1. Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model.

    Nature Communications 6:7339 (2015) PMID 26081744 PMCID PMC4557302

    Diseases of lipid metabolism are a major cause of human morbidity, but no animal model entirely recapitulates human lipoprotein metabolism. Here we develop a xenograft mouse model using hepatocytes from a patient with familial hypercholesterolaemia caused by loss-of-function mutations in the low...