Gene Therapy Holds Hope for Cholesterol Problems

A Michigan researcher has used gene therapy in rabbits to correct an inherited disorder, characterized by high levels of cholesterol, that is a primary contributor to heart disease in humans. The finding potentially opens the door to a new era in treating hardening of the arteries and liver disease.

The rabbits have a genetic defect called familial hypercholesterolemia. They have a defective gene for a liver protein, called the LDL receptor, that is important for clearing low density lipoprotein, the so-called bad cholesterol. About one in 500 Americans has a mild form of the disorder that causes an increased risk of heart attacks and strokes.

Michigan biochemist James Wilson reported last week in the Proceedings of the National Academy of Sciences that he removed liver cells from animals with the disease, used genetic engineering techniques to insert a healthy gene for the LDL receptor, and then injected the cells into other rabbits with the disease.

The treated rabbits showed a 30% drop in blood cholesterol levels within three days. The effect persisted for only two weeks, however, because the cells were eventually rejected. Wilson now is working on ways to prevent that rejection.