Advances in gene therapy for muscular dystrophies. - PubMed - NCBI
F1000Res. 2016 Aug 18;5. pii: F1000 Faculty Rev-2030. doi: 10.12688/f1000research.8735.1. eCollection 2016.
Advances in gene therapy for muscular dystrophies.
Abdul-Razak H1, Malerba A1, Dickson G1.
Author information
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.
PMID:
27594988
PMCID:
PMC4991540
DOI:
10.12688/f1000research.8735.1
[PubMed]
Free PMC Article
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