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- W2494748207 endingPage "89" @default.
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- W2494748207 abstract "Duchenne muscular dystrophy (DMD) is an X-linked progressive degenerative muscle disorder caused by the absence of dystrophin. There is no curative therapy, although innovative therapeutic approaches have been aggressively investigated over recent years. Currently, the international clinical trial registry platform for this disease has been constructed and clinical trials for innovative therapeutic approaches are underway. Among these, exon skipping and read-through of nonsense mutations are in the most advanced stages, with exon skipping theoretically applicable to a larger number of patients. To date, exon skipping that targets exons 51, 44, 45, and 53 is being globally investigated including in USA, EU, and Japan. The latest announcement from Japan was made, demonstrating successful dystrophin production in muscles of patients with DMD after treating with exon 53 skipping antisense oligonucleotides (ASOs). However, the innovative therapeutic approaches have demonstrated limited efficacy. To address this issue in exon skipping, studies to unveil the mechanism underlying gymnotic delivery of ASO uptake in living cells have been conducted in an effort to improve in vivo delivery. Further, establishing the infrastructures to integrate multi-institutional clinical trials are needed to facilitate the development of successful therapies for DMD, which ultimately is applicable to other myopathies and neurodegenerative diseases, including spinal muscular atrophy and motor neuron diseases." @default.
- W2494748207 created "2016-08-23" @default.
- W2494748207 creator A5031552222 @default.
- W2494748207 creator A5036962501 @default.
- W2494748207 creator A5068661720 @default.
- W2494748207 creator A5071796422 @default.
- W2494748207 creator A5074650312 @default.
- W2494748207 date "2016-01-01" @default.
- W2494748207 modified "2023-10-15" @default.
- W2494748207 title "Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials." @default.
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