Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism

摘要

The invention provides a recombinant viral vector comprising 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 usefuil in the treatment of metabolic disorders caused by the accumulation of LDL in plasma, such as familial hypercholesterolemia or familial combined hyperlipidemia.