シンポジウム５‐２　難治性筋疾患の病態機序‐ＣＫ発見から５０年‐治療の時代へ　モルフォリノをもちいたＤｕｃｈｅｎｎｅ型筋ジストロフィーの治療

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  • 武田 伸一
    国立精神・神経センタートランスレーショナル・メディカルセンター 国立精神・神経センター神経研究所遺伝子疾患治療研究部

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タイトル別名
  • Exon skipping therapy for Duchenne muscular dystrophy by using antisense Morpholino
  • モルフォリノをもちいたDuchenne型筋ジストロフィーの治療
  • モルフォリノ オ モチイタ Duchenneガタ キンジストロフィー ノ チリョウ

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抄録

Duchenne muscular dystrophy (DMD) is caused by the lack of dystrophin protein at the sarcolemma. Exon skipping by antisense oligonucleotides is a novel method to restore the reading frame of the mutated DMD gene, and rescue dystrophin production. We recently reported that systemic delivery of Morpholino antisense oligonucleotides targeting exon 6 and 8 of the canine DMD gene, efficiently recovered functional dystrophin proteins at the sarcolamma of dystrophic dogs, and improved performance of affected dogs without serious side effects (Yokota et al., Ann Neurol. 65 (6): 667-676, 2009). To optimize therapeutic antisense Morpholinos for more frequent mutations of the DMD gene, we designed antisense Morpholinos targeting exon 51 of the mouse DMD gene, and injected them separately or in combination into the muscles of mdx52 mice, in which exon 52 has been deleted by a gene targeting technique (Araki et al., 1997). We also tried systemic delivery of antisense Morpholino to skip exon 51 in mdx52 mice. It is important to verify the effectiveness and side effects of antisense Morpholino in experimental animal models such as dystrophic dogs or mdx52 mice, before clinical trials in DMD patients.<br>

収録刊行物

  • 臨床神経学

    臨床神経学 49 (11), 856-858, 2009

    日本神経学会

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