2012
Overcoming reprogramming resistance of Fanconi anemia cells
Müller LU, Milsom MD, Harris CE, Vyas R, Brumme KM, Parmar K, Moreau LA, Schambach A, Park IH, London WB, Strait K, Schlaeger T, DeVine AL, Grassman E, D'Andrea A, Daley GQ, Williams DA. Overcoming reprogramming resistance of Fanconi anemia cells. Blood 2012, 119: 5449-5457. PMID: 22371882, PMCID: PMC3369681, DOI: 10.1182/blood-2012-02-408674.Peer-Reviewed Original ResearchConceptsFA cellsFA pathwayFA DNA repair pathwayFanconi anemiaDNA double-strand breaksFanconi anemia cellsStem cellsDNA repair pathwaysDouble-strand breaksDisease-specific iPSCsPluripotent stem cellsFuture translational applicationsGenomic integrityHuman primary cellsHematopoietic stem cellsHematopoietic differentiationChromosomal instabilityMolecular characterizationGene correctionTransgenic expressionDNA damageGenetic correctionHematopoietic cellsPrimary cellsPathway
2009
Gene Targeting of a Disease-Related Gene in Human Induced Pluripotent Stem and Embryonic Stem Cells
Zou J, Maeder ML, Mali P, Pruett-Miller SM, Thibodeau-Beganny S, Chou BK, Chen G, Ye Z, Park IH, Daley GQ, Porteus MH, Joung JK, Cheng L. Gene Targeting of a Disease-Related Gene in Human Induced Pluripotent Stem and Embryonic Stem Cells. Cell Stem Cell 2009, 5: 97-110. PMID: 19540188, PMCID: PMC2720132, DOI: 10.1016/j.stem.2009.05.023.Peer-Reviewed Original ResearchConceptsHuman ES cellsZinc finger nucleasesSequence-specific double-strand breaksEngineered zinc finger nucleasesGene targetingGFP reporter geneEmbryonic stem cellsDouble-strand breaksHuman Induced Pluripotent StemSpecific genetic modificationsHuman iPS cellsES cellsFinger nucleasesTransgene expressionInduced pluripotent stemHomologous recombinationGFP geneInsertional mutagenesisReporter geneDifferent genesGenetic modificationIPS cellsDonor DNAGenesCell types