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Trends Mol Med. 2014 Feb;20(2):91-104. doi: 10.1016/j.molmed.2013.11.004. Epub 2013 Dec 24.

iPSC-derived neurons as a higher-throughput readout for autism: promises and pitfalls.

Author information

1
Center for Biomedical Informatics, Harvard Medical School, Boston, MA 02115, USA.
2
Amherst College Class of 2013 Undergraduate Neuroscience Program, Amherst, MA, 01002, USA.
3
Stem Cell Program, Boston Children's Hospital, Boston, MA 02115, USA.
4
Center for Biomedical Informatics, Harvard Medical School, Boston, MA 02115, USA; Divisions of Genetics and Developmental Medicine, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA.
5
Center for Biomedical Informatics, Harvard Medical School, Boston, MA 02115, USA; Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA. Electronic address: Isaac_Kohane@hms.harvard.edu.

Abstract

The elucidation of disease etiologies and establishment of robust, scalable, high-throughput screening assays for autism spectrum disorders (ASDs) have been impeded by both inaccessibility of disease-relevant neuronal tissue and the genetic heterogeneity of the disorder. Neuronal cells derived from induced pluripotent stem cells (iPSCs) from autism patients may circumvent these obstacles and serve as relevant cell models. To date, derived cells are characterized and screened by assessing their neuronal phenotypes. These characterizations are often etiology-specific or lack reproducibility and stability. In this review, we present an overview of efforts to study iPSC-derived neurons as a model for autism, and we explore the plausibility of gene expression profiling as a reproducible and stable disease marker.

KEYWORDS:

autism; gene expression; high-throughput assay; iPSC

PMID:
24374161
PMCID:
PMC4117413
DOI:
10.1016/j.molmed.2013.11.004
[Indexed for MEDLINE]
Free PMC Article

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