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Stem Cells Transl Med. 2014 Nov;3(11):1275-86. doi: 10.5966/sctm.2014-0073. Epub 2014 Oct 1.

The Autism Spectrum Disorders Stem Cell Resource at Children's Hospital of Orange County: Implications for Disease Modeling and Drug Discovery.

Author information

1
National Human Neural Stem Cell Resource, Centers for Neuroscience and Translational Research, Children's Hospital of Orange County Research Institute, Orange, California, USA; Department of Developmental and Cell Biology, University of California, Irvine, Irvine, California, USA; Medical Investigation of Neurodevelopmental Disorders (MIND) Institute, Department of Pediatrics, and Department of Biochemistry and Molecular Medicine, University of California, Davis, Sacramento, California, USA.
2
National Human Neural Stem Cell Resource, Centers for Neuroscience and Translational Research, Children's Hospital of Orange County Research Institute, Orange, California, USA; Department of Developmental and Cell Biology, University of California, Irvine, Irvine, California, USA; Medical Investigation of Neurodevelopmental Disorders (MIND) Institute, Department of Pediatrics, and Department of Biochemistry and Molecular Medicine, University of California, Davis, Sacramento, California, USA pschwartz@choc.org.

Abstract

The autism spectrum disorders (ASDs) comprise a set of neurodevelopmental disorders that are, at best, poorly understood but are the fastest growing developmental disorders in the United States. Because animal models of polygenic disorders such as the ASDs are difficult to validate, the derivation of induced pluripotent stem cells (iPSCs) by somatic cell reprogramming offers an alternative strategy for identifying the cellular mechanisms contributing to ASDs and the development of new treatment options. Access to statistically relevant numbers of ASD patient cell lines, however, is still a limiting factor for the field. We describe a new resource with more than 200 cell lines (fibroblasts, iPSC clones, neural stem cells, glia) from unaffected volunteers and patients with a wide range of clinical ASD diagnoses, including fragile X syndrome. We have shown that both normal and ASD-specific iPSCs can be differentiated toward a neural stem cell phenotype and terminally differentiated into action-potential firing neurons and glia. The ability to evaluate and compare data from a number of different cell lines will facilitate greater insight into the cause or causes and biology of the ASDs and will be extremely useful for uncovering new therapeutic and diagnostic targets. Some drug treatments have already shown promise in reversing the neurobiological abnormalities in iPSC-based models of ASD-associated diseases. The ASD Stem Cell Resource at the Children's Hospital of Orange County will continue expanding its collection and make all lines available on request with the goal of advancing the use of ASD patient cells as disease models by the scientific community.

KEYWORDS:

Autism spectrum disorder; Differentiation; Disease modeling; Induced pluripotent stem cells; Neural stem cell; Neuron

PMID:
25273538
PMCID:
PMC4214842
DOI:
10.5966/sctm.2014-0073
[Indexed for MEDLINE]
Free PMC Article

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