发明名称 |
RNAI INDUCED HUNTINGTIN GENE SUPPRESSION |
摘要 |
The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model. |
申请公布号 |
WO2016102664(A1) |
申请公布日期 |
2016.06.30 |
申请号 |
WO2015EP81157 |
申请日期 |
2015.12.23 |
申请人 |
UNIQURE IP B.V. |
发明人 |
KONSTANTINOVA, PAVLINA STEFANOVA;JANA, MINIARIKOVÁ |
分类号 |
C12N15/113;A61K31/713;A61P25/14 |
主分类号 |
C12N15/113 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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