期刊
NATURE REVIEWS DRUG DISCOVERY
卷 10, 期 8, 页码 621-637出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nrd3459
关键词
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资金
- Great Ormond Street Children's Charity
- Medical Research Council (MRC)
- Muscular Dystrophy Campaign
- French Muscular Dystrophy Association (AFM)
- Wellcome Trust
- TREAT-NMD
- Parkinson's UK
- Action Duchenne
- Duchenne Ireland
- Muscular Dystrophy Ireland
- MRC [G0500822, G0900887, G0601943, G0502130] Funding Source: UKRI
- Medical Research Council [G0502130, G0601943, G0500822, G0900887] Funding Source: researchfish
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.
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