Therapeutic approaches for Duchenne muscular dystrophy
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...
| Main Authors: | , , |
|---|---|
| Format: | Journal article |
| Language: | English |
| Published: |
Springer Nature
2023
|
| _version_ | 1826312459504320512 |
|---|---|
| author | Roberts, TC Wood, MJA Davies, KE |
| author_facet | Roberts, TC Wood, MJA Davies, KE |
| author_sort | Roberts, TC |
| collection | OXFORD |
| description | 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. |
| first_indexed | 2024-03-07T08:29:17Z |
| format | Journal article |
| id | oxford-uuid:7ffec45d-7cef-46f5-943f-24044a222575 |
| institution | University of Oxford |
| language | English |
| last_indexed | 2024-03-07T08:29:17Z |
| publishDate | 2023 |
| publisher | Springer Nature |
| record_format | dspace |
| spelling | oxford-uuid:7ffec45d-7cef-46f5-943f-24044a2225752024-03-04T10:49:53ZTherapeutic approaches for Duchenne muscular dystrophyJournal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:7ffec45d-7cef-46f5-943f-24044a222575EnglishSymplectic ElementsSpringer Nature2023Roberts, TCWood, MJADavies, KEDuchenne 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. |
| spellingShingle | Roberts, TC Wood, MJA Davies, KE Therapeutic approaches for Duchenne muscular dystrophy |
| title | Therapeutic approaches for Duchenne muscular dystrophy |
| title_full | Therapeutic approaches for Duchenne muscular dystrophy |
| title_fullStr | Therapeutic approaches for Duchenne muscular dystrophy |
| title_full_unstemmed | Therapeutic approaches for Duchenne muscular dystrophy |
| title_short | Therapeutic approaches for Duchenne muscular dystrophy |
| title_sort | therapeutic approaches for duchenne muscular dystrophy |
| work_keys_str_mv | AT robertstc therapeuticapproachesforduchennemusculardystrophy AT woodmja therapeuticapproachesforduchennemusculardystrophy AT davieske therapeuticapproachesforduchennemusculardystrophy |