alternative but effective means of improving cellular function or repairing gene
mutations. The first viral-mediated gene transfer for any muscle disease was carried out at …