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Advances in gene therapy for muscular dystrophies [version 1; referees: 2 approved]
- Source :
- F1000Research. 5:F1000 Faculty Rev-2030
- Publication Year :
- 2016
- Publisher :
- London, UK: F1000 Research Limited, 2016.
-
Abstract
- Duchenne muscular dystrophy (DMD) is a recessive lethal inherited muscular dystrophy caused by mutations in the gene encoding dystrophin, a protein required for muscle fibre integrity. So far, many approaches have been tested from the traditional gene addition to newer advanced approaches based on manipulation of the cellular machinery either at the gene transcription, mRNA processing or translation levels. Unfortunately, despite all these efforts, no efficient treatments for DMD are currently available. In this review, we highlight the most advanced therapeutic strategies under investigation as potential DMD treatments.
Details
- ISSN :
- 20461402
- Volume :
- 5
- Database :
- F1000Research
- Journal :
- F1000Research
- Notes :
- Editorial Note on the Review Process F1000 Faculty Reviews are commissioned from members of the prestigious F1000 Faculty and are edited as a service to readers. In order to make these reviews as comprehensive and accessible as possible, the referees provide input before publication and only the final, revised version is published. The referees who approved the final version are listed with their names and affiliations but without their reports on earlier versions (any comments will already have been addressed in the published version). The referees who approved this article are: Shin'ichi Takeda, National Center of Neurology and Psychiatry, Tokyo, Japan No competing interests were disclosed. Giulio Cossu, University of Manchester, Manchester, UK No competing interests were disclosed., , [version 1; referees: 2 approved]
- Publication Type :
- Academic Journal
- Accession number :
- edsfor.10.12688.f1000research.8735.1
- Document Type :
- review
- Full Text :
- https://doi.org/10.12688/f1000research.8735.1