公开(公告)号：US06632670B1

公开(公告)日：2003-10-14

申请号：US09029705

申请日：1998-11-16

申请人： Samuel C. Wadsworth ,  Karen Vincent ,  Susan Piraino ,  Sirkka Kyostio

发明人： Samuel C. Wadsworth ,  Karen Vincent ,  Susan Piraino ,  Sirkka Kyostio

IPC分类号： C12N15864

CPC分类号： C12N7/00 ,  A01K2217/05 ,  A61K48/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14152

摘要： The present invention is directed to methods for generating high titer, contaminant free, recombinant AAV vectors, methods and genetic constructs for producing recombinant AAV vectors conveniently and in large quantities, methods for the delivery of all essential viral proteins required in trans for high yields of recombinant AAV, recombinant AAV vectors for use in gene therapy, novel packaging cell lines which obviate the need for cotransfection of vector and helper plasmids, helper plasmids and vector plasmid backbone constructs, a reporter assay for determining AAV vector yield. Further provided are recombinant AAV vectors in a pharmaceutically acceptable carrier, methods of delivering a transgene of interest to a cell, compositions and methods for delivering a DNA sequence encoding a desired polypeptide to a cell, and transgenic non-human mammals that express a human chromosome 19 AAV integration locus.

摘要翻译： 本发明涉及用于产生高效价，无污染的重组AAV载体的方法，用于方便和大量生产重组AAV载体的方法和遗传构建体，用于递送反式所需的所有必需病毒蛋白的高产率的方法 重组AAV，用于基因治疗的重组AAV载体，新型包装细胞系，其不需要共转染载体和辅助质粒，辅助质粒和载体质粒骨架构建体，用于测定AAV载体产量的报道分析。 还提供了药学上可接受的载体中的重组AAV载体，将感兴趣的转基因递送至细胞的方法，用于将编码所需多肽的DNA序列递送至细胞的组合物和方法，以及表达人染色体的转基因非人哺乳动物 19 AAV整合轨迹。