METHODS AND COMPOSITIONS FOR GENE THERAPY FOR THE TREATMENT OF DEFECTS IN LIPOPROTEIN METABOLISM
摘要
The invention provides a recombinant viral vector comprising the DNA of, or corresponding to, at least a portion of the genome of an adenovirus, which portion is capable of infecting a hepatic cell; and a human VLDL receptor gene operatively linked to regulatory sequences directing its expression. The vector is capable of expressing the normal VLDL receptor gene product in hepatic cells in vivo or in vitro. This viral vector is useful in the treatment of metabolic disorders caused by the accumulation of LDL in plasma, such as familial hypercholesterolemia or familial combined hyperlipidemia.
申请公布号
WO9626286(A1)
申请公布日期
1996.08.29
申请号
WO1996US03041
申请日期
1996.02.23
申请人
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA;WILSON, JAMES, M.;KOZARSKY, KAREN;STRAUSS, JEROME, III
发明人
WILSON, JAMES, M.;KOZARSKY, KAREN;STRAUSS, JEROME, III