2012
Engineered Zinc-Finger Proteins Can Compensate Genetic Haploinsufficiency by Transcriptional Activation of the Wild-Type Allele: Application to Willams-Beuren Syndrome and Supravalvular Aortic Stenosis
Zhang P, Huang A, Morales-Ruiz M, Starcher BC, Huang Y, Sessa WC, Niklason LE, Giordano FJ. Engineered Zinc-Finger Proteins Can Compensate Genetic Haploinsufficiency by Transcriptional Activation of the Wild-Type Allele: Application to Willams-Beuren Syndrome and Supravalvular Aortic Stenosis. Human Gene Therapy 2012, 23: 1186-1199. PMID: 22891920, PMCID: PMC3498887, DOI: 10.1089/hum.2011.201.Peer-Reviewed Original ResearchMeSH KeywordsAllelesAortic Stenosis, SupravalvularCell LineCell MovementCell ProliferationDosage Compensation, GeneticElastinGene ExpressionGene Expression RegulationHaploinsufficiencyHumansMutationNonsense Mediated mRNA DecayOrgan SpecificityProtein EngineeringTranscriptional ActivationWilliams SyndromeZinc FingersConceptsZinc finger protein transcription factorsTranscriptional activationWild-type alleleWilliams-Beuren syndromeMutant allelesEngineered Zinc Finger ProteinsElastin geneTargeted transcriptional activationCompensatory expressionSplice variantsZinc finger proteinProtein transcription factorsNonsense-mediated decayWild-type cellsMultiple splice variantsElastin expressionGene replacement strategyMutant proteinsHaploinsufficient genesTranscription factorsComplex genesNatural stoichiometryDistinct genetic syndromesGenesGenetic diseases
2006
p204 Protein Overcomes the Inhibition of the Differentiation of P19 Murine Embryonal Carcinoma Cells to Beating Cardiac Myocytes by Id Proteins*
Ding B, Liu CJ, Huang Y, Yu J, Kong W, Lengyel P. p204 Protein Overcomes the Inhibition of the Differentiation of P19 Murine Embryonal Carcinoma Cells to Beating Cardiac Myocytes by Id Proteins*. Journal Of Biological Chemistry 2006, 281: 14893-14906. PMID: 16556596, DOI: 10.1074/jbc.m511748200.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsCell DifferentiationCell Line, TumorFeedback, PhysiologicalGATA4 Transcription FactorGene Expression RegulationHomeobox Protein Nkx-2.5Homeodomain ProteinsInhibitor of Differentiation Protein 1Inhibitor of Differentiation Protein 2Inhibitor of Differentiation ProteinsMiceMyocytes, CardiacNuclear ProteinsPhosphoproteinsTranscription FactorsTranscriptional ActivationConceptsD proteinsP204 proteinP19 cellsP19 murine embryonal carcinoma cellsEmbryonal carcinoma stem cellsNuclear export signalMurine embryonal carcinoma cellsP19 embryonal carcinoma stem cellsCardiac transcription factor GATA4Embryonal carcinoma cellsTranscription factor GATA4Carcinoma stem cellsExport signalSynergistic transactivationTranscription factorsNKX2.5 proteinPositive feedback loopCardiac myocytesDifferentiation proteinGATA4ProteinStem cellsDifferentiationP204Carcinoma cells