Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)
Tolar J, Park IH, Xia L, Lees CJ, Peacock B, Webber B, McElmurry RT, Eide CR, Orchard PJ, Kyba M, Osborn MJ, Lund TC, Wagner JE, Daley GQ, Blazar BR. Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome). Blood 2010, 117: 839-847. PMID: 21037085, PMCID: PMC3035077, DOI: 10.1182/blood-2010-05-287607.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsBone Marrow CellsCell DifferentiationCells, CulturedChild, PreschoolDNA MethylationHEK293 CellsHematopoietic SystemHomeodomain ProteinsHumansIduronidaseInduced Pluripotent Stem CellsInfantKeratinocytesKruppel-Like Factor 4Kruppel-Like Transcription FactorsMaleMesodermMiceMucopolysaccharidosis INanog Homeobox ProteinOctamer Transcription Factor-3Promoter Regions, GeneticProto-Oncogene Proteins c-mycSOXB1 Transcription FactorsStromal CellsTransfectionConceptsHematopoietic cell transplantationMPS IHMucopolysaccharidosis type IL-iduronidaseNonhematopoietic cellsStem cellsLife-saving measureInduced pluripotent stem cellsAutologous stem cellsAutologous hematopoietic graftsType IPluripotent stem cellsAllogeneic transplantationSignificant morbidityImmunologic complicationsInsidious onsetCell transplantationHematopoietic graftsImmune reactionsAnatomical sitesCongenital deficiencyIdeal graftDonor cellsLysosomal storageKnown benefits