| 发明名称 | SIRNA-based therapy of Fibrodyplasia Ossificans Progressiva (FOP) | ||
| 摘要 | This invention is directed to mutated Activin A type I receptor proteins (ACVR1) and isolated nucleic acids encoding same. The invention also relates to compositions and methods for siRNA-based regulation of mutated ACVR1 expression in the treatment of Fibrodysplasia Ossificans Progressiva (FOP). | ||
| 申请公布号 | US8859752(B2) | 申请公布日期 | 2014.10.14 |
| 申请号 | US201213654208 | 申请日期 | 2012.10.17 |
| 申请人 | The Trustees of the University of Pennsylvania | 发明人 | Kaplan Frederick S.;Shore Eileen M. |
| 分类号 | C12N15/11;C07H21/04;A61K31/713;C12N15/113 | 主分类号 | C12N15/11 |
| 代理机构 | Pearl Cohen Zedek Latzer Baratz LLP | 代理人 | Cohen Mark S.;Pearl Cohen Zedek Latzer Baratz LLP |
| 主权项 | 1. A method of treating Fibrodysplasia Ossificans Progressiva (FOP) in a subject, comprising the step of administering to said subject a therapeutically effective amount of a siRNA specific against a nucleic acid encoding a mutated Activin A type I receptor (ACVR1) represented by SEQ ID NO: 21 relative to a nucleic acid encoding wild-type Activin A type I receptor protein (ACVR1) as set forth in SEQ ID NO: 26, wherein said siRNA has a passenger strand selected from SEQ ID NO: 56 or SEQ ID NO: 57. | ||
| 地址 | Philadelphia PA US | ||