公开(公告)号：US07531647B2

公开(公告)日：2009-05-12

申请号：US12107015

申请日：2008-04-21

申请人： Jiing-Kuan Yee ,  Gilles H Michel

发明人： Jiing-Kuan Yee ,  Gilles H Michel

IPC分类号： C12N15/33 ,  C12N15/49 ,  C12N15/52 ,  C07H21/02 ,  C07H21/04 ,  A61K48/00

CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N2740/16043 ,  C12N2740/16045

摘要： Murine leukemia virus (MLV) and lentivirus vectors have been used previously to deliver genes to hematopoietic stem cells (HSCs) in human gene therapy trials. However, these vectors integrate randomly into the host genome, leading to disruption or inactivation of vital host genes. The present invention discloses a novel lentiviral vector system that overcomes this problem by integrating into a host genome in a site-specific manner.

摘要翻译： 以前已经使用鼠白血病病毒（MLV）和慢病毒载体在人类基因治疗试验中将基因递送至造血干细胞（HSC）。 然而，这些载体随机并入宿主基因组，导致重要宿主基因的破坏或失活。 本发明公开了一种通过以位点特异性方式整合入宿主基因组来克服该问题的新型慢病毒载体系统。

公开(公告)号：US20080200663A1

公开(公告)日：2008-08-21

申请号：US12107015

申请日：2008-04-21

申请人： Jiing-Kuan Yee ,  Gilles H. Michel

发明人： Jiing-Kuan Yee ,  Gilles H. Michel

IPC分类号： C07H21/04

CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N2740/16043 ,  C12N2740/16045

摘要： Murine leukemia virus (MLV) and lentivirus vectors have been used previously to deliver genes to hematopoietic stem cells (HSCs) in human gene therapy trials. However, these vectors integrate randomly into the host genome, leading to disruption or inactivation of vital host genes. The present invention discloses a novel lentiviral vector system that overcomes this problem by integrating into a host genome in a site-specific manner.

摘要翻译： 以前已经使用鼠白血病病毒（MLV）和慢病毒载体在人类基因治疗试验中将基因递送至造血干细胞（HSC）。 然而，这些载体随机并入宿主基因组，导致重要宿主基因的破坏或失活。 本发明公开了一种通过以位点特异性方式整合入宿主基因组来克服该问题的新型慢病毒载体系统。