Contributions of Japanese patients to development of antisense therapy for DMD

Abstract Introduction Duchenne muscular dystrophy (DMD) is a fatal progressive muscle wasting disease considered untreatable since its first description in 1868. In 1987, the dystrophin gene responsible for DMD was cloned. This paved the way for the development of therapies. Antisense oligonucleotid...

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Veröffentlicht in:	Brain & development (Tokyo. 1979) 2016-01, Vol.38 (1), p.4-9
Hauptverfasser:	Matsuo, Masafumi, Takeshima, Yasuhiro, Nishio, Hisahide
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Antisense oligonucleotide Becker muscular dystrophy Child Duchenne muscular dystrophy Dystrophin - genetics Exon skipping Exons Genetic Therapy - methods Humans Japan Male Muscular Dystrophy, Duchenne - genetics Muscular Dystrophy, Duchenne - therapy Neurology Oligonucleotides, Antisense - therapeutic use Reading frame Treatment
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