Biallelic Mutations in the LSR Gene Cause a Novel Type of Infantile Intrahepatic Cholestasis.

Uehara, Tomoko; Yamada, Mamiko; Umetsu, Shuichiro; Nittono, Hiroshi; Suzuki, Hisato; Fujisawa, Tomoo; Takenouchi, Toshiki; Inui, Ayano et al. · J Pediatr · 2020

case_report · Level V

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Abstract

We identified biallelic pathogenic mutations in the Lipolysis-stimulated lipoprotein receptor (LSR) gene in a patient with infantile intrahepatic cholestasis. We established that mutations in the LSR gene, which encodes a protein which is critical for the formation of tricellular tight junctions in the liver, are a novel cause of pediatric cholestasis.

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