发明名称 |
Compounds and methods for modulating expression APOB |
摘要 |
The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing. |
申请公布号 |
EP2458006(A1) |
申请公布日期 |
2012.05.30 |
申请号 |
EP20110189591 |
申请日期 |
2007.05.07 |
申请人 |
ISIS PHARMACEUTICALS, INC. |
发明人 |
BHANOT, SANJAY;GEARY, RICHARD S.;MCKAY, ROBERT;MONIA, BRETT P.;SETH, PUNIT P.;SIWKOWSKI, ANDREW M.;SWAYZE, ERIC E.;WANCEWICZ, EDWARD |
分类号 |
C12N15/113;A61K31/7088;A61K31/712 |
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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