2010
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
2008
Disease-Specific Induced Pluripotent Stem Cells
Park IH, Arora N, Huo H, Maherali N, Ahfeldt T, Shimamura A, Lensch MW, Cowan C, Hochedlinger K, Daley GQ. Disease-Specific Induced Pluripotent Stem Cells. Cell 2008, 134: 877-886. PMID: 18691744, PMCID: PMC2633781, DOI: 10.1016/j.cell.2008.07.041.Peer-Reviewed Original ResearchMeSH KeywordsBone Marrow CellsCell LineFibroblastsGenetic Diseases, InbornHumansKaryotypingMesenchymal Stem CellsMutationPluripotent Stem CellsConceptsParkinson's diseaseDown syndromeBecker muscular dystrophyType 1 diabetes mellitusHuntington's diseaseStem cellsPluripotent stem cellsDiabetes mellitusDisease-SpecificLesch-Nyhan syndromeDisease-specific stem cellsDiseased patientsCarrier stateTumor cell linesDiseaseSyndromeMuscular dystrophyAdenosine deaminasePatientsDrug developmentInduced pluripotent stem cellsType IIICell linesDiamond syndromeDisease investigation