[Gene therapy of hyperlipoproteinemia]

Gene therapy is an ultimate treatment of inherited diseases. Hyperlipoproteinemia is largely genetically determined. In particular, familial hypercholesterolemia (FH) is one of the most prevalent genetic diseases which result from the mutated LDL receptor. Retrovirus-mediated ex vivo gene transfer of the LDL receptor has been tested in homozygotes of FH. The outcome was far from a success in terms of the improvement of hypercholesterolemia. Experimentally, LDL receptor knockout mice have been widely used as a model of FH. Manipulation of several genes which are distinct from the LDL receptor in these animals has proven to have cholesterol-lowering effects. Further development of vector is needed to obtain practically satisfactory results in humans.