| May 25, 2015Autism spectrum disorders (ASD) comprise a group of developmental disabilities that can cause considerable behavioral, communication and social challenges. There are likely many causes for ASD, including biologic, genetic and environmental factors, and currently there are no cures. Because of genetic links to ASD, a number of mouse models which target risk genes have been generated in an effort to model the disorders and to develop research tools to screen possible therapeutics.
But according to Dr. Alysson Muotri, associate professor of Pediatrics and Cellular and Molecular Medicine at the University of California, due to important biological and behavioral differences between humans and mice, animal models may not have helped, and in fact may have hindered, progress in this important area of research. He recently stated, “The unreliability of the mouse model has slowed translational progress in ASD, as the vast majority of drugs tested in mice have not worked in humans.”
In a personal communication with NAVS, Dr. Muotri shared, “I started shifting away from animal models to study autism because of the poor translational potential and the genetic limitations imposed by the species-specific genomic architecture between humans and mice.”
His lab has chosen to work with cells from individuals with ASD to model the disorder. Dental cells are extracted from the baby teeth of children that have ASD, which are donated to the lab by the children’s family. These cells are used to create induced pluripotent stem cells, and then ultimately reprogrammed into brain cells. Using this approach, the Muorti lab is better able to understand how specific gene changes can lead to problems with neurons. Muorti’s team has even identified promising therapeutic strategies which have reversed some of the defects observed in the ASD cells.
Importantly, Dr. Muotri points out that it is essential for research funding to be prioritized to support the development of more human-relevant approaches, including cell-based models, human brain imaging and computational simulations, all of which have more direct relevance to humans than animal models.
What do you think of using human stem cells to better understand the pathways affected in different types of autism and to possibly identify personalized treatments? Send your questions and comments to sciencecorner@navs.org. I look forward to hearing from you.
–Dr. Pam Osenkowski, Director of Science Programs
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