Understanding and reversing brain dysfunction linked to autism and intellectual disability
Official title: Novel SETD5-based Molecular Mechanisms and Therapeutic Tools to Understand and Revert Neuronal Dysfunction Associated with Intellectual disability and Autism
Funded at University of California, San Diego · NIH-11010745 · Data updated 2026-11-12
This study is looking into how certain genes might affect young children with autism and intellectual disabilities, especially those diagnosed early, to find new ways to help improve their social and communication skills.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project start | March 5, 2022 |
| Project end | December 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $664,069 |
| NIH project number | 5R01MH127077-04 |
| Project ID | NIH-11010745 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children aged two years or younger who have been diagnosed with autism spectrum disorder or intellectual disability.
✕Less likely to help
Patients who are older than 11 years or do not have a diagnosis of autism spectrum disorder or intellectual disability may not benefit from this research.
What this research studies
This research investigates the molecular mechanisms behind autism spectrum disorder (ASD) and intellectual disability (ID) in young children, particularly those diagnosed by age two. It employs advanced techniques, including CRISPR technology, to explore genetic variations and their effects on brain function. The goal is to identify potential therapeutic tools that could help improve the social, emotional, and communication skills of affected individuals. By understanding these mechanisms, the research aims to pave the way for effective treatments that address the root causes of these neurodevelopmental disorders.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve the quality of life for children with autism and intellectual disabilities.
How similar studies have gone: Other research has shown promise in using genetic and molecular approaches to understand and potentially treat autism and related disorders, indicating that this approach is building on existing knowledge.
Where this research is happening
-
University of California, San Diego
La Jolla, United States Active
Researchers
- Principal investigator: Muotri, Alysson R. — University of California, San Diego
- Study coordinator: Muotri, Alysson R.
Related research
Other NIH-funded projects on the same topics.
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Understanding brain responses in children with autism and intellectual disability
This project uses child-friendly EEG and eye-tracking to measure brain responses and looking patterns in 6–11-year-old children with autism and intellectual disability.
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Understanding and fixing SETD5-related brain problems in children with autism and intellectual disability
Developing treatments that target the SETD5 gene to help young children with autism and intellectual disability.
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Including individuals with intellectual disabilities in autism research
This study is looking at how children with autism and intellectual disabilities think and pay attention, using cool tools like brain scans and eye trackers, to help us find better ways to support them.
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Understanding genetic causes of intellectual disability across diverse populations
Aiming to find gene variants that contribute to intellectual disability in people from African, Asian, and European backgrounds to improve diagnosis and future treatments.
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Understanding early cognitive skills in children with Down syndrome
We’re studying how early thinking skills develop in young children with Down syndrome and what factors influence severe intellectual disability later on.
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Including Adults with Intellectual Disability in Precision Medicine
This grant funds a study to help adults with intellectual disability participate more fully in precision medicine research and understand how to respect their consent and privacy while sharing useful health information.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.