Antisense oligonucleotide-mediated upregulation of Jag1 ameliorates liver disease phenotypes in a mouse model of Alagille syndrome
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Alagille syndrome (ALGS) is a rare genetic disorder that affects multiple organ systems, especially the liver, leading to life-threatening complications. In most cases, ALGS is caused by haploinsufficient mutations in the JAG1 gene, resulting in impaired liver... ...
