Open AccessConcise Review: Exciting Cells: Modeling Genetic Epilepsies with Patient‐Derived Induced Pluripotent Stem Cells
Author(s) -
Tidball Andrew M.,
Parent Jack M.
Publication year - 2016
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.2203
Subject(s) - induced pluripotent stem cell , epilepsy , biology , neuroscience , drug discovery , rett syndrome , dravet syndrome , computational biology , embryonic stem cell , bioinformatics , genetics , gene
Human induced pluripotent stem cell (iPSC) models of epilepsy are becoming a revolutionary platform for mechanistic studies and drug discovery. The skyrocketing pace of epilepsy gene discovery is vastly outstripping the development of in vivo animal models. Currently, antiepileptic drug prescribing to patients with specific genetic epilepsies is based on small‐scale clinical trials and empiricism; however, rapid production of patient‐derived iPSC models will allow for precision therapy. We review iPSC‐based studies that have already afforded novel discoveries in diseases with epileptic phenotypes, as well as challenges to using iPSC‐based neurological disease models. We also discuss iPSC‐derived cardiomyocyte studies of arrhythmia‐inducing ion channelopathies that exemplify novel drug discovery and use of multielectrode array technology that can be translated to epilepsy research. Beyond initial studies of Rett, Timothy, Phelan‐McDermid, and Dravet syndromes, the stage is set for groundbreaking iPSC‐based mechanistic and therapeutic discoveries in genetic epilepsies with the potential to impact patient treatment and quality of life. S tem C ells 2016;34:27–33