Understanding and reversing brain dysfunction linked to autism and intellectual disability
Novel SETD5-based Molecular Mechanisms and Therapeutic Tools to Understand and Revert Neuronal Dysfunction Associated with Intellectual disability and Autism
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 ID | NIH-11010745 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are children aged two years or younger who have been diagnosed with autism spectrum disorder or intellectual disability.
Not a fit: 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.
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 performed: 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
La Jolla, United States
- 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.
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 below.