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Development of Antisense Oligonucleotide Gapmers for the Treatment of Huntington's Disease

Tejal Aslesh1,2, Toshifumi Yokota3,4

  • 1Neuroscience and Mental Health Institute, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada.

Methods in Molecular Biology (Clifton, N.J.)
|September 1, 2020
PubMed

Keywords:
2′-O-methoxyethyl (MOE)Eteplirsen (brand name Exondys 51)GapmerHTTRX (Alternative Names: HTT ASO; IONIS-HTTRx; ISIS-443139; ISIS-HTT Rx; RG 6042; RO7234292)Huntingtin (HTT)Nusinersen (brand name Spinraza)Short interfering RNA (siRNA)Trinucleotide repeat disordersWVE-120101/WVE-120102Zinc-finger proteins (ZFPs)

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