发明授权
US08802080B2 Raav expression systems for genetic modification of specific capsid proteins
有权
用于特异性衣壳蛋白遗传修饰的Raav表达系统
- 专利标题: Raav expression systems for genetic modification of specific capsid proteins
- 专利标题(中): 用于特异性衣壳蛋白遗传修饰的Raav表达系统
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申请号: US10513059申请日: 2003-05-01
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公开(公告)号: US08802080B2公开(公告)日: 2014-08-12
- 发明人: Kenneth H. Warrington , Shaun R. Opie , Nicholas Muzyczka
- 申请人: Kenneth H. Warrington , Shaun R. Opie , Nicholas Muzyczka
- 申请人地址: US FL Gainesville
- 专利权人: University of Florida Research Foundation, Inc.
- 当前专利权人: University of Florida Research Foundation, Inc.
- 当前专利权人地址: US FL Gainesville
- 代理机构: Haynes and Boone, LLP
- 国际申请: PCT/US03/13583 WO 20030501
- 国际公布: WO2004/027019 WO 20040401
- 主分类号: C12N15/864
- IPC分类号: C12N15/864 ; C12N7/01 ; C12N5/10 ; C12N15/63 ; C07K14/015
摘要:
Disclosed are improved recombinant adeno-associated viral (rAAV) vectors having mutations in one or more capsid proteins. Exemplary vectors are provided that have altered affinity for heparin or heparin sulfate, as well as vectors, expression systems, and rAAV virions that lack functional VP2 protein expression, but are nevertheless, fully virulent. Also provided by the invention are rAAV vector-based compositions, virus particles, host cells, and pharmaceutical formulations that comprise them useful in the expression of selected therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in selected mammals, including organs, tissues, and human host cells.
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