发明名称 |
COMPOUNDS AND METHODS FOR MODULATING GENE EXPRESSION |
摘要 |
<p>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.</p> |
申请公布号 |
EP2021472(B1) |
申请公布日期 |
2011.06.29 |
申请号 |
EP20070811872 |
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;A61K31/712;A61K31/7125;C07H21/04 |
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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