A copy of this work was available on the public web and has been preserved in the Wayback Machine. The capture dates from 2017; you can also visit the original URL.
The file type is
Background-Familial hypercholesterolemia (FH) that results from LDL receptor (LDLR) deficiency affects Ϸ1 in 500 persons in the heterozygous state and Ϸ1 in 1 million persons in the homozygous state. We tested a novel gene therapy strategy for the treatment of FH in a mouse model. Methods and Results-We delivered the VLDL receptor (VLDLR) to the liver of LDLR-deficient mice and compared the effect of a helper-dependent adenoviral vector with all viral coding sequences deleted (HD-Ad-mVLDLR)doi:10.1161/01.cir.103.9.1274 pmid:11238273 fatcat:itnzgsw7cvg7dgfl762mus4p4y