Disease Focus: Autism
Investigation of synaptic defects in autism using patient-derived induced pluripotent stem cells
Autism spectrum disorders (ASD) are a group of neurodevelopmental diseases that occur in as many as 1 in 150 children in the United States. Three hallmarks of autism are dysfunctional communication, impaired social interaction, and restricted and repetitive interests and activities. Even though no single genetic defect has been ascribed to having a causative role […]
Cellular tools to study brain diseases affecting synaptic transmission
There is a group of brain diseases that are caused by functional abnormalities. The brains of patients afflicted with these diseases which include autism spectrum disorders, schizophrenia, depression, and mania and other psychiatric diseases have a normal appearance and show no structural changes. Neurons, the cellular units of the brain, function by making connections (or […]
Development of small molecule screens for autism using patient-derived iPS cells
Autism Spectrum Disorders (ASDs) are a heritable group of neuro-developmental disorders characterized by language impairments, difficulties in social integrations, and the presence of stereotyped and repetitive behaviors. There are no treatments for ASDs, and very few targets for drug development. Recent evidence suggests that some types of ASDs are caused by defects in calcium signaling […]
Developing a drug-screening system for Autism Spectrum Disorders using human neurons
Autism and autism spectrum disorders (ASD) are complex neurodevelopmental diseases that affect 1 in 150 children in the United States. Such diseases are mainly characterized by deficits in verbal communication, impaired social interaction, and limited and repetitive interests and behavior. Because autism is a complex spectrum of disorders, a different combination of genetic mutations is […]
Development of Induced Pluripotent Stem Cells for Modeling Human Disease
Human embryonic stem cells (hESC) hold great promise in regenerative medicine and cell replacement therapies because of their unique ability to self-renew and their developmental potential to form all cell lineages in the body. Traditional techniques for generating hESC rely on surplus IVF embryos and are incompatible with the generation of genetically diverse, patient or […]
MicroRNAs in Human Stem Cell Differentiation and Mental Disorders
Many mental disorders are closely associated with problems that occur during brain development in early life. For instance, by 2 years of age, autistic children have larger brains than normal kids, likely due to, at least in part, excess production of neurons and support cells, the building blocks of the nervous system. In autistic brains, […]
Reversing Autism in the Lab with help from Stem Cells and the Tooth Fairy
People with autism have difficulty with language and social interaction as if they are trapped inside their own brains. There is no cure. Dr. Alysson Muotri of UCSD devised a Fairy Tooth Kit Collection campaign in which autistic and unaffected kids could donate their baby teeth when they fell out. Using the induced pluripotent stem […]
Autism and Stem Cells: A Mom’s Perspective
Jen, mother of 10 year-old Milo who has a severe form of autism describes the impact of the disorder on her family and shares her thoughts about stem cell research. This video was presented during at the Spotlight on Autism seminar at the March 13th 2014 governing Board meeting of California’s Stem Cell Agency. For […]
Seminar Intro: Autism, Toxicology, Environmental Health and Stem Cell Science
Since World War II, over 80,000 chemical compounds have been developed in the U.S. About 3,000 of these chemicals are produced at rate of one million pounds per year. Do these chemicals have potential public health risks? Can exposure to these chemicals be linked to childhood development disorders, such as autism? What challenges do environmental […]
Eric Roberts: Pesticides and Autism
Since World War II, over 80,000 chemical compounds have been developed in the U.S. About 3,000 of these chemicals are produced at rate of one million pounds per year. Do these chemicals have potential public health risks? Can exposure to these chemicals be linked to childhood development disorders, such as autism? What challenges do environmental […]