Abstract
Background and Aims: Lecithin:cholesterol acyltransferase (LCAT) is the unique enzyme able to esterify cholesterol in human biological fluids and plays a major role in HDL maturation. Loss of function mutations in the LCAT gene lead to familial LCAT deficiency (FLD, OMIM #245900), a rare recessive disorder with clinical consequences and no cure. In this study the in vitro efficacy of a novel small molecule LCAT activator (DS compound) was tested.
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