公开(公告)号：US20030207445A1

公开(公告)日：2003-11-06

申请号：US10425323

申请日：2003-04-29

申请人： CELL GENESYS, INC.

发明人： Cherylene A. Schauber ,  Christopher D. Pacheco

IPC分类号： C12N015/00 ,  C12N015/63 ,  C12N015/74 ,  C07H021/04 ,  C12N015/09 ,  C12N015/70 ,  C12N015/86

CPC分类号： C12N15/86 ,  C12N2740/16043 ,  C12N2740/16045 ,  C12N2740/16052 ,  C12N2810/60 ,  C12N2810/6081

摘要： The present invention provides a retroviral gene delivery system that resists complement inactivation through the incorporation of a complement regulatory protein into retroviral particles. In particular, the present invention provides a lentiviral packaging system comprising at least two vectors: a first vector which comprises a nucleotide sequence comprising a gag, a pol, or gag and pol genes; and a second vector which comprises a nucleotide sequence comprising a gene that encodes a complement regulatory protein (CRP) and, optionally, a gene that encodes a heterologous or functionally modified envelope protein. The genes encoding a heterologous or functionally modified envelope protein and a CRP are provided either together in a second nucleotide sequence, or separately in second and third nucleotide sequences. Producer cells comprising the packaging constructs of the present invention and a transgene can be used to produce recombinant retroviral particles for transgene delivery.

摘要翻译： 本发明提供了通过将补体调节蛋白并入逆转录病毒颗粒中抵抗补体失活的逆转录病毒基因递送系统。 特别地，本发明提供了包含至少两个载体的慢病毒包装系统：第一载体，其包含包含gag，pol或gag和pol基因的核苷酸序列; 和包含编码补体调节蛋白（CRP）的基因和任选地编码异源或功能修饰的包膜蛋白的基因的核苷酸序列的第二载体。 编码异源或功能修饰的包膜蛋白和CRP的基因一起提供在第二核苷酸序列中，或分别在第二和第三核苷酸序列中提供。 包含本发明的包装构建体和转基因的生产细胞可用于产生用于转基因递送的重组逆转录病毒颗粒。