Volume 26 Issue 3-4, April 2019
Helper-dependent Adenovirus (HD-Ad) vector-mediated expression of a chimeric low density lipoprotein receptor (LDLR)-transferrin protein reduces aortic atherosclerosis development in a Familial Hypercholesterolemia (FH) mouse model. Gene therapy approaches for FH are of increasing interest since they may represent a definitive or long-lasting cure for patients. HD-Ad vector-mediated expression of a chimeric secreted protein, composed of the extracellular portion of the LDLR and a transferrin dimer, led to significant, long-lasting reduction of aortic atherosclerosis in LDLR-deficient mice, a model of human FH. The image shows Oil-Red-O lipid staining of aortic plaque deposits in treated and untreated LDLR-deficient mice. See paper by Leggiero et al. in this issue of Gene Therapy.
