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Cells 2018, 7(12), 253; https://doi.org/10.3390/cells7120253

Induced Pluripotent Stem Cells for Duchenne Muscular Dystrophy Modeling and Therapy

1
Institute of Medical Biology, Genetics and Clinical Genetics, Faculty of Medicine, Comenius University, Sasinkova 4, 811 08 Bratislava, Slovakia
2
Institute of Histology and Embryology, Faculty of Medicine, Comenius University, Sasinkova 4, 811 08 Bratislava, Slovakia
*
Author to whom correspondence should be addressed.
Received: 21 November 2018 / Revised: 30 November 2018 / Accepted: 5 December 2018 / Published: 7 December 2018
(This article belongs to the Section Stem Cells)
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Abstract

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder, caused by mutation of the DMD gene which encodes the protein dystrophin. This dystrophin defect leads to the progressive degeneration of skeletal and cardiac muscles. Currently, there is no effective therapy for this disorder. However, the technology of cell reprogramming, with subsequent controlled differentiation to skeletal muscle cells or cardiomyocytes, may provide a unique tool for the study, modeling, and treatment of Duchenne muscular dystrophy. In the present review, we describe current methods of induced pluripotent stem cell generation and discuss their implications for the study, modeling, and development of cell-based therapies for Duchenne muscular dystrophy. View Full-Text
Keywords: induced pluripotent stem cells; cell reprogramming; Duchenne muscular dystrophy; disease modeling; cell-based therapy induced pluripotent stem cells; cell reprogramming; Duchenne muscular dystrophy; disease modeling; cell-based therapy
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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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Danisovic, L.; Culenova, M.; Csobonyeiova, M. Induced Pluripotent Stem Cells for Duchenne Muscular Dystrophy Modeling and Therapy. Cells 2018, 7, 253.

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