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
2008
Physiological variations of stem cell factor and stromal‐derived factor‐1 in murine models of liver injury and regeneration
Swenson ES, Kuwahara R, Krause DS, Theise ND. Physiological variations of stem cell factor and stromal‐derived factor‐1 in murine models of liver injury and regeneration. Liver International 2008, 28: 308-318. PMID: 18290773, PMCID: PMC2846401, DOI: 10.1111/j.1478-3231.2007.01659.x.Peer-Reviewed Original ResearchConceptsStromal-derived factor-1Oval cell proliferationLiver injuryLiver irradiationBile ductCell proliferationSDF-1 levelsArterial smooth muscleFactor 1Cell factorMarrow-derived progenitorsNormal mouse liverPlasma levelsBACKGROUND/Murine modelStem cell factorKupffer cellsSmooth muscleInjuryRegenerative responseOval cellsDihydrocollidineMouse liverMiceLiver progenitors