Using CRISPR technology to improve sensory and motor skills in a mouse model of autism
Official title: CRISPRa-based rescue of sensorimotor deficits in the Scn2a+/- mouse model of autism spectrum disorder
Funded at University of California, San Francisco · NIH-11059916 · Data updated 2026-11-12
This study is exploring a new way to boost a gene linked to autism in mice to see if it can help improve their sensory and motor skills, which could lead to better treatments for people with autism.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | April 1, 2024 |
| Project end | January 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $571,865 |
| NIH project number | 5R01MH136475-02 |
| Project ID | NIH-11059916 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for participation or benefit from this research would be individuals with autism spectrum disorder linked to SCN2A mutations.
✕Less likely to help
Patients without SCN2A mutations or those with other forms of autism may not benefit from this specific research.
What this research studies
This research investigates the use of CRISPR activation technology to enhance the function of a gene associated with autism, specifically in a mouse model that mimics certain aspects of the disorder. The study focuses on restoring the expression of the SCN2A gene, which is crucial for proper neuronal function, to alleviate deficits in sensory processing and motor skills. By applying this innovative genetic approach, the researchers aim to improve both cellular and behavioral outcomes in these mice, potentially leading to new therapeutic strategies for autism. The research will involve detailed assessments of the effects of CRISPRa on brain function and behavior in the mouse model.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that improve sensory and motor functions in individuals with autism spectrum disorder.
How similar studies have gone: Other research has shown promise in using CRISPR technology for genetic interventions, suggesting potential success for this approach in autism treatment.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Bender, Kevin J — University of California, San Francisco
- Study coordinator: Bender, Kevin J
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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.