The Human Model: Changing Focus on Autism Research

Abstract The lack of live human brain cells for research has slowed progress toward understanding the mechanisms underlying autism spectrum disorders. A human model using reprogrammed patient somatic cells offers an attractive alternative, as it captures a patient’s genome in relevant cell types. De...

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Bibliographic Details
Published in:Biological psychiatry (1969) 2016-04, Vol.79 (8), p.642-649
Main Author: Muotri, Alysson Renato
Format: Article
Language:English
Subjects:
Animals
Autism spectrum disorders
Autistic Disorder - physiopathology
Brain
Brain - physiopathology
Drug screening
Human induced pluripotent stem cells
Human neurons
Human-specific disease modeling
Humans
Induced Pluripotent Stem Cells - physiology
Models, Biological
Psychiatry
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Summary:Abstract The lack of live human brain cells for research has slowed progress toward understanding the mechanisms underlying autism spectrum disorders. A human model using reprogrammed patient somatic cells offers an attractive alternative, as it captures a patient’s genome in relevant cell types. Despite the current limitations, the disease-in-a-dish approach allows for progressive time course analyses of target cells, offering a unique opportunity to investigate the cellular and molecular alterations before symptomatic onset. Understanding the current drawbacks of this model is essential for the correct data interpretation and extrapolation of conclusions applicable to the human brain. Innovative strategies for collecting biological material and clinical information from large patient cohorts are important for increasing the statistical power that will allow for the extraction of information from the noise resulting from the variability introduced by reprogramming and differentiation methods. Working with large patient cohorts is also important for understanding how brain cells derived from diverse human genetic backgrounds respond to specific drugs, creating the possibility of personalized medicine for autism spectrum disorders.
ISSN:0006-3223
1873-2402
DOI:10.1016/j.biopsych.2015.03.012