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Ann Clin Neurophysiol > Volume 14(2); 2012 > Article
Ann Clin Neurophysiol. 2012; 14(2): 53-58.
Gene Therapy of Inherited Muscle Diseases
Jin-Hong Shin
Copyright © 2012 The Korean Society of Clinical Neurophysiology
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
For the last decades, molecular genetics has achieved great advances that the genes on the list of inherited muscle diseasesare piling up. Those diseases of overlapping clinico-pathologic findings are now understood with discrete molecularpathogeneses. We are facing an exciting era that the long-waited gene therapy may eventually come true. Skipping of dystrophinexon 51 is on successful clinical trials, which will benefit about 13% of the children suffering from Duchenne musculardystrophy. Exon skipping is under active investigation to expand the candidates. Hopefully it may cover majority ofDuchenne muscular dystrophy mutations and some of other diseases. Adeno-associated virus is one of the most versatiletools for gene transfer. It may overcome the limitation of exon skipping. Here we review exon skipping technique ofDuchenne muscular dystrophy and briefly discuss the other strategies being studied to cure inherited muscle diseases.
Key words: Gene therapy, Muscular dystrophy, Antisense oligonucleotide, Adeno-associated virus
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