Update for Duchenne Muscular Dystrophy - Hope for Causal Treatment of a Disorder Presenting in Early Childhood?

Abstract

Duchenne muscular dystrophy (DMD) is a lethal muscle wasting disease that affects 1 in 3,600 to 6,000 males. DMD is not curable until today. In this mini-review, myoblast transplantation and stem cell therapy, read-through therapy, antisense oligonucleotide-mediated therapy, vector-mediated gene therapy, and CRISPR/Cas9-mediated gene editing are shortly addressed, approaches which alone or combined have the potential for causal treatment of DMD.