| 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).
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| 申请公布号 |
US2013041017(A1) |
申请公布日期 |
2013.02.14 |
| 申请号 |
US201213654208 |
申请日期 |
2012.10.17 |
| 申请人 |
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA;THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA |
发明人 |
KAPLAN FREDERICK S.;SHORE EILEEN M. |
| 分类号 |
A61K31/713;A61P19/00;C07H21/02 |
主分类号 |
A61K31/713 |
| 代理机构 |
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