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.