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  • Title: [Advances of treatment for Duchenne muscular dystrophy with exon skipping].
    Author: Yang J, Zhang C.
    Journal: Zhonghua Yi Xue Yi Chuan Xue Za Zhi; 2011 Aug; 28(4):406-8. PubMed ID: 21811980.
    Abstract:
    Duchenne muscular dystrophy (DMD) is a lethal muscular disorder caused by mutations in the dystrophin gene for which no mutation targeted therapy has been available so far. However, a new method named exon-skipping mediated by antisense oligonucleotides has considerable potential for DMD therapy. In this review, the principle, basic research and clinical research of exon-skipping are mainly summarized.
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