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Yanhong Shiet al. Nat Rev Drug Discov. 2017 Feb.

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Since the advent of induced pluripotent stem cell (iPSC) technology a decade ago, enormous progress has been made in stem cell biology and regenerative medicine. Human iPSCs have been widely used for disease modelling, drug discovery and cell therapy development. Novel pathological mechanisms have been elucidated, new drugs originating from iPSC screens are in the pipeline and the first clinical trial using human iPSC-derived products has been initiated. In particular, the combination of human iPSC technology with recent developments in gene editing and 3D organoids makes iPSC-based platforms even more powerful in each area of their application, including precision medicine. In this Review, we discuss the progress in applications of iPSC technology that are particularly relevant to drug discovery and regenerative medicine, and consider the remaining challenges and the emerging opportunities in the field.

Conflict of Interest

J.C.W. is a co-founder of Stem Cell Theranostics. S.Y. is a scientific advisor of iPS Academia Japan without salary. The other authors declare no conflict of interest.

Figure 1

A schematic for human iPSC-based

Figure 1

A schematic for human iPSC-based disease modeling. Human iPSCs are derived from individual

A schematic for human iPSC-based disease modeling. Human iPSCs are derived from individual patients and differentiated into specific cell types. To develop new therapies, the resultant cells are used to observe disease-specific phenotypes and identify novel pathological mechanisms,. Human iPSC-based disease modeling with patient-specific cells now provides an exciting new approach for the development of personalized diagnosis and medicine.

Figure 2

A schematic for human iPSCs-based

Figure 2

A schematic for human iPSCs-based cell therapy. Human iPSC-based cell therapy development usually

A schematic for human iPSCs-based cell therapy. Human iPSC-based cell therapy development usually includes the following steps: 1) Collect somatic cells from patients and culture somatic cells from affected patients; 2) Reprogram patient somatic cells into iPSCs; 3) Use genome editing technology or viral transduction method to repair patient iPSCs and turn them into genetically corrected iPSCs; 4) Differentiate the corrected iPSCs into desired cell types to serve as genetically matched healthy donor cells; 5) Perform quality control test for cell identity, purity, activity, and safety; and 6) Transplant the genetically matched healthy cells into patients for cell therapy.

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Induced pluripotent stem cell technology: a decade of progress