Article

Targeting ABCB4 using mRNA-LNP for the treatment of rare liver diseases

2023-04-11

Abstract excerpt

Mutations in the ABCB4 gene lead to a wide-spectrum of rare liver diseases including progressive familial intrahepatic cholestasis type 3 (PFIC3) and low-phospholipid associated cholelithiasis (LPAC) syndrome. PFIC3 patients develop symptoms during late infancy, including severe itching, jaundice, and failure to thrive. The condition may progress to liver failure during childhood or adulthood. This is a highly unm...

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Literature Corpus work: 94a1a280-9db5-5e36-b39d-4b2181e1b34f
DOI: 10.1101/2023.04.11.535868

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