Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis.

<h4>Background</h4>Familial hypercholesterolemia (FH) is an autosomal codominant disorder caused by mutations in the low-density lipoprotein receptor (LDLR) gene. Homozygous FH patients (hoFH) have severe hypercholesterolemia leading to life threatening atherosclerosis in childhood and a...

Full description

Bibliographic Details
Main Authors: Sadik H Kassim, Hui Li, Luk H Vandenberghe, Christian Hinderer, Peter Bell, Dawn Marchadier, Aisha Wilson, Debra Cromley, Valeska Redon, Hongwei Yu, James M Wilson, Daniel J Rader
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2010-10-01
Series:PLoS ONE
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/pmid/20976059/pdf/?tool=EBI