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Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications

1
Technology Development Division, BioMarin Pharmaceutical Inc, 105 Digital Drive, Novato, CA 94949, USA
2
Institute of Neurophysiology and Center for Molecular Medicine, University of Cologne, Robert-Koch Str. 39, 50931 Cologne, Germany
*
Author to whom correspondence should be addressed.
Cells 2019, 8(5), 403; https://doi.org/10.3390/cells8050403
Received: 9 April 2019 / Revised: 23 April 2019 / Accepted: 26 April 2019 / Published: 30 April 2019
(This article belongs to the Special Issue iPS Cells for Disease Modeling)
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PDF [701 KB, uploaded 30 April 2019]
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Abstract

Induced pluripotent stem cell (iPSC)-based disease modelling and the cell replacement therapy approach have proven to be very powerful and instrumental in biomedical research and personalized regenerative medicine as evidenced in the past decade by unraveling novel pathological mechanisms of a multitude of monogenic diseases at the cellular level and the ongoing and emerging clinical trials with iPSC-derived cell products. iPSC-based disease modelling has sparked widespread enthusiasm and has presented an unprecedented opportunity in high throughput drug discovery platforms and safety pharmacology in association with three-dimensional multicellular organoids such as personalized organs-on-chips, gene/base editing, artificial intelligence and high throughput “omics” methodologies. This critical review summarizes the progress made in the past decade with the advent of iPSC discovery in biomedical applications and regenerative medicine with case examples and the current major challenges that need to be addressed to unleash the full potential of iPSCs in clinical settings and pharmacology for more effective and safer regenerative therapy. View Full-Text
Keywords: induced pluripotent stem cells; cell replacement therapy; drug discovery; safety pharmacology; disease modeling; autologous cell therapy; allogenic cell therapy; clinical trials with stem cells induced pluripotent stem cells; cell replacement therapy; drug discovery; safety pharmacology; disease modeling; autologous cell therapy; allogenic cell therapy; clinical trials with stem cells
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Doss, M.X.; Sachinidis, A. Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications. Cells 2019, 8, 403.

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