发明名称 |
VIRUS VECTORS FOR HIGHLY EFFICIENT TRANSGENE DELIVERY |
摘要 |
The invention provides viral vector formulations and methods of uses thereof for delivery of transgenes or therapeutic nucleic acids to human subjects. The formulations include a vector and suitable amounts of empty capsids, viral genome-containing capsids, or viral capsid proteins which are optionally chemically or structurally modified and which bind to neutralizing anti- AAV antibodies thereby reducing or preventing antibody-mediated clearance of the vector, but still allowing the genome-containing (therapeutic) vector to transduce target cells and achieve therapeutic gene transfer. |
申请公布号 |
CA2856137(A1) |
申请公布日期 |
2013.05.30 |
申请号 |
CA20122856137 |
申请日期 |
2012.11.21 |
申请人 |
THE CHILDREN'S HOSPITAL OF PHILADELPHIA |
发明人 |
MINGOZZI, FEDERICO;ANGUELA, XAVIER;WRIGHT, J. FRASER;HIGH, KATHERINE A. |
分类号 |
A61K48/00;A61K35/76;C12N7/01;C12N15/86;C12N15/864;C12Q1/70 |
主分类号 |
A61K48/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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