A Novel Small Molecule Inhibits Intrahepatocellular Accumulation of Z-Variant Alpha 1-Antitrypsin In Vitro and In Vivo
Zhang X, Pham K, Li D, Schutte RJ, Gonzalo DH, Zhang P, Oshins R, Tan W, Brantly M, Liu C, Ostrov DA. A Novel Small Molecule Inhibits Intrahepatocellular Accumulation of Z-Variant Alpha 1-Antitrypsin In Vitro and In Vivo. Cells 2019, 8: 1586. PMID: 31817705, PMCID: PMC6953066, DOI: 10.3390/cells8121586.Peer-Reviewed Original ResearchConceptsLiver diseaseAssociated liver injuryChronic liver diseaseMutant alpha 1Common genetic causePiZ miceCurative therapyLiver injuryLiver fibrosisNovel small moleculesAnimal modelsAlpha 1Genetic causeDiseaseIntracellular accumulationAlphaCandidate compoundsHuman alphaMutant alphaCell modelAATDFibrosisInjuryTherapySmall moleculesTargeting the site encoded by SERPINA1*E342K for treating alpha‐1 antitrypsin deficiency‐associated liver diseases
Zhang X, Pham K, Li D, Schutte RJ, Brantly M, Liu C, Ostrov DA. Targeting the site encoded by SERPINA1*E342K for treating alpha‐1 antitrypsin deficiency‐associated liver diseases. FEBS Letters 2019, 593: 1849-1862. PMID: 31116417, DOI: 10.1002/1873-3468.13452.Peer-Reviewed Original ResearchConceptsLiver diseaseAlpha1-antitrypsin deficiencyLiver pathologyAAT deficiencyHepatocellular carcinomaClinical utilityIntracellular accumulationE342K mutationK mutation