Concise Review: Induced Pluripotent Stem Cell-Based Drug Discovery for Mitochondrial Disease.
Stem Cells
; 35(7): 1655-1662, 2017 07.
Article
em En
| MEDLINE
| ID: mdl-28544378
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. Stem Cells 2017;351655-1662.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Fármacos Neuroprotetores
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Doenças Mitocondriais
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Descoberta de Drogas
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Células-Tronco Pluripotentes Induzidas
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Mitocôndrias
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Neurônios
Tipo de estudo:
Prognostic_studies
Limite:
Humans
Idioma:
En
Revista:
Stem Cells
Ano de publicação:
2017
Tipo de documento:
Article