Ontology highlight
ABSTRACT:
SUBMITTER: Vitiello L
PROVIDER: S-EPMC6929176 | biostudies-literature | 2019 Nov
REPOSITORIES: biostudies-literature
International journal of molecular sciences 20191130 23
<b>:</b> Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. The disease is caused by the lack of dystrophin, a structurally essential protein; hence, a definitive cure would necessarily have to pass through some form of gene and/or cell therapy. Cell- and genetic-based therapeutics for DMD have been explored since the 1990s and recently, two of the latter have been approved for clinical use, but their efficacy is still very low. In parallel, ther ...[more]