发明名称 |
MODULATION OF PRE-MRNA USING SPLICE MODULATING OLIGONUCLEOTIDES AS THERAPEUTIC AGENTS IN THE TREATMENT OF DISEASE |
摘要 |
The present invention encompasses a class of compounds known as splice modulating oligonucleotides (SMOs) that modulate pre-mRNA splicing, thereby affecting expression and functionality of a specific protein in a cell. The present invention further provides compositions and methods for modulating pre-mRNA splicing using a SMO of the invention to abrogate disease-causing mutations in a protein. Accordingly, the present invention provides compositions and methods of treating a subject at risk of, susceptible to, or having a disease, disorder, or condition associated with aberrant or unwanted target pre-mRNA expression or activity. |
申请公布号 |
EP2381965(A2) |
申请公布日期 |
2011.11.02 |
申请号 |
EP20100732104 |
申请日期 |
2010.01.14 |
申请人 |
PHILADELPHIA HEALTH AND EDUCATION CORPORATION |
发明人 |
LUTZ, GORDON, J.;TALLENT, MELANIE K.;LYKENS, NICOLE MICHELE |
分类号 |
A61K48/00;C12N15/11;C12N15/113 |
主分类号 |
A61K48/00 |
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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