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Updated: May 3, 2026

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脊髄小胞性アタキア2型に対するアンチセンセスオリゴヌクレオチド療法

Daniel R Scoles1, Pratap Meera2, Matthew D Schneider1

  • 1Department of Neurology, University of Utah, 175 North Medical Drive East, 5th Floor, Salt Lake City, Utah 84132, USA.

Nature
|April 14, 2017
PubMed
まとめ

アンチセンスオリゴヌクレオチド (ASO) は,神経変性疾患である脊髄小胞性アタキア2型 (SCA2) に対する有望な新しい治療法です. ASO7治療はATXN2遺伝子の発現を減少させ,運動機能を改善し,マウスモデルでタンパク質レベルを正常化させました.

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