COMPOSITIONS AND METHODS FOR TREATMENT OF FAMILIAL HYPERCHOLESTEROLEMIA AND ELEVATED LOW-DENSITY LIPOPROTEIN CHOLESTEROL

摘要

Gene therapy compositions and methods are provided for targeting a very low-density lipoprotein receptor gene ( VLDLR ) and a low-density lipoprotein receptor gene ( LDLR ) to lower total cholesterol and/or low-density lipoprotein cholesterol (LDL-C) in a patient, thereby treating or mitigating familial hypercholesterolemia.