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Cells 2019, 8(1), 20; https://doi.org/10.3390/cells8010020

iPSCs as a Platform for Disease Modeling, Drug Screening, and Personalized Therapy in Muscular Dystrophies

Center for Stem Cell and Regenerative Medicine (CSCRM), The Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases (IMM), The University of Texas Health Science Center at Houston, Houston, TX 77030, USA
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Received: 6 November 2018 / Revised: 20 December 2018 / Accepted: 26 December 2018 / Published: 3 January 2019
(This article belongs to the Special Issue Stem Cells in Personalized Medicine)
Full-Text   |   PDF [236 KB, uploaded 3 January 2019]

Abstract

Induced pluripotent stem cells (iPSCs) are the foundation of modern stem cell-based regenerative medicine, especially in the case of degenerative disorders, such as muscular dystrophies (MDs). Since their introduction in 2006, many studies have used iPSCs for disease modeling and identification of involved mechanisms, drug screening, as well as gene correction studies. In the case of muscular dystrophies, these studies commenced in 2008 and continue to address important issues, such as defining the main pathologic mechanisms in different types of MDs, drug screening to improve skeletal/cardiac muscle cell survival and to slow down disease progression, and evaluation of the efficiency of different gene correction approaches, such as exon skipping, Transcription activator-like effector nucleases (TALENs), Zinc finger nucleases (ZFNs) and RNA-guided endonuclease Cas9 (CRISPR/Cas9). In the current short review, we have summarized chronological progress of these studies and their key findings along with a perspective on the future road to successful iPSC-based cell therapy for MDs and the potential hurdles in this field. View Full-Text
Keywords: iPSCs; muscular dystrophies; DMD; duchenne; gene correction; CRISPR/Cas9; stem cells iPSCs; muscular dystrophies; DMD; duchenne; gene correction; CRISPR/Cas9; stem cells
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).

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Ortiz-Vitali, J.L.; Darabi, R. iPSCs as a Platform for Disease Modeling, Drug Screening, and Personalized Therapy in Muscular Dystrophies. Cells 2019, 8, 20.

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