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Nat Rev Neurol. 2017 May;13(5):265-278. doi: 10.1038/nrneurol.2017.45. Epub 2017 Apr 18.

Human induced pluripotent stem cells for modelling neurodevelopmental disorders.

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Child Study Center, Yale University School of Medicine, 230 South Frontage Road, New Haven, Connecticut 06520, USA.
Department of Health Sciences Research, Center for Individualized Medicine, 200 First Street SW, Rochester, Minnesota 55905, USA.
Department of Neuroscience, Yale Kavli Institute for Neuroscience, Yale University School of Medicine, 200 South Frontage Road, New Haven, Connecticut 06510, USA.


We currently have a poor understanding of the pathogenesis of neurodevelopmental disorders, owing to the fact that postmortem and imaging studies can only measure the postnatal status quo and offer little insight into the processes that give rise to the observed outcomes. Human induced pluripotent stem cells (hiPSCs) should, in principle, prove powerful for elucidating the pathways that give rise to neurodevelopmental disorders. hiPSCs are embryonic-stem-cell-like cells that can be derived from somatic cells. They retain the unique genetic signature of the individual from whom they were derived, and thus enable researchers to recapitulate that individual's idiosyncratic neural development in a dish. In the case of individuals with disease, we can re-enact the disease-altered trajectory of brain development and examine how and why phenotypic and molecular abnormalities arise in these diseased brains. Here, we review hiPSC biology and possible experimental designs when using hiPSCs to model disease. We then discuss existing hiPSC models of neurodevelopmental disorders. Our hope is that, as some studies have already shown, hiPSCs will illuminate the pathophysiology of developmental disorders of the CNS and lead to therapeutic options for the millions that are affected by these conditions.

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