Antisense oligonucleotide-mediated upregulation of Jag1 ameliorates liver disease phenotypes in a mouse model of Alagille syndrome

作者：Lingdi Zhang, Nima Niknejad, Jian Jian Li, Lansha Peng, Surendra Jain, Derek Steiner, Rich Thai, Xing Yu, Yanfeng Wang, Xue‐hai Liang · 发表于：Molecular Therapy — Nucleic Acids · 年份：2025 · DOI：10.1016/j.omtn.2025.102694 · 被引用次数：5 · 研究领域：Pediatric Hepatobiliary Diseases and Treatments、Congenital Anomalies and Fetal Surgery、Gallbladder and Bile Duct Disorders

mice were treated with ASO, improved bile duct development was observed, along with trends of reduced plasma bile acids, bilirubin, and triglyceride levels, implying improved liver function. Together, these results suggest that ASO-induced JAG1 upregulation may provide a promising therapeutic opportunity for ALGS patients, especially when treated at an early age.