Target intelligence / Profile preview

ATP-binding cassette sub-family B member 4 (ABCB4)

Target
ABCB4
Molecular classification
Transporter, ATP-binding cassette transporter (ABC transporter), P-glycoprotein family
01

Overview

ATP-binding cassette sub-family B member 4 (ABCB4, also known as multidrug resistance protein 3 or MDR3) is a membrane-associated transporter belonging to the superfamily of ATP-binding cassette proteins. It functions primarily by transporting phosphatidylcholine from hepatocytes into bile—a process essential for protecting biliary epithelium from toxic effects of unbound bile acids and maintaining proper digestion of fats. Loss-of-function mutations cause progressive familial intrahepatic cholestasis type 3 (PFIC3), low-phospholipid associated cholelithiasis syndrome, chronic liver disease, and increase susceptibility to intrahepatic cholestasis during pregnancy. The gene is also implicated in cancer biology through its downregulation in certain neoplastic conditions.

Other names
Multidrug resistance protein 3MDR3P-glycoprotein 3PFIC-3GBD1PGY3ATP-binding cassette, sub-family B (MDR/TAP), member 4ABC21
02

Mechanism of action

For drugs targeting this molecule, mechanisms would include modulation of phospholipid transport into bile to restore normal bile composition or reduce toxic effects of unbound bile acids. Inhibitors could theoretically block substrate transport; however, no approved direct inhibitors are currently noted in the search results.

03

Biological functions

Phospholipid transport (specifically phosphatidylcholine)Maintenance of bile composition and secretion in the liver
04

Disease associations

Progressive familial intrahepatic cholestasis type 3 (PFIC3)Intrahepatic cholestasis of pregnancy (ICP)Low phospholipid-associated cholelithiasis syndrome (LPAC)Chronic cholangiopathy and adult biliary fibrosis
05

Safety considerations

Mutations can lead to severe liver diseases such as PFIC type 3.Impaired function increases risk for gallstones, biliary sludge, and complications like pancreatitis.Variants can cause early-onset gallstone disease even after cholecystectomy.
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Interacting drugs

No specific drugs are directly listed in the provided results. However, as a multidrug resistance protein, it may interact with various compounds affecting bile acid or lipid metabolism. This can be supplemented from general knowledge that some bile acid sequestrants or modulators may affect its function.
07

Biomarkers

Downregulation is associated with tumorigenesis in cervical preneoplastic lesions and may serve as a prognostic marker for progression.Quantification has been proposed as a diagnostic tool for primary sclerosing cholangitis.

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