| 发明名称 | Adeno-associated virus factor VIII vectors | ||
| 摘要 | The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity. | ||
| 申请公布号 | US9504762(B2) | 申请公布日期 | 2016.11.29 |
| 申请号 | US201414482648 | 申请日期 | 2014.09.10 |
| 申请人 | BIOMARIN PHARMACEUTICAL INC. | 发明人 | Colosi Peter Cameron;Nathwani Amit;Mcintosh Jenny;Tuddenham Edward |
| 分类号 | A01N63/00;A01N65/00;A61K48/00;C12N7/00;C07K14/755;C12N15/86 | 主分类号 | A01N63/00 |
| 代理机构 | Marshall, Gerstein & Borun LLP | 代理人 | Marshall, Gerstein & Borun LLP |
| 主权项 | 1. An adeno-associated virus (AAV) vector, comprising an AAV2 5′ inverted terminal repeat (ITR), a liver specific transcriptional regulatory region, a functionally active FVIII coding region, a polyadenylation sequence, an AAV2 3′ ITR, and optionally one or more introns, wherein the functionally active FVIII coding region comprises nucleotides 923-5296 of SEQ ID NO: 9. | ||
| 地址 | Novato CA US | ||