norUDCA, a Novel Therapeutic Approach to the Disposal of A1- antitrypsin Mutant Z Proteins

α1-antitrypsin deficiency (AATD) is a well-known genetic disease. No effective medical therapy is currently available for the liver disease. 24-norursodeoxycholic acid (norUDCA) has shown potent anti-cholestatic, anti-inflammatory, and anti-fibrotic properties in experimental and human cholestatic liver diseases. In this minireview, we discuss the role of exogenous norUDCA in reducing accumulation of a1-antitrypsin mutant Z proteins (AATZ) in the livers of PiZ mice and the in vitro model, HTOZ cells. In these model systems, norUDCA reduces AATZ accumulation and reduces the associated toxicity, but human trials will be needed to explore further use.