Autism Fact Sheet

Autism Fact Sheet

CIRM funds several research projects investigating how the brain normally matures during development by studying stem cells as they differentiate into different types of nerves. This work could lead to a better understanding of how diseases such as autism form, and to future therapies. CIRM also funds a project looking specifically at the development of mental disorders.

If you want to learn more about CIRM funding decisions or make a comment directly to our board, join us at a public meeting. You can find agendas for upcoming public meetings on our meetings page.

Learn more about stem cell research:
Stem Cell Basics Primer | Stem Cell Videos | What We Fund

Find clinical trials:
CIRM does not track stem cell clinical trials. If you or a family member is interested in participating in a clinical trial, please see the national trial database to find a trial near you: clinicaltrials.gov

The role of stem cell research in autism

Autism is not one specific condition but a range of developmental disorders called Autism Spectrum Disorders (ASD) that can lead to difficulties with verbal and non-verbal communication, impaired social skills and other behavioral problems.

According to the Centers for Disease Control and Prevention 1 in 88 children eight years of age has ASD, with boys four times more likely than girls to be affected. The precise cause of ASD is not known, however, there seems to be a strong genetic component to it.

While behavioral therapy has been shown to help those with ASD there is no cure. Some medications have shown effectiveness in easing symptoms but none treat the underlying cause of the problem.

CIRM funds several research projects investigating how the brain normally matures during development by studying stem cells as they differentiate or change into different types of nerves. This work could lead to a better understanding of how diseases such as autism form, and to future therapies.

Several CIRM-funded research projects are also using stem cells derived from people with ASD to learn more about how the symptoms of ASD arise and also to screen for drugs. These types of projects start with cells taken from people with ASD. Even if those cells come from the skin they have the same genes as cells in the brain that show symptoms in the disease. These cells can then be reprogrammed into an embryonic-like state called an iPS cell, and matured into brain cells. Those resulting brain cells are genetically identical to brain cells in the person who donated the tissue.

CIRM-funded scientists have shown that these autism-like cells behave very differently in a lab dish than normal cells. Studying those cells can help scientists learn what goes wrong in the disease and guide them toward new therapies. What’s more, the scientists can expose those cells to drugs and see which ones alleviate symptoms in the lab dish.

CIRM Grants Targeting Autism

Researcher name Institution Grant Title Approved funds
Alysson Muotri University of California, San Diego Developing a drug-screening system for Autism Spectrum Disorders using human neurons $1,410,697
Fred Gage The Salk Institute for Biological Studies Development of Induced Pluripotent Stem Cells for Modeling Human Disease $1,737,720
Fen-Biao Gao The J. David Gladstone Institutes MicroRNAs in Human Stem Cell Differentiation and Mental Disorders $748,800
Theo Palmer Stanford University Development of small molecule screens for autism using patient-derived iPS cells $1,797,981
Marius Wernig Stanford University Cellular tools to study brain diseases affecting synaptic transmission $1,664,382
Yi Sun University of California, Los Angeles Studying neurotransmission of normal and diseased human ES cell-derived neurons in vivo $1,382,400
Anirvan Ghosh University of California, San Diego Investigation of synaptic defects in autism using patient-derived induced pluripotent stem cells $843,597
Joachim Hallmayer Stanford University Induced pluripotent stem cells from children with autism spectrum disorders $530,265
Thomas Novak Cellular Dynamics International Generation and characterization of high-quality, footprint-free human induced pluripotent stem cell lines from 3,000 donors to investigate multigenic diseases $16,000,000
Steven Madore Coriell Institute for Medical Research The CIRM Human Pluripotent Stem Cell Biorepository – A Resource for Safe Storage and Distribution of High Quality iPSCs $9,942,175
Alysson Muotri University of California, San Diego A drug-screening platform for autism spectrum disorders using human astrocytes $1,656,456
Total:
$37,714,473.00

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