| 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 | US9493788(B2) | 申请公布日期 | 2016.11.15 |
| 申请号 | US201514598462 | 申请日期 | 2015.01.16 |
| 申请人 | The Trustees of the University of Pennsylvania | 发明人 | Gao Guangping;Wilson James M.;Alvira Mauricio R. |
| 分类号 | C12N15/63;C12N15/861;C12N15/86;C07K14/005;C07K14/755;C12N7/00;A61K35/761;A61K38/17;A61K38/45;A61K38/48;C07K14/705;C12N9/10;C12N9/64;A61K48/00 | 主分类号 | C12N15/63 |
| 代理机构 | Howson & Howson, LLP | 代理人 | Howson & Howson, LLP |
| 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 ornithine transcarbamylase (OTC), 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 | Philadelphia PA US | ||