发明名称 Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
摘要 The present invention provides isolated adenovirus-associated viruses ("AAV"), including AAV isolates designated AAV3B and AAV6. The invention also provides nucleic acid molecules of AAV3B (SEQ ID NO: 1) or AAV6 (SEQ ID NO: 2), including DNA or RNA, and provides substantially purified polypeptides encoded by AAV3B or AAV6, as well as antibodies specific for such polypeptides. The invention also provides infectious AAV3B and AAV6 clones; AAV viral vectors, which can be hybrid AAV viral vectors; AAV vector plasmids; and AAV helper plasmids; each comprising at least a portion of an AAV3B (SEQ ID NO: 1) or AAV6 (SEQ ID NO: 2) nucleic acid molecule. The invention further provides host cells containing at least a portion of an AAV3B or AAV6 nucleic acid molecule and progeny cells derived therefrom, and provides non-human transgenic mammals having an AAV vector genome stably integrated in a chromosome. The invention also provides methods of producing a polypeptide or an RNA by expressing the polypeptide or the RNA from an AAV viral vector in a cell, and methods of treating a pathologic condition in a mammal, comprising transducing cells of the mammal with an AAV viral vector containing a heterologous nucleic acid sequence. The invention also provides AAV3B or AAV6 nucleotide sequences, which can be useful, for example, as probes for identifying the presence of AAV3B or AAV6 nucleic acids in a sample. The present invention further provides a method of identifying an AAV viral vector suitable for administration to an individual.
申请公布号 US6156303(A) 申请公布日期 2000.12.05
申请号 US19970873168 申请日期 1997.06.11
申请人 UNIVERSITY OF WASHINGTON 发明人 RUSSELL, DAVID W.;RUTLEDGE, ELIZABETH A.
分类号 A61K48/00;C07K14/015;C12N7/00;C12N15/864;(IPC1-7):A61K48/00;C12N15/64;C12N5/10 主分类号 A61K48/00
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