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Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications
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 multi...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6562607/ https://www.ncbi.nlm.nih.gov/pubmed/31052294 http://dx.doi.org/10.3390/cells8050403 |
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author | Doss, Michael Xavier Sachinidis, Agapios |
author_facet | Doss, Michael Xavier Sachinidis, Agapios |
author_sort | Doss, Michael Xavier |
collection | PubMed |
description | 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. |
format | Online Article Text |
id | pubmed-6562607 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-65626072019-06-17 Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications Doss, Michael Xavier Sachinidis, Agapios Cells Review 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. MDPI 2019-04-30 /pmc/articles/PMC6562607/ /pubmed/31052294 http://dx.doi.org/10.3390/cells8050403 Text en © 2019 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Doss, Michael Xavier Sachinidis, Agapios Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications |
title | Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications |
title_full | Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications |
title_fullStr | Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications |
title_full_unstemmed | Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications |
title_short | Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications |
title_sort | current challenges of ipsc-based disease modeling and therapeutic implications |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6562607/ https://www.ncbi.nlm.nih.gov/pubmed/31052294 http://dx.doi.org/10.3390/cells8050403 |
work_keys_str_mv | AT dossmichaelxavier currentchallengesofipscbaseddiseasemodelingandtherapeuticimplications AT sachinidisagapios currentchallengesofipscbaseddiseasemodelingandtherapeuticimplications |