发明名称 SINGLE NUCLEOTIDE POLYMORPHISM (SNP) TARGETING THERAPIES FOR THE TREATMENT OF HUNTINGTON'S DISEASE
摘要 The present invention relates to the discovery of (SNPs) significantly associated with Huntington's disease (HD). The present invention utilizes RNA silencing technology (e.g. RNAi) against such SNPs optimally combined with select additional SNP targeting silencing agents, thereby resulting in an effective treatment of significantly-sized patient populations. Silencing agents having enhanced discriminatory properties are also featured.
申请公布号 US2012136039(A1) 申请公布日期 2012.05.31
申请号 US201013263961 申请日期 2010.04.08
申请人 ARONIN NEIL;PFISTER EDITH;ZAMORE PHILLIP D.;UNIVERSITY OF MASSACHUSETTS 发明人 ARONIN NEIL;PFISTER EDITH;ZAMORE PHILLIP D.
分类号 A61K31/713;C07H21/02 主分类号 A61K31/713
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