Related Experiment Video

Updated: May 1, 2026

Cell Type-specific Gene Expression Profiling in the Mouse Liver
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Cell Type-specific Gene Expression Profiling in the Mouse Liver

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Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency

Pavel Strnad1, Mattias Mandorfer1, Gourab Choudhury1

  • 1From the Department of Internal Medicine III, University Hospital, RWTH (Rheinisch-Westfälische Technische Hochschule) Aachen, Health Care Provider of the European Reference Network on Rare Liver Disorders (ERN RARE-LIVER), Aachen, Germany (P.S., C.T.); the Division of Gastroenterology and Hepatology, Department of Internal Medicine III, Medical University of Vienna, ERN RARE-LIVER, Vienna (M.M.); the Department of Respiratory Medicine, Royal Infirmary of Edinburgh University Hospital, University of Edinburgh, Edinburgh (G.C.), and the Department of Hepatology, Addenbrooke's Hospital, Cambridge University Hospitals NHS Foundation Trust, Cambridge (W.G.) - both in the United Kingdom; the Division of Gastroenterology, University of California San Diego School of Medicine, La Jolla (R.L.), and Arrowhead Pharmaceuticals, Pasadena (T.S., T.C., M.Y., B.D.G., J.C.H., J.S.M.) - both in California; and the Departments of Pediatrics and Biochemistry and Molecular Biology, Saint Louis University School of Medicine, St. Louis (J.H.T.).

The New England Journal of Medicine
|June 24, 2022
PubMed
Summary

Fazirsiran significantly reduced Z-alpha-1-antitrypsin (Z-AAT) protein accumulation in the liver for patients with AAT deficiency liver disease. This RNA interference therapy also improved liver enzymes and showed potential for fibrosis regression.

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