Autism Fact Sheet

CIRM funds many projects seeking to better understand autism and to translate those discoveries into new therapies.


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 68 childrenĀ 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 induced pluripotent stemĀ cell (iPSC), 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 NameInstitutionGrant TitleGrant TypeAward Amount
Xin JinScripps Research InstituteFunctional genomics to study cellular convergence across ASD risk genes in neurodevelopmentFoundation – Discovery Stage Research Projects$1,575,001
Dr. Gerald LipshutzUniversity of California, Los AngelesGene Therapy for SLC6A8 Creatine Transporter DisorderQuest – Discovery Stage Research Projects$2,296,920
Professor Alysson R MuotriUniversity of California, San DiegoA treatment for Rett syndrome using glial-restricted neural progenitor cellsQuest – Discovery Stage Research Projects$1,402,240
Dr. Joseph G. GleesonUniversity of California, San DiegoNovel antisense therapy to treat genetic forms of neurodevelopmental disease.Quest – Discovery Stage Research Projects$1,180,654
Dr. David J Segal PhDUniversity of California, DavisAAV9-Cas13 gene therapy for Angelman syndromeQuest – Discovery Stage Research Projects$1,364,903
Stuart A LiptonScripps Research InstituteDrug Development for Autism Spectrum Disorder Using Human Patient iPSCsQuest – Discovery Stage Research Projects$1,827,576
Dr. David J Segal PhDUniversity of California, DavisMSC delivery of an artificial transcription factor to the brain as a treatment for Angelman SyndromeQuest – Discovery Stage Research Projects$1,055,319
Professor Alysson R MuotriUniversity of California, San DiegoA drug-screening platform for autism spectrum disorders using human astrocytesEarly Translational IV$1,656,456
Dr. Joachim Franz HallmayerStanford UniversityInduced pluripotent stem cells from children with autism spectrum disordersTissue Collection for Disease Modeling$530,265
Dr. Yi Eve SunUniversity of California, Los AngelesStudying neurotransmission of normal and diseased human ES cell-derived neurons in vivoBasic Biology III$1,382,400
Dr. Anirvan Ghosh Dr.University of California, San DiegoInvestigation of synaptic defects in autism using patient-derived induced pluripotent stem cellsBasic Biology III$843,597
Dr. Marius Wernig MD, PhDStanford UniversityCellular tools to study brain diseases affecting synaptic transmissionTools and Technologies II$1,664,382
Dr. Theo D. Palmer PhDStanford UniversityDevelopment of small molecule screens for autism using patient-derived iPS cellsTools and Technologies II$1,797,606
Professor Alysson R MuotriUniversity of California, San DiegoDeveloping a drug-screening system for Autism Spectrum Disorders using human neuronsEarly Translational II$1,376,198
Dr. Fred H GageSalk Institute for Biological StudiesDevelopment of Induced Pluripotent Stem Cells for Modeling Human DiseaseNew Cell Lines$1,737,720
Dr. Fen-Biao N. GaoGladstone Institutes, J. DavidMicroRNAs in Human Stem Cell Differentiation and Mental DisordersSEED Grant$748,800

CIRM Autism Videos

Reversing Autism in the Lab with help from Stem Cells and the Tooth Fairy

Autism and Stem Cells: A Mom’s Perspective

Seminar Intro: Autism, Toxicology, Environmental Health and Stem Cell Science

Eric Roberts: Pesticides and Autism

Michael McMaster: Human Embryonic Stem Cells for Predictive Toxicology

Tracy Woodruff: So Many Chemicals…So Little Time

News and Information


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Stem Cell FAQ | Stem Cell Videos | What We Fund