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Article

Gene Therapy for Progressive Familial Intrahepatic Cholestasis: Current Progress and Future Prospects.

International journal of molecular sciences - 29 Dec 2020

Bosma Piter J, Wits Marius, Oude-Elferink Ronald Pj

Abstract excerpt

Progressive Familial Intrahepatic Cholestasis (PFIC) are inherited severe liver disorders presenting early in life, with high serum bile salt and bilirubin levels. Six types have been reported, two of these are caused by deficiency of an ABC transporter; ABCB11 (bile salt export pump) in type 2; ABCB4 (phosphatidylcholine floppase) in type 3. In addition, ABCB11 function is affected in 3 other types of PFIC. A...

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Identifiers

PMID: 33383947
DOI: 10.3390/ijms22010273
PMCID: PMC7796371

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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