发明名称 |
TRANSTHYRETIN ALLELE SELECTIVE UNA OLIGOMERS FOR GENE SILENCING |
摘要 |
This invention provides UNA oligomers for selectively inhibiting V30M TTR expression, which can be used in treating amyloidosis. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis by administering a UNA oligomer to a subject. |
申请公布号 |
WO2015148582(A1) |
申请公布日期 |
2015.10.01 |
申请号 |
WO2015US22345 |
申请日期 |
2015.03.24 |
申请人 |
ARCTURUS THERAPEUTICS, INC. |
发明人 |
TACHIKAWA, KIYOSHI;PAYNE, JOSEPH E.;CHIVUKULA, PADMANABH |
分类号 |
A61K31/713 |
主分类号 |
A61K31/713 |
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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