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• W2148976511 abstract "Muscular dystrophies are groups of inherited progressive diseases of the muscle caused by mutations of diverse genes related to normal muscle function. Although there is no current effective treatment for these devastating diseases, various molecular strategies have been developed to restore the expressions of the associated defective proteins. In preclinical animal models, both viral and nonviral vectors have been shown to deliver recombinant versions of defective genes. Antisense oligonucleotides have been shown to modify the splicing mechanism of mesenger ribonucleic acid to produce an internally deleted but partially functional dystrophin in an experimental model of Duchenne muscular dystrophy. In addition, chemicals can induce readthrough of the premature stop codon in nonsense mutations of the dystrophin gene. On the basis of these preclinical data, several experimental clinical trials are underway that aim to demonstrate efficacy in treating these devastating diseases." @default.
• W2148976511 created "2016-06-24" @default.
• W2148976511 creator A5045241802 @default.
• W2148976511 creator A5060245852 @default.
• W2148976511 date "2010-01-01" @default.
• W2148976511 modified "2023-09-29" @default.
• W2148976511 title "Gene Therapy for Muscular Dystrophies: Progress and Challenges" @default.
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• W2148976511 doi "https://doi.org/10.3988/jcn.2010.6.3.111" @default.
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