Autism research is a field of scientific investigation focused on understanding the causes, characteristics, and treatments of autism spectrum disorder (ASD), a neurodevelopmental condition that affects social interaction, communication, and behavior.
It is currently the fastest-growing neurodevelopmental disorder in the United States, impacting approximately one in 36 children (CDC). The increasing awareness and diagnosis of ASD, coupled with the limitations of existing therapies, underscore the need for more research to comprehend the underlying causes of ASD, establish best practices in clinical care, and develop improved treatments for lifelong outcomes.
From cellular studies to service-oriented research, UCLA CART scientists engage in groundbreaking investigations to unveil the root causes of ASD, identify signs indicating the risk of autism and prognosis, and advance the science of ASD treatments.
Research on autism diagnosis focuses on improving methods for identifying Autism Spectrum Disorder (ASD). Scientists explore behavioral assessments, standardized tests, and clinical observations, aiming to enhance accuracy and early detection. The research also investigates innovative technologies for screening and considers diverse manifestations of ASD across individuals, including cultural and gender differences. The goal is to refine diagnostic criteria, facilitating early identification and personalized interventions for better outcomes in individuals with autism.
Like most common human brain disorders, ASD has a strong genetic component. UCLA CART scientists have led the field of ASD genetics for more than two decades. Advances in genetics have changed the understanding of the underlying biological basis of ASD. Now that the genetic variants that cause ASD can be identified in nearly a quarter of autistic individuals, genetic testing has become a standard part of the assessment for all autistic children. These genes affect basic processes in brain development. This knowledge has allowed the team to begin to elucidate the biological basis of autism and it provides important clues as to ASD’s molecular and cellular origins. By understanding the effects of genetic variants that cause ASD, physicians and scientists can develop more specific and effective treatments that are tailored to the individual. This individualization of all aspects of diagnosis, treatment, and prevention is referred to as precision health. Implementing precision autism, delivering the right treatment, at the right time, to every patient, is a key goal of UCLA CART.
Brain mechanisms are analyzed at many levels. At the molecular or cellular level, the team creates stem cell models based on genetic findings and study the effects of specific autism risk genes in scientific models. This is a major effort of several investigators in UCLA CART and in many collaborating laboratories in the UCLA neuroscience community. This allows researchers to connect molecular pathway dysfunction based on specific mutations to individual neuronal cells and circuits. The next step is to connect circuits to behavior, which also can be done in scientific models by manipulating these circuits to test treatments, etc., followed by discovering how these models relate to human behavior. UCLA CART researchers use state-of-the-art brain imaging technologies to understand what is unique about the developing brain in autistic individuals. Using functional magnetic resonance imaging (fMRI) and electroencephalography (EEG) to examine brain activity and wiring, UCLA researchers are beginning to identify the brain mechanisms that lead to autism symptoms. Combining brain-based measures with genetic information enables investigators to determine how different risk factors for autism affect brain development and, ultimately, developmental outcome. MRI and EEG are safe, noninvasive methods that can be used to study individuals across the lifespan, including infants at high-risk for developing autism.
The knowledge gained about genetic mechanisms and their effects on brain function can now be used to inform the development and testing of new drugs for ASD using human stem-cell-based systems and high throughput drug screening. UCLA investigators can make “autism in a dish” using new stem cell technology, and can use this system to screen for new compounds that can reverse the effects of the autism-causing mutations. This work provides a novel and unprecedented opportunity to develop therapies for individuals with ASD. Specific, focused, drug-therapy studies will enable the development of a scientific model based on genetic findings for ASD and support the integration of EEG and imaging as biomarkers of treatment response. Findings can identify existing drugs that were previously unidentified as promising potential treatments for core problems of ASD to be repurposed and studied.
Current treatments for ASD often produce only modest benefits. For example, despite community early-intervention programs, as many as 25-to-30 percent of autistic children never develop functional language. UCLA CART faculty includes preeminent international experts in ASD intervention who are focused on developing and enhancing interventions to address the major gaps in treatment. UCLA CART investigators have developed some of the best evidence-based interventions for ASD, including the earliest studies on applied behavior analysis; social skills training programs, such as the UCLA Program for the Education and Enrichment of Relational Skills (PEERS®), UCLA Children’s Friendship Training, modified cognitive behavioral protocols for treating anxiety and adaptive skill deficits, and Joint Attention, Symbolic Play, Engagement, and Regulation (JASPER) therapy, proven to address core deficits in ASD in many rigorous studies. UCLA CART investigators helped direct clinical trials that led to the first FDA-approved medication for the treatment of severe behavioral problems in autistic children. UCLA CART’s existing teams and staff are recognized as leading some of the most sophisticated intervention programs in the world.
Our team has integrated its research, clinical, and treatment programs to provide a unique, groundbreaking multidisciplinary approach to studying and caring for individuals on the autism spectrum.
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