发明名称 METHODS FOR THE PRODUCTION OF CHIMERIC ADENO-ASSOCIATED VIRUS (AAV) VECTORS, COMPOSITIONS OF CHIMERIC AAV VECTORS, AND METHODS OF USE THEREOF
摘要 The present invention provides methods for making and using chimeric AAV particles having broad tissue tropism. The AAV particles may be used to deliver nucleic acid sequences encoding a desired protein wherein infection by chimeric AAV particles provides means to transduce cells with the nucleic acid sequences. Such gene transduction results in production of the desired protein in a cell, and thus establishes or restores the activity of the protein in the cell. The present invention also provides pharmaceutical compositions comprising such chimeric AAV particles for use in the therapeutic treatment of patients.
申请公布号 WO03088899(A3) 申请公布日期 2005.08.11
申请号 WO2003US10600 申请日期 2003.04.07
申请人 THE CHILDREN'S HOSPITAL OF PHILADELPHIA;XIAO, WEIDONG;HAUCK, BERND 发明人 XIAO, WEIDONG;HAUCK, BERND
分类号 A61K;A61K48/00;C12N5/06;C12N7/00;C12N15/00;C12N15/63;C12N15/861;C12N15/864 主分类号 A61K
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