Curr Pathobiol Rep (2017) 5:243–252 DOI 10.1007/s40139-017-0147-5 PATHOBIOLOGY OF ORPHAN DISEASES (S RANGANATHAN, SECTION EDITOR) Liver Disease in Alpha-1 Antitrypsin Deficiency: Current Approaches and Future Directions 1,2 1,2,3,4,5 Ellen L. Mitchell & Zahida Khan Published online: 10 July 2017 Springer Science+Business Media, LLC 2017 Abstract serum protein also predisposes patients to pan lobar emphy- Purpose of Review The aim of the study is to review the liver sema as adults. At this time, the only approved therapy for disease caused by alpha-1 antitrypsin deficiency (A1ATD), A1ATD-associated liver disease is orthotopic liver transplan- including pathogenesis, epidemiology, diagnostic testing, tation, which is curative. However, there has been significant and recent therapeutic developments. recent progress in the development of small molecule thera- Recent Findings Therapeutic approaches target several intra- pies with potential both to preserve the native liver and pre- cellular pathways to reduce the cytotoxic effects of the vent hepatotoxicity. misfolded mutant globular protein (ATZ) on the hepatocyte. These include promoting ATZ transport out of the endoplas- . . . Keywords PiZ mouse Autophagy ATZ Liver . . mic reticulum (ER), enhancing ATZ degradation, and transplantation SERPINA1 Bile acid preventing ATZ globule-aggregation. Summary A1ATD is the leading genetic cause of liver
Current Pathobiology Reports – Springer Journals
Published: Jul 10, 2017
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