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对2型脊髓小脑动症的抗意义寡核酸治疗

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
概括

反感性寡核酸 (ASO) 是一种有前途的新疗法,用于治疗神经退行性疾病 - - 2型脊髓脑动症 (SCA2). 在小鼠模型中,ASO7治疗成功降低了ATXN2基因表达,改善了运动功能,并使蛋白质水平正常化.

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