2011
Gene-Correction Rescues Reprogramming of Fanconi Anemia Fibroblasts and Enables Hematopoietic Differentiation of FA Induced Pluripotent Stem Cells in Vitro and In Vivo
Mueller L, Milsom M, Harris C, Vyas R, Brumme K, Parmar K, Schambach A, Grassman E, Park I, Wendy L, Strait K, Schlaeger T, Devine A, D'Andrea A, Daley G, Williams D. Gene-Correction Rescues Reprogramming of Fanconi Anemia Fibroblasts and Enables Hematopoietic Differentiation of FA Induced Pluripotent Stem Cells in Vitro and In Vivo. Blood 2011, 118: 672. DOI: 10.1182/blood.v118.21.672.672.Peer-Reviewed Original ResearchFA cellsFA pathwayFanconi anemiaPluripotent stem cellsHematopoietic differentiationGenomic instabilityStem cellsIPSC linesDouble-strand DNA breaksHematopoietic cellsFANCD2 foci formationPatient iPSC linesHematopoietic differentiation potentialFanconi anemia fibroblastsTail-tip fibroblastsInduced pluripotent stem cellsDefective DNA repairGreen fluorescent proteinFatal bone marrow failureGenomic integritySignificant chromosomal imbalancesHematopoietic stem cellsComparative genomic hybridizationComplementation groupsDNA repair
2010
Induced pluripotent stem cells: A novel frontier in the study of human primary immunodeficiencies
Pessach IM, Ordovas-Montanes J, Zhang SY, Casanova JL, Giliani S, Gennery AR, Al-Herz W, Manos PD, Schlaeger TM, Park IH, Rucci F, Agarwal S, Mostoslavsky G, Daley GQ, Notarangelo LD. Induced pluripotent stem cells: A novel frontier in the study of human primary immunodeficiencies. Journal Of Allergy And Clinical Immunology 2010, 127: 1400-1407.e4. PMID: 21185069, PMCID: PMC3081993, DOI: 10.1016/j.jaci.2010.11.008.Peer-Reviewed Original ResearchMeSH KeywordsAdaptive ImmunityCell DedifferentiationCell DifferentiationCell LineCell TransdifferentiationDNAGene ExpressionGenes, mycHumansImmunity, InnateImmunologic Deficiency SyndromesInduced Pluripotent Stem CellsKaryotypingKruppel-Like Factor 4Kruppel-Like Transcription FactorsOctamer Transcription Factor-3Proto-Oncogene MasSOXB1 Transcription FactorsConceptsInduced pluripotent stem cellsKrueppel-like factor 4Pluripotent stem cellsStem cellsIPSC linesHuman embryonic stem cellsEmbryonic stem cellsExpression of genesTranscription factor 4Patient-derived iPSC linesFactor 4Region Y-box 2Patient dermal fibroblastsTranscription factorsSomatic cellsDermal fibroblastsHuman primary immunodeficienciesEmbryoid bodiesExogenous expressionHuman diseasesGene correctionCell typesProto-oncogeneEmbryonic layersPolycistronic lentiviral vector