| 发明名称 |
COMPOSITIONS AND METHODS FOR TREATING DISEASES |
| 摘要 |
The present invention provides compositions and methods of use pertaining to rAAV-mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells. |
| 申请公布号 |
US2016369297(A1) |
申请公布日期 |
2016.12.22 |
| 申请号 |
US201614994477 |
申请日期 |
2016.01.13 |
| 申请人 |
University of Florida Research Foundation, Inc. |
发明人 |
Byrne Barry John;Falk Darin J.;Pacak Christina;Deruisseau Lara Robert;Mah Cathryn;Fuller David D. |
| 分类号 |
C12N15/86;C12N7/00;A61K9/00 |
主分类号 |
C12N15/86 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A method of improving impaired neuromuscular junction integrity, comprising administering, to a subject with impaired neuromuscular junction integrity, an effective amount of a composition comprising a rAAV 2/9 vector, wherein the rAAV2/9 vector comprises a heterologous nucleic acid molecule operably linked to a promoter, and the therapeutic composition is administered to the subject via intramuscular, intrathoracic, intraspinal, intrathecal, or intravenous injection. |
| 地址 |
Gainesville FL US |
