UCL Institute of Child Health and Great Ormond Street Hospital, 30 Guildford Street, London WC1N 1EH, UK.
Nat Rev Drug Discov. 2011 Aug 1;10(8):621-37. doi: 10.1038/nrd3459.
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.
肌肉骨骼疾病的基因治疗策略
