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http://dx.doi.org/10.22742/JIG.2019.1.1.1

Genetic Therapies for Duchenne Muscular Dystrophy and Beyond  

Shin, Jin-Hong (Department of Neurology, Pusan National University Yangsan Hospital, Pusan National University School of Medicine)
Publication Information
Journal of Interdisciplinary Genomics / v.1, no.1, 2019 , pp. 1-5 More about this Journal
Abstract
Progressive weakness of skeletal muscle is the hallmark of muscular dystrophies. It is often accompanied by cardiomyopathy and respiratory insufficiency. It has generally been perceived as incurable diseases, while the advent of genetic therapy is changing the paradigm. Most research and achievements have been for the treatment of Duchenne muscular dystrophy, while it is promising to hope for therapies for other myopathies. Drugs for nonsense read-through and exon skipping are already approved for clinical use in Europe and the United States, respectively. Gene therapy using adeno-associated virus is in early phase of clinical trial. In this review, most promising genetic therapies will be briefly described.
Keywords
Duchenne muscular dystrophy; Gene therapy; Exon skipping; Antisense oligo; Read-through;
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