Open AccessConcise Review: Induced Pluripotent Stem Cell‐Based Drug Discovery for Mitochondrial Disease
Author(s) -
Inak Gizem,
Lorenz Carmen,
Lisowski Pawel,
Zink Annika,
Mlody Barbara,
Prigione Alessandro
Publication year - 2017
Publication title -
stem cells
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.159
H-Index - 229
eISSN - 1549-4918
pISSN - 1066-5099
DOI - 10.1002/stem.2637
Subject(s) - induced pluripotent stem cell , biology , drug discovery , disease , mitochondrial disease , mitochondrion , neuroscience , mitochondrial dna , bioinformatics , computational biology , microbiology and biotechnology , genetics , embryonic stem cell , pathology , medicine , gene
A bstract 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. S tem C ells 2017;35:1655–1662