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Gene Therapy of Inherited Muscle Diseases  

Shin, Jin-Hong (Department of Neurology, Pusan National University Yangsan Hospital)
Publication Information
Annals of Clinical Neurophysiology / v.14, no.2, 2012 , pp. 53-58 More about this Journal
Abstract
For the last decades, molecular genetics has achieved great advances that the genes on the list of inherited muscle diseases are piling up. Those diseases of overlapping clinico-pathologic findings are now understood with discrete molecular pathogeneses. We are facing an exciting era that the long-waited gene therapy may eventually come true. Skipping of dystrophin exon 51 is on successful clinical trials, which will benefit about 13% of the children suffering from Duchenne muscular dystrophy. Exon skipping is under active investigation to expand the candidates. Hopefully it may cover majority of Duchenne muscular dystrophy mutations and some of other diseases. Adeno-associated virus is one of the most versatile tools for gene transfer. It may overcome the limitation of exon skipping. Here we review exon skipping technique of Duchenne muscular dystrophy and briefly discuss the other strategies being studied to cure inherited muscle diseases.
Keywords
Gene therapy; Muscular dystrophy; Antisense oligonucleotide; Adeno-associated virus;
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