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Chunk #7 — Induced pluripotent stem cells (iPSCs) for disease modelling — iPSC technology

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Editing the genome of hiPSC with CRISPR/Cas9: disease models.
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The advent of induced pluripotent stem cell (iPSC) technology (Takahashi et al. 2007; Takahashi and Yamanaka 2006) has revolutionised many fields, notably those of disease modelling and cellular therapeutics due to our ability to generate such pluripotent stem cells from essentially any human, including those with disease (Avior et al. 2016). Somatic cells can be reprogrammed to a pluripotent stem cell state similar to that present in very early embryogenesis through transient expression of four transcription factors (Oct4, Sox2, Klf4 and c-Myc) (Takahashi et al. 2007; Takahashi and Yamanaka 2006). Importantly, such cells are diploid and karyotypically normal, can self-renew for many cell divisions and can be differentiated into a broad range of different cell types. These characteristics lend themselves to the study of development and cellular function both in normal and disease states, and also allow large numbers of cells to be produced for high throughput genetic and drug screening as well as cell therapy. This has led to the inception of several large-scale initiatives for deriving iPSCs from thousands of normal and diseased patients (California Institute for Regenerative