发明名称 VECTORS AND TRANSGENIES WITH REGULATORY ELEMENTS FOR GENE DELIVERY TO THE LIVER
摘要 <p>The invention is directed to novel combinations of liver specific enhancers and promoter elements for achieving persistent transgene expression in the liver. The liver specific enhancer elements may be derived from either the human serum albumin, prothrombin, α-1microglobulin or aldolase genes in single copies or in multimerized form linked to elements derived from the cytomegalovirus intermediate early (CMV), α-1-antitrypsin or albumin promoters. In a preferred embodiment of the invention, an adenoviral vector comprising a liver specific enhancer/promoter combination operably linked to a transgene is administered to recipient cells. In other embodiments of the invention, adeno-associated viral vectors, retroviral vectors, lentiviral vectors or a plasmid comprising the liver specific enhancer/promoter combination linked to a transgene is administered to recipient cells. Also within the scope of the invention are promoter elements derived from the human prothrombin gene and the β-fibrinogen gene.</p>
申请公布号 WO2001036620(A2) 申请公布日期 2001.05.25
申请号 US2000031444 申请日期 2000.11.15
申请人 发明人
分类号 主分类号
代理机构 代理人
主权项
地址