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ATP-binding cassette transporter A1: key player in cardiovascular and metabolic disease at local and systemic level

Van Eck, Miranda

doi: 10.1097/MOL.0000000000000088

Purpose of review ATP-binding cassette transporter A1 (ABCA1) facilitates cellular cholesterol efflux to lipid-poor apolipoprotein AI (apoAI) and plays a key role in the formation and function of HDL. This review summarizes the advances and new insights in the role of ABCA1 in cardiovascular and metabolic diseases from studies in genetically engineered mice.

Recent findings Recent studies show that low HDL associated with liver-specific deletion of ABCA1 does not affect macrophage reverse cholesterol transport or atherosclerosis susceptibility. In the intestine, ABCA1 contributes to the packaging of dietary cholesterol into HDL. Locally in the arterial wall, ABCA1 influences atherosclerosis by acting not only in bone marrow-derived cells but also in endothelial cells and smooth muscle cells. Furthermore, other than its established role in regulating insulin secretion by β-cells, evidence is provided that adipocyte-specific ABCA1 prevents fat storage and the development of impaired glucose tolerance. Moreover, new insights are provided on the post-transcriptional regulation of ABCA1 expression by microRNAs.

Summary Recent studies underscore the importance of ABCA1 in the prevention of cardiovascular and metabolic diseases. Furthermore, the discovery of the extensive regulation of ABCA1 expression by microRNAs has unraveled novel therapeutic targets for ABCA1-based strategies for the treatment of these diseases.

Division of Biopharmaceutics, Cluster BioTherapeutics, Leiden Academic Centre for Drug Research, Leiden University, The Netherlands

Correspondence to Prof Dr Miranda Van Eck, Division of Biopharmaceutics, Leiden Academic Centre for Drug Research, Gorlaeus Laboratories, Einsteinweg 55, 2333CC Leiden, The Netherlands. Tel: +31 71 5276238; fax: +31 71 5276032; e-mail:

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