2016
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
Bahal R, Ali McNeer N, Quijano E, Liu Y, Sulkowski P, Turchick A, Lu YC, Bhunia DC, Manna A, Greiner DL, Brehm MA, Cheng CJ, López-Giráldez F, Ricciardi A, Beloor J, Krause DS, Kumar P, Gallagher PG, Braddock DT, Mark Saltzman W, Ly DH, Glazer PM. In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery. Nature Communications 2016, 7: 13304. PMID: 27782131, PMCID: PMC5095181, DOI: 10.1038/ncomms13304.Peer-Reviewed Original ResearchConceptsNanoparticle deliveryGene correctionReversal of splenomegalyPeptide nucleic acidLow off-target effectsVivo correctionGenome editingOff-target effectsGene editingHaematopoietic stem cellsNucleic acidsDonor DNAStem cellsΓPNAΒ-thalassaemiaNanoparticlesDeliveryEditingSCF treatmentTriplex formation
2010
SENP1-mediated GATA1 deSUMOylation is critical for definitive erythropoiesis
Yu L, Ji W, Zhang H, Renda MJ, He Y, Lin S, Cheng EC, Chen H, Krause DS, Min W. SENP1-mediated GATA1 deSUMOylation is critical for definitive erythropoiesis. Journal Of Experimental Medicine 2010, 207: 1183-1195. PMID: 20457756, PMCID: PMC2882842, DOI: 10.1084/jem.20092215.Peer-Reviewed Original ResearchConceptsSmall ubiquitin-like modifier (SUMO) modificationImportant regulatory mechanismEmbryonic day 13.5Down-regulation correlatesFetal liverCre-loxP systemEmbryonic lethalityProtein functionDefinitive erythropoiesisGene promoterDNA bindingRegulatory mechanismsGene expressionGATA1SENP1Fetal liver cellsProtein analysisDay 13.5Global deletionProteinSubsequent erythropoiesisKnockout miceErythropoiesisLiver cellsDeSUMOylation
2000
Regulation of CD34 transcription by Sp1 requires sites upstream and downstream of the transcription start site
Taranenko N, Krause D. Regulation of CD34 transcription by Sp1 requires sites upstream and downstream of the transcription start site. Experimental Hematology 2000, 28: 974-984. PMID: 10989198, DOI: 10.1016/s0301-472x(00)00492-6.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsAntigens, CD34Antigens, NuclearBinding SitesDNADNA HelicasesDNA MethylationDNA ProbesDNA-Binding ProteinsGene Expression RegulationGuanineKu AutoantigenMiceNuclear ProteinsPromoter Regions, GeneticSp1 Transcription FactorTranscription FactorsTranscription, GeneticTransfectionTumor Cells, CulturedUntranslated RegionsConceptsTranscription start sitePromoter activityCD34 promoterStart siteDrosophila S2 cellsHematopoietic cellsTranscription factor bindsDNA binding sitesElectromobility shift assaysSequence-specific mannerS2 cellsTranscriptional regulationMolecular regulationMethylation interferenceDeletion analysisEarly hematopoiesisNuclear proteinsRegulatory regionsFactor bindsShift assaysGene expressionUntranslated regionHematopoietic stemSp1CD34 transcription