Autism Fact Sheet

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

Description

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 children 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 stem 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 Name	Institution	Grant Title	Grant Type	Award Amount
Professor Alysson R Muotri	University of California, San Diego	A drug-screening platform for autism spectrum disorders using human astrocytes	Early Translational IV	$1,656,456
Dr. Joachim Franz Hallmayer	Stanford University	Induced pluripotent stem cells from children with autism spectrum disorders	Tissue Collection for Disease Modeling	$530,265
Dr. Yi Eve Sun	University of California, Los Angeles	Studying neurotransmission of normal and diseased human ES cell-derived neurons in vivo	Basic Biology III	$1,382,400
Dr. Anirvan Ghosh Dr.	University of California, San Diego	Investigation of synaptic defects in autism using patient-derived induced pluripotent stem cells	Basic Biology III	$843,597
Dr. Marius Wernig MD, PhD	Stanford University	Cellular tools to study brain diseases affecting synaptic transmission	Tools and Technologies II	$1,664,382
Dr. Theo D. Palmer PhD	Stanford University	Development of small molecule screens for autism using patient-derived iPS cells	Tools and Technologies II	$1,797,606
Professor Alysson R Muotri	University of California, San Diego	Developing a drug-screening system for Autism Spectrum Disorders using human neurons	Early Translational II	$1,376,198
Dr. Fred H Gage	Salk Institute for Biological Studies	Development of Induced Pluripotent Stem Cells for Modeling Human Disease	New Cell Lines	$1,737,720
Dr. Fen-Biao N. Gao	Gladstone Institutes, J. David	MicroRNAs in Human Stem Cell Differentiation and Mental Disorders	SEED Grant	$748,800
Alex Pollen	University of California, San Francisco	Deep phenotyping of human brain organoid models of autism spectrum disorder to unravel disease heterogeneity and develop biomarkers and treatments	ReMIND – Discovery Stage ReMIND	$12,297,272
Xin Jin	Scripps Research Institute	Functional genomics to study cellular convergence across ASD risk genes in neurodevelopment	Foundation – Discovery Stage Research Projects	$1,575,001
Dr. Gerald Lipshutz	University of California, Los Angeles	Gene Therapy for SLC6A8 Creatine Transporter Disorder	Quest – Discovery Stage Research Projects	$2,296,920
Professor Alysson R Muotri	University of California, San Diego	A treatment for Rett syndrome using glial-restricted neural progenitor cells	Quest – Discovery Stage Research Projects	$1,402,240
Dr. Joseph G. Gleeson	University of California, San Diego	Novel antisense therapy to treat genetic forms of neurodevelopmental disease.	Quest – Discovery Stage Research Projects	$1,180,654
Dr. David J Segal PhD	University of California, Davis	AAV9-Cas13 gene therapy for Angelman syndrome	Quest – Discovery Stage Research Projects	$1,364,903
Stuart A Lipton	Scripps Research Institute	Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs	Quest – Discovery Stage Research Projects	$1,827,576
Dr. David J Segal PhD	University of California, Davis	MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome	Quest – Discovery Stage Research Projects	$1,055,319
				Total: $34,737,309.03

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