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