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Concise review: Induced pluripotent stem cell-based drug discovery for mitochondrial disease

Official URL:https://doi.org/10.1002/stem.2637
PubMed:View item in PubMed
Creators Name:Inak, G. and Lorenz, C. and Lisowski, P. and Zink, A. and Mlody, B. and Prigione, A.
Journal Title:Stem Cells
Journal Abbreviation:Stem Cells
Date:22 May 2017
Abstract:High attrition rates and loss of capital plague the drug discovery process. This is particularly evident for mitochondrial disease that typically involves neurological manifestations and is caused by nuclear or mitochondrial DNA defects. This group of heterogeneous disorders is difficult to target because of the variability of the symptoms among individual patients and the lack of viable modeling systems. The use of induced pluripotent stem cells (iPSCs) might significantly improve the search for effective therapies for mitochondrial disease. iPSCs can be used to generate patient-specific neural cell models in which innovative compounds can be identified or validated. Here we discuss the promises and challenges of iPSC-based drug discovery for mitochondrial disease with a specific focus on neurological conditions. We anticipate that a proper use of the potent iPSC technology will provide critical support for the development of innovative therapies against these untreatable and detrimental disorders.
ISSN:1066-5099
Publisher:Wiley-Blackwell (U.S.A.)
Item Type:Review

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