Mutation specific drug therapy for progressive familial or benign recurrent intrahepatic cholestasis: A new tool in a near future?

Received 20 February 2010; received in revised form 18 March 2010; accepted 18 March 2010. published online 07 May 2010.

Abbreviations: PFIC, progressive familial intrahepatic cholestasis, BRIC, benign recurrent intrahepatic cholestasis, LT, liver transplantation, ER, endoplasmic reticulum, ERAD, endoplasmic reticulum associated degradation, 4-PBA, 4-phenylbutyrate, 6-ECDCA, 6-ethyl chenodeoxycholic acid

Keywords: BSEP, MDR3, FIC1, ABCB11, ABCB4, ATP8B1, PFIC, BRIC, Treatment, Nuclear receptor, Chaperone drug, Stop codon skipping

No abstract is available. To read the body of this article, please view the Full Text online.

Pediatric Hepatology and National Reference Centre for Biliary Atresia, Bicêtre Hospital, Assistance Publique – Hôpitaux de Paris, University Paris Sud 11, Paris, France

INSERM U757, University Paris Sud 11, Orsay, France

Corresponding author at: Service d’Hépatologie Pédiatrique, Hôpital Bicêtre, 78, rue du Général Leclerc, 94275 Le Kremlin-Bicêtre Cedex, France. Tel.: +33 1 45 21 31 68; fax: +33 1 45 21 28 16.

PII: S0168-8278(10)00379-X

doi:10.1016/j.jhep.2010.03.012

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