2024
Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease
Lessard S, Rimmelé P, Ling H, Moran K, Vieira B, Lin Y, Rajani G, Hong V, Reik A, Boismenu R, Hsu B, Chen M, Cockroft B, Uchida N, Tisdale J, Alavi A, Krishnamurti L, Abedi M, Galeon I, Reiner D, Wang L, Ramezi A, Rendo P, Walters M, Levasseur D, Peters R, Harris T, Hicks A. Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease. Scientific Reports 2024, 14: 24298. PMID: 39414860, PMCID: PMC11484757, DOI: 10.1038/s41598-024-74716-7.Peer-Reviewed Original ResearchConceptsHematopoietic stem cellsSickle cell diseaseTreatment of sickle cell diseaseFetal hemoglobinCell therapyReactivation of fetal hemoglobinCell diseaseMonths of follow-upStem cellsReactivate fetal hemoglobinResults of preclinical studiesPotential treatmentEngraftment in vivoAutologous cell therapyNovel cell therapiesVaso-occlusive crisisIncreased total hemoglobinErythroid progenyHealthy donorsPreclinical studiesClinical developmentFollow-upErythroid enhancerBCL11A erythroid enhancerGATAA motifs
2017
Catheter-directed Intraportal Delivery of Endothelial Cell Therapy for Liver Regeneration: A Feasibility Study in a Large-Animal Model of Cirrhosis.
Lee KS, Santagostino SF, Li D, Ramjit A, Serrano K, Ginsberg MD, Ding BS, Rafii S, Madoff DC. Catheter-directed Intraportal Delivery of Endothelial Cell Therapy for Liver Regeneration: A Feasibility Study in a Large-Animal Model of Cirrhosis. Radiology 2017, 285: 114-123. PMID: 28498793, DOI: 10.1148/radiol.2017162617.Peer-Reviewed Original ResearchConceptsEndothelial cell therapyMean proliferation indexLiver endothelial cellsCell therapyEndothelial cellsIntraportal deliveryProliferation indexPorcine modelCatheter-directed deliveryTreatment of cirrhosisInduction of cirrhosisMasson's trichrome stainingUnpaired t-testLarge animal studiesAutologous cell therapyHepatic specimensHepatobiliary injuryTranscatheter infusionHepatic arteryFluorescence-activated cellPathologic examinationHepatic fibrosisDuctal damageLiver fibrosisSurrogate marker
2013
Therapeutic Potential of Human Induced Pluripotent Stem Cells in Experimental Stroke
Chang DJ, Lee N, Park IH, Choi C, Jeon I, Kwon J, Oh SH, Shin DA, Tae J, Lee DR, Lee H, Hong K, Daley G, Song J, Moon H. Therapeutic Potential of Human Induced Pluripotent Stem Cells in Experimental Stroke. Cell Transplantation 2013, 22: 1427-1440. PMID: 23044029, DOI: 10.3727/096368912x657314.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsApoptosisBehavior, AnimalCell DifferentiationCell LineCell TrackingDisease Models, AnimalGliosisHumansInduced Pluripotent Stem CellsInfarction, Middle Cerebral ArteryInflammationMagnetic Resonance ImagingMaleMiceNeural Stem CellsNeurogenesisNeuronsRatsRats, Sprague-DawleyStem Cell TransplantationStrokeConceptsMiddle cerebral artery occlusionNeural precursor cellsNeural stem cellsStroke-induced inflammatory responseTherapeutic potentialMCAO stroke modelCerebral artery occlusionPeri-infarct areaTreatment of strokeLimited therapeutic optionsStem cellsAutologous cell therapyEndogenous neurogenesisExperimental strokePluripotent stem cellsArtery occlusionIschemic strokeBehavioral recoveryTherapeutic optionsNeurological functionInflammatory responseRobust therapeutic potentialStroke modelMRI resultsAnimal models
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