Exon-skipping therapy for Duchenne muscular dystrophy.

摘要

Various therapeutic approaches such as gene therapy with viral vectors, stem-cell transplantation, or administration of pharmaceutical agents have been extensively investigated for the lethal muscle disorder Duchenne muscular dystrophy, which is caused by loss of the DMD-encoded sarcolemmal protein, dystrophin. Recently, an exon-skipping strategy using antisense oligonucleotides has drawn much attention as a treatment for this disorder. This approach is intended to recover a disrupted open reading frame and help to produce a shortened but functional dystrophin at the sarcolemma and, subsequently, a milder phenotype.