In collaboration with the University of Pennsylvania, Targeted Genetics is evaluating the effectiveness of using adeno-associated virus (AAV) to deliver genes encoding proteins that may impact the metabolism of cholesterol and other lipids and ultimately, elevate levels of the "good" cholestral to fight against lipd disorders. Approximately four million people in the United States have a genetic predisposition to lipid disorders, such as familial hypercholesterolemia, familial combined hyperlipidemia, polygenic hypercholesterolemia, and low levels of high-density lipoprotein (HDL) cholesterol.
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