| 发明名称 |
RAAV-BASED COMPOSITIONS AND METHODS FOR TREATING ALPHA-1 ANTI-TRYPSIN DEFICIENCIES |
| 摘要 |
The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency). |
| 申请公布号 |
EP2699680(A2) |
申请公布日期 |
2014.02.26 |
| 申请号 |
EP20120774597 |
申请日期 |
2012.04.20 |
| 申请人 |
UNIVERSITY OF MASSACHUSETTS |
发明人 |
FLOTTE, TERENCE;MUELLER, CHRISTIAN |
| 分类号 |
C12N15/11;A61K35/76;A61K48/00;C12N7/01;C12N15/33;C12N15/67 |
主分类号 |
C12N15/11 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
|
| 地址 |
|
