发明名称 |
Adeno-associated virus vectors encoding factor VIII and methods of using the same |
摘要 |
The present invention provides recombinant adeno-associated virus (rAAV) vectors comprising a heterologous nucleotide sequence encoding factor VIII (factor VIII). In preferred embodiments, the factor VIII is a B-domain deleted factor VIII. Also provided are methods of producing a high titer stock of the inventive rAAV/factor VIII vectors. Another aspect of the invention is a method of delivering a nucleotide sequence encoding factor VIII to a cell, preferably for subsequent administration to a subject. The present invention further provides methods of administering rAAV/factor VIII to a subject, e.g., for the treatment of hemophilia. The rAAV vector may be administered by any route, but is preferably administered to the liver.
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申请公布号 |
US2002131956(A1) |
申请公布日期 |
2002.09.19 |
申请号 |
US20020095718 |
申请日期 |
2002.03.12 |
申请人 |
THE UNIVERSITY OF NORTH CAROLINA |
发明人 |
WALSH CHRISTOPHER E.;CHAO HENGJUN;BURSTEIN HAIM;LYNCH CARMEL;STEPAN TONY;MUNSON KEITH |
分类号 |
C12N15/09;A61K35/76;A61K48/00;A61P7/04;C07K14/02;C07K14/755;C12N1/15;C12N1/19;C12N1/21;C12N5/10;C12N7/00;C12N7/01;C12N15/12;C12N15/36;C12N15/864;C12R1/93;(IPC1-7):A61K48/00;C12N15/861 |
主分类号 |
C12N15/09 |
代理机构 |
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