Therapeutic approaches for Duchenne muscular dystrophy. Overview of therapeutical strategies targeting the primary and secondary defects of DMD

      Аннотация: Duchenne muscular dystrophy (DMD), a severe neuromuscular disorder for which at the moment is no cure. It is caused by mutations in the DMD gene, leading to a complete absence of the dystrophin protein. Dystrophin is an important protein for stabilization of muscle cells. This books gives an overview of several strategies aiming to (partly) correct the underlying genetic defect and restoring dystrophin synthesis. Especially attention is given to antisense oligonucleotide mediated exon skipping. Furthermore various therapeutical strategies trying to alleviate the secondary symptoms are discussed.

Ключевые слова: Therapy, Duchenne Muscular Dystrophy, Genetic disorder, antisense oligonucleotides, genetic therapies