| 发明名称 |
ADENO-ASSOCIATED VIRUS (AAV) SEROTYPE 8 SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR |
| 摘要 |
Sequences of a serotype 8 adeno-associated virus and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. |
| 申请公布号 |
US2016201088(A1) |
申请公布日期 |
2016.07.14 |
| 申请号 |
US201615084615 |
申请日期 |
2016.03.30 |
| 申请人 |
The Trustees of the University of Pennsylvania |
发明人 |
Gao Guangping;Wilsom James M.;Alvira Mauricio R. |
| 分类号 |
C12N15/86;C12N7/00;A61K38/48;C12N9/64 |
主分类号 |
C12N15/86 |
| 代理机构 |
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代理人 |
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| 主权项 |
1: An adeno-associated virus (AAV)8 viral vector comprising an AAV8 capsid having packaged therein a heterologous gene operably linked to regulatory sequences which direct its expression, wherein the heterologous gene encodes factor IX, wherein the AAV8 capsid comprises a vp3 capsid protein having the sequence of aa 204 to 738 of SEQ ID NO: 2, or a sequence which is at least 95% identical to said sequence of aa 204 to 738 of SEQ ID NO: 2. |
| 地址 |
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