Methods and compositions for the treatment of defects in lipoprotein metabolism
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Karen Kozarsky, Jerome Strauss, III
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Abstract
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.
Claims10
A recombinant adenoviral vector comprising AAV 5′ and 3′ ITRs flanking a human very low density lipoprotein (VLDL) receptor minigene sequence comprising a CMV promoter operatively linked to the nucleic acid sequence encoding the human VLDL receptor.
Dependencies are inferred from the claim text.