Article
ASO silencing of a glycosyltransferase, Poglut1 , improves the liver phenotypes in mouse models of Alagille syndrome.
Hepatology (Baltimore, Md.) - 1 Nov 2023
Niknejad Nima, Fox Duncan, Burwinkel Jennifer L, Zarrin-Khameh Neda, Cho Soomin, Soriano Armand, Cast Ashley E, Lopez Mario F, Huppert Kari A, Rigo Frank, Huppert Stacey S, Jafar-Nejad Paymaan, Jafar-Nejad Hamed
Abstract excerpt
BACKGROUND AND AIMS: Paucity of intrahepatic bile ducts (BDs) is caused by various etiologies and often leads to cholestatic liver disease. For example, in patients with Alagille syndrome (ALGS), which is a genetic disease primarily caused by mutations in jagged 1 ( JAG1) , BD paucity often results in severe cholestasis and liver damage. However, no mechanism-based therapy exists to restore the biliary system in...
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