Developing a targeted therapy for a genetic mental health disorder
Official title: Preclinical development of a precision therapy for a monogenic mental health disorder
Funded at University of Florida · NIH-11075913 · Data updated 2026-11-12
This study is looking for new medications to help people with SYNGAP1-related developmental and behavioral challenges by finding small molecules that boost a key brain protein that they often lack, with the hope of creating more effective treatments tailored to their specific needs.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | April 16, 2024 |
| Project end | January 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $1,535,513 |
| NIH project number | 5U01MH136567-02 |
| Project ID | NIH-11075913 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with SYNGAP1-related developmental disorders, particularly those experiencing significant behavioral and cognitive challenges.
✕Less likely to help
Patients with mental health disorders not linked to SYNGAP1 mutations may not benefit from this research.
What this research studies
This research focuses on creating new drug candidates to treat SYNGAP1-related developmental and behavioral disorders. By using a novel screening platform, researchers aim to identify small molecules that can enhance the production of a crucial protein in the brain that is often deficient in affected individuals. The approach is designed to be precision-based, meaning it targets the specific genetic causes of the disorder, potentially leading to more effective treatments. The research will involve preclinical testing to ensure safety and efficacy before moving to human trials.
Why it matters
Potential benefit: If successful, this research could provide a new, effective treatment option for individuals with SYNGAP1-related disorders, improving their mental health and social functioning.
How similar studies have gone: Previous research has successfully identified small molecules that enhance protein expression in similar genetic disorders, suggesting a promising avenue for this approach.
Where this research is happening
-
University of Florida
Gainesville, United States Active
Researchers
- Principal investigator: Miller, Courtney a — University of Florida
- Study coordinator: Miller, Courtney a
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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.