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Updated: Jul 17, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
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SCNM1は,マウスの病気の重症度を変化させる推定RNAスプライシング因子です

David A Buchner1, Michelle Trudeau, Miriam H Meisler

  • 1Department of Human Genetics, University of Michigan School of Medicine, Ann Arbor, MI 48109-0618, USA.

Science (New York, N.Y.)
|August 16, 2003
PubMed
まとめ

遺伝的背景が遺伝的疾患を修正する. ネズミでは,SCNM1 (ナトリウムチャネル変形剤1) の突然変異が,ナトリウムチャネル遺伝子スプライシングに影響することによって,運動障害を致死性神経疾患に変換します.

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