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The development of effective therapies for neuromuscular disorders such as Duchenne muscular dystrophy (DMD) is hampered by considerable challenges: skeletal muscle is the most abundant tissue in the body, and many neuromuscular disorders are multisystemic conditions. However, despite these barriers there has recently been substantial progress in the search for novel treatments. In particular, the use of antisense oligonucleotides, which are designed to target RNA and modulate pre-mRNA splicing to restore functional protein isoforms or directly inhibit the toxic effects of pathogenic RNAs, offers great promise and these approaches are now being tested in the clinic. Here, we review recent advances in the development of such antisense oligonucleotides and other promising novel approaches, including the induction of readthrough nonsense mutations.

Original publication

DOI

10.1038/nrd3459

Type

Journal article

Journal

Nat Rev Drug Discov

Publication Date

01/08/2011

Volume

10

Pages

621 - 637

Keywords

Animals, Clinical Trials as Topic, Codon, Terminator, Genetic Therapy, Humans, MicroRNAs, Muscular Atrophy, Spinal, Muscular Dystrophy, Duchenne, Myotonic Dystrophy, Oligonucleotides, Antisense, RNA, RNA Splicing