Precision Gene-Based Therapy for PFIC: Matching Platform to Disease Biology
Progressive familial intrahepatic cholestasis (PFIC) encompasses genetically heterogeneous disorders affecting bile transport, membrane organisation, epithelial polarity and bile-acid signalling. Current medical and surgical treatments can improve symptoms and disease course in selected patients, but substantial unmet...
Ziyu Jiang, S. Tamir Rashid, Paul Gissen et al.
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