2020
Identification of small molecules by screening a mixture-based scaffold compound library for treatment of alpha-1 antitrypsin deficiency
Zhang X, Santos R, Debevec G, Li D, Schutte R, Pham K, Liu C, Ostrov DA, Giulianotti M. Identification of small molecules by screening a mixture-based scaffold compound library for treatment of alpha-1 antitrypsin deficiency. Biochemical And Biophysical Research Communications 2020, 527: 317-323. PMID: 32446387, DOI: 10.1016/j.bbrc.2020.04.037.Peer-Reviewed Original ResearchConceptsSmall moleculesCompound librariesStructural diversityActive compoundsPreliminary structure-activity relationshipScaffold libraryIndividual active compoundsStructure-activity relationshipsMixture librariesTitration experimentsCrystal structureMolecular dockingPolymerization sitePromising scaffoldCompoundsMixture formatMoleculesPosition R1Mixture samplesScaffoldsPotential binding sitesBinding sitesDocking
2019
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 molecules