发明名称 |
Compounds and methods for modulating expression of GCCR |
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.
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申请公布号 |
US8372967(B2) |
申请公布日期 |
2013.02.12 |
申请号 |
US20070299583 |
申请日期 |
2007.05.07 |
申请人 |
ISIS PHARMACEUTICALS, INC.;BHANOT SANJAY;GEARY RICHARD;MCKAY ROBERT;MONIA BRETT P.;SETH PUNIT P.;SIWKOWSKI ANDREW M.;SWAYZE ERIC E.;WANCEWICZ EDWARD |
发明人 |
BHANOT SANJAY;GEARY RICHARD;MCKAY ROBERT;MONIA BRETT P.;SETH PUNIT P.;SIWKOWSKI ANDREW M.;SWAYZE ERIC E.;WANCEWICZ EDWARD |
分类号 |
C07H21/02;A61K31/70;C07H21/04;C12N15/113;C12Q1/68 |
主分类号 |
C07H21/02 |
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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