| 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 |
WO2012145624(A3) |
申请公布日期 |
2013.02.07 |
| 申请号 |
WO2012US34446 |
申请日期 |
2012.04.20 |
| 申请人 |
UNIVERSITY OF MASSACHUSETTS;FLOTTE, TERENCE;MUELLER, CHRISTIAN |
发明人 |
FLOTTE, TERENCE;MUELLER, CHRISTIAN |
| 分类号 |
C12N15/11;A61K35/76;A61K48/00;C12N7/01;C12N15/33 |
主分类号 |
C12N15/11 |
| 代理机构 |
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代理人 |
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| 主权项 |
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