发明名称 |
COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF PCSK9. |
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 |
MX2008014100(A) |
申请公布日期 |
2009.02.25 |
申请号 |
MX20080014100 |
申请日期 |
2007.05.07 |
申请人 |
ISIS PHARMACEUTICAL, INC. |
发明人 |
SANJAY BHANOT;RICHARD S. GEARY;ROBERT MCKAY;BRETT P. MONIA;PUNIT P. SETH;ANDREW M. SIWKOWSKI;ERIC E. SWAYZE;EDWARD WANCEWICZ |
分类号 |
A61K31/70;C07H21/02;C07H21/04;C12N15/113;C12Q1/68 |
主分类号 |
A61K31/70 |
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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