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Mini Review Open Access
Volume 6 | Issue 1 | DOI: https://doi.org/10.33696/Gastroenterology.6.061

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

  • 1Department of Pediatrics, the Fifth Affiliated Hospital of Zhengzhou University, 3# Kangfuqian Street, Zhengzhou, Henan 450052, China
  • 2Department of Pediatrics, Saint Louis University School of Medicine, Cardinal Glennon Children’s Hospital, 1465 South Grand Boulevard, St. Louis, MO 63104, USA
+ Affiliations - Affiliations

Corresponding Author

Youcai Tang, tangyoucai@hotmail.com; Jeffrey H. Teckman, jeff.teckman@health.slu.edu

Received Date: July 31, 2024

Accepted Date: May 12, 2025

Abstract

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.

Keywords

NorUDCA, Autophagy, AATZ, AMPK

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