Journal article : Review
Therapeutic approaches for Duchenne muscular dystrophy
- Abstract:
- Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a priority candidate for molecular and cellular therapeutics. Although rare, it is the most common inherited myopathy affecting children and so has been the focus of intense research activity. It is caused by mutations that disrupt production of the dystrophin protein, and a plethora of drug development approaches are under way that aim to restore dystrophin function, including exon skipping, stop codon readthrough, gene replacement, cell therapy and gene editing. These efforts have led to the clinical approval of four exon skipping antisense oligonucleotides, one stop codon readthrough drug and one gene therapy product, with other approvals likely soon. Here, we discuss the latest therapeutic strategies that are under development and being deployed to treat DMD. Lessons from these drug development programmes are likely to have a major impact on the DMD field, but also on molecular and cellular medicine more generally. Thus, DMD is a pioneer disease at the forefront of future drug discovery efforts, with these experimental treatments paving the way for therapies using similar mechanisms of action being developed for other genetic diseases.
- Publication status:
- Published
- Peer review status:
- Peer reviewed
Actions
Access Document
- Files:
-
-
(Preview, Accepted manuscript, pdf, 768.3KB, Terms of use)
-
- Publisher copy:
- 10.1038/s41573-023-00775-6
Authors
- Publisher:
- Springer Nature
- Journal:
- Nature Reviews Drug Discovery More from this journal
- Volume:
- 22
- Issue:
- 11
- Pages:
- 917–934
- Place of publication:
- England
- Publication date:
- 2023-08-31
- Acceptance date:
- 2023-07-28
- DOI:
- EISSN:
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1474-1784
- ISSN:
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1474-1776
- Pmid:
-
37652974
- Language:
-
English
- Keywords:
- Subtype:
-
Review
- Pubs id:
-
1525847
- Local pid:
-
pubs:1525847
- Deposit date:
-
2023-09-21
Terms of use
- Copyright holder:
- Springer Nature Limited
- Copyright date:
- 2023
- Rights statement:
- © Springer Nature Limited 2023. Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.
- Notes:
- This is the accepted manuscript version of the article. The final version is available from Springer Nature at: 10.1038/s41573-023-00775-6
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