Autism Fact Sheet

Disease Fact Sheets
 All Diseases

Autism

Embryonic stem cells can mature into all cells of the nervous system. These could be used to directly treat some diseases or could be useful for studying the origins of disease and designing new therapies. Lean more about this image by clicking on it or see more nervous system images on our Flickr Photostream.

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

One of the more exciting stem cell research announcements of 2010 occurred in the study of autism, and was done by researchers funded by CIRM. A collaborative effort between researchers at the Salk Institute for Biological Studies and the University of California San Diego, successfully used human induced pluripotent stem (iPS) cells derived from patients with Rett syndrome to replicate autism in the lab and study the molecular pathogenesis of the disease. Rett syndrome is the most physically disabling of the autism spectrum disorders. Primarily affecting girls, the symptoms of Rett syndrome often become apparent just after they have learned to walk and say a few words. (Here is a summary of the award that led to this publication.)

The scientists’ findings, published in the Nov. 12, 2010, issue of Cell, revealed disease-specific cellular defects, including fewer functional connections between Rett neurons as well as defects in nerve cell signaling, and demonstrated that these symptoms are reversible – highlighting the potential that the disease may one day be reversed in patients, raising the hope that, one day, autism may be a treatable condition. These findings are the basis for a new CIRM-funded project to develop a screening platform to identify therapeutics using Autism Spectrum Disorder (ASD) patient-derived cells (here is a summary of that award).

In other research, also funded by CIRM, Stanford University scientists are looking at some heritable ASDs that may be caused by defects in calcium signaling during development of the nervous system. (Here is a summary of that award.) The researchers are developing iPS cells that have a mutant calcium channel similar to that seen in ASDs. These cells will then be used to test drugs to see if the symptoms of ASD can be reversed.

CIRM-funded scientists at the Gladstone Institutes in San Francisco are looking at developmental defects associated with mental disorders caused by single-gene mutations, such as Rett syndrome and fragile X syndrome, the most common form of inherited mental retardation, whose clinical features overlap with autism. (Here is a summary of that award.)

CIRM Grants Targeting Autism

CIRM Autism Videos

News and Information

Resources