公开(公告)号：EP3137120A4

公开(公告)日：2018-03-14

申请号：EP15785973

申请日：2015-05-01

申请人： UNIV WASHINGTON

发明人： LIEBER ANDRE ,  PAPAYANNOPOULOU THALIA ,  RICHTER MAXIMILIAN ,  SAYDAMINOVA KAMOLA

IPC分类号： A61K48/00

CPC分类号： C12N15/86 ,  A61K35/761 ,  A61K48/0058 ,  A61K48/0091 ,  C12N7/00 ,  C12N15/111 ,  C12N15/113 ,  C12N15/907 ,  C12N2310/141 ,  C12N2330/51 ,  C12N2710/10045 ,  C12N2710/10052 ,  C12N2710/10345 ,  C12N2710/10352 ,  C12N2800/24 ,  C12N2800/50 ,  C12N2800/80 ,  C12N2800/90 ,  C12N2810/6018 ,  C12N2840/007 ,  C12N2840/102 ,  C12N2999/007

摘要： The present invention provides recombinant nucleic acid expression cassetie and helper dependent adenovirus, where the expression cassettes utilize a miRNA based system for controlling expression of nucleases in helper dependent adenoviral viral producer cells, thus permitting production and use for in in vivo gene editing in CD34+ cells.

公开(公告)号：EP0797455A4

公开(公告)日：2001-11-07

申请号：EP95943818

申请日：1995-12-14

申请人： UNIV WASHINGTON

发明人： KAY MARK A ,  LIEBER ANDRE

IPC分类号： C12N15/09 ,  A61K31/70 ,  A61K35/76 ,  A61K38/46 ,  A61K38/48 ,  A61K48/00 ,  A61P1/16 ,  A61P31/12 ,  C12N7/00 ,  C12N9/72 ,  C12N15/113 ,  C12N15/58 ,  C12N15/00 ,  C12N15/52

CPC分类号： C12N15/1131 ,  A61K38/482 ,  A61K48/00 ,  C12N9/6456 ,  C12N9/6459 ,  C12N2310/121 ,  C12N2799/022 ,  C12N2799/027 ,  C12Y304/21069

摘要： A combination of retroviral and adenoviral vectors are used for high efficiency gene transfer into hepatocytes, resulting in long term gene expression. Hepatocytes are transduced in vivo with a recombinant adenovirus vector that expresses a molecule capable of inducing hepatocyte regeneration, such as urokinase plasminogen activator (uPA) or tissue plasminogen activator (tPA), resulting in a high rate of liver regeneration. During the regenerative phase, ex vivo or in vivo retroviral-mediated gene transfer into hepatocytes results in greater transduction efficiencies. The compositions and methods thus provide new means for gene therapy, and transgenic non-human animals useful in developing new therapeutic and preventative agents. The vectors can be used for high efficiency transduction of ribozymes specific for hepatitis C virus RNA.

公开(公告)号：EP2414385A4

公开(公告)日：2012-11-28

申请号：EP10764877

申请日：2010-03-31

申请人： UNIV WASHINGTON ,  COMPLIMENT CORP

发明人： LIEBER ANDRE ,  WANG HONGJIE ,  BERENSON RONALD JAY ,  CARTER DARRICK

IPC分类号： C07K14/075 ,  A61K38/16 ,  A61K39/395 ,  A61P35/00 ,  A61P37/02 ,  C07K14/725 ,  C12N15/63

CPC分类号： C07K14/005 ,  A61K38/00 ,  A61K39/39558 ,  C12N2710/10322 ,  G01N2333/4716 ,  G01N2500/04 ,  A61K2300/00