Modelling Fanconi anemia pathogenesis and therapeutics using integration-free patient-derived iPSCs.Fanconi anaemia (FA) is a recessive disorder characterized by genomic instability, congenital abnormalities, cancer predisposition and bone marrow (BM) failure.  Here, we derive integration free-induced pluripotent stem cells (iPSCs) from an FA patient without genetic complementation and report in situ gene correction in FA-iPSCs as well as the generation of isogenic FANCA-deficient human embryonic stem cell (ESC) lines.  FA cellular phenotypes are recapitulated in iPSCs/ESCs and their adult stem/progenitor cell derivatives.  We validate our model as a drug-screening platform by identifying several compounds that improve hematopoietic differentiation of FA-iPSCs. 
iPSCs
Modelling Fanconi anemia pathogenesis and therapeutics using integration-free patient-derived iPSCs.Fanconi anaemia (FA) is a recessive disorder characterized by genomic instability, congenital abnormalities, cancer predisposition and bone marrow (BM) failure.  Here, we derive integration free-induced pluripotent stem cells (iPSCs) from an FA patient without genetic complementation and report in situ gene correction in FA-iPSCs as well as the generation of isogenic FANCA-deficient human embryonic stem cell (ESC) lines.  FA cellular phenotypes are recapitulated in iPSCs/ESCs and their adult stem/progenitor cell derivatives.  We validate our model as a drug-screening platform by identifying several compounds that improve hematopoietic differentiation of FA-iPSCs. 
iPSCs
Modelling Fanconi anemia pathogenesis and therapeutics using integration-free patient-derived iPSCs.Fanconi anaemia (FA) is a recessive disorder characterized by genomic instability, congenital abnormalities, cancer predisposition and bone marrow (BM) failure.  Here, we derive integration free-induced pluripotent stem cells (iPSCs) from an FA patient without genetic complementation and report in situ gene correction in FA-iPSCs as well as the generation of isogenic FANCA-deficient human embryonic stem cell (ESC) lines.  FA cellular phenotypes are recapitulated in iPSCs/ESCs and their adult stem/progenitor cell derivatives.  We validate our model as a drug-screening platform by identifying several compounds that improve hematopoietic differentiation of FA-iPSCs. 
iPSCs
Modelling Fanconi anemia pathogenesis and therapeutics using integration-free patient-derived iPSCs.Fanconi anaemia (FA) is a recessive disorder characterized by genomic instability, congenital abnormalities, cancer predisposition and bone marrow (BM) failure.  Here, we derive integration free-induced pluripotent stem cells (iPSCs) from an FA patient without genetic complementation and report in situ gene correction in FA-iPSCs as well as the generation of isogenic FANCA-deficient human embryonic stem cell (ESC) lines.  FA cellular phenotypes are recapitulated in iPSCs/ESCs and their adult stem/progenitor cell derivatives.  We validate our model as a drug-screening platform by identifying several compounds that improve hematopoietic differentiation of FA-iPSCs. 
iPSCs
Modelling Fanconi anemia pathogenesis and therapeutics using integration-free patient-derived iPSCs.Fanconi anaemia (FA) is a recessive disorder characterized by genomic instability, congenital abnormalities, cancer predisposition and bone marrow (BM) failure.  Here, we derive integration free-induced pluripotent stem cells (iPSCs) from an FA patient without genetic complementation and report in situ gene correction in FA-iPSCs as well as the generation of isogenic FANCA-deficient human embryonic stem cell (ESC) lines.  FA cellular phenotypes are recapitulated in iPSCs/ESCs and their adult stem/progenitor cell derivatives.  We validate our model as a drug-screening platform by identifying several compounds that improve hematopoietic differentiation of FA-iPSCs. 
iPSCs