Precision therapy to boost SynGAP for SYNGAP1-related autism
Official title: Preclinical development of a precision therapy for a monogenic mental health disorder
Funded at University of Florida · NIH-11301912 · Data updated 2026-11-12
This project is creating small, brain-penetrant drugs to raise SynGAP protein levels for people with SYNGAP1-related developmental disability and autism.
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 | 2026 |
| Award (this year) | $1,781,729 |
| NIH project number | 5U01MH136567-03 |
| Project ID | NIH-11301912 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be people with a confirmed pathogenic SYNGAP1 variant (SYNGAP1 haploinsufficiency / SYNGAP1-DEE).
✕Less likely to help
People without SYNGAP1 mutations or whose neurodevelopmental symptoms come from other genetic or non-genetic causes are unlikely to benefit from this approach.
What this research studies
Researchers are using a lab screening platform to find small molecules that increase SynGAP protein in neurons derived from models relevant to SYNGAP1 haploinsufficiency. Promising compounds will be optimized and tested in cell and animal models to check brain penetration, dosing, and safety before picking preclinical drug candidates. The team aims for orally deliverable molecules that can be adjusted by dose and could eventually be tested in people with genetically confirmed SYNGAP1 variants. This work focuses on a specific genetic cause of autism and related developmental and epileptic encephalopathy to create a precision treatment approach.
Why it matters
Potential benefit: If successful, these drugs could raise SynGAP levels in the brain and potentially improve cognition, social function, and seizure control in people with SYNGAP1-DEE.
How similar studies have gone: Related approaches to boost deficient proteins with small molecules are experimental; some have shown promise in lab and animal models but none are yet proven treatments for SYNGAP1 patients.
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.