J Pharmacol Exp Ther 2020 12 26;375(3):463-468. Epub 2020 Sep 26.
Centro E. Grossi Paoletti, Dipartimento di Scienze Farmacologiche e Biomolecolari (C.P., A.O., M.T., A.S., S.S., L.C.) and Dipartimento di Scienze Farmacologiche e Biomolecolari (T.L., I.E.), Università degli Studi di Milano, Milan, Italy; Specialty Medicine Research Laboratories I, Daiichi Sankyo Co., Ltd., Tokyo, Japan (K.K., N.K.); and Medical Affairs Planning Department, Daiichi Sankyo Co., Ltd., Tokyo, Japan (K.Y.)
Lecithin:cholesterol acyltransferase (LCAT) is a unique plasma enzyme able to esterify cholesterol, and it plays an important role in HDL maturation and promotion of reverse cholesterol transport. Familial LCAT deficiency (FLD; OMIM number 245900) is a rare recessive disease that results from loss-of-function mutations in the gene and has no cure. In this study, we assessed the in vitro efficacy of a novel small-molecule LCAT activator. Read More