Generation of human β-thalassemia induced pluripotent stem cells from amniotic fluid cells with non-

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  Objective Induced pluripotent stem cells (iPSCs) derived from somatic cells of patients represent a powerful tool for biomedical research and may have a wide range of applications in cell and gene therapy.However, the safety issues and the low efficiency associated with generation of human iPS cells have limited their usage in clinical settings.Cell types can significantly influence reprogramming efficiency and kinetics.Methods Here, we show that amniotic fluid cells from the prenatal diagnosis of a β-thalassemia patient could be efficiently reprogrammed using episomal, non-integrating, oriP/EBNA1-based plasmids.Results We demonstrated that the patient derived hiPSCs could be characterized for their expression of pluripotency marker and be differentiated into various somatic cell types in vitro and in vivo.Moreover, microarray analysis demonstrates a high correlation coefficient between human β-thalassemia iPS cells and human embryonic stem cells, but a low correlation coefficient between human β-thalassemia amniotic fluid cells and humanβ-thalassemia iPS cells.Furthermore, iPSCs can be readily derived from hAFCs in a feeder-free conditions, thereby eliminating the potential variability caused by using feeder cells.Conclusions Our results indicate that hAFCs represent an accessible source of cells that can be reprogrammed into pluripotent stem cells with a non-integrating plasmid.Therefore, hAFCs may become a preferred cell type in the future for generation of patient-specific iPS cells without any exogenous genetic material.
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