Understanding how a protein affects brain function in Fragile X syndrome
Official title: Fragile X messenger ribonucleoprotein-1 mediated regulation of the Clathrin-Associated Adaptor Complex Protein-2 (AP2)
Funded at Emory University · NIH-10817391 · Data updated 2026-11-12
This study is looking at how a protein called FMRP helps control important processes in brain cells for people with Fragile X syndrome, with the hope of finding new ways to understand and address the challenges they face.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project start | January 1, 2024 |
| Project end | December 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $156,500 |
| NIH project number | 1R03MH135518-01 |
| Project ID | NIH-10817391 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Fragile X syndrome or related intellectual disabilities.
✕Less likely to help
Patients without Fragile X syndrome or those with other unrelated neurological conditions may not benefit from this research.
What this research studies
This research investigates the role of a specific protein, FMRP, in regulating the endocytic process, which is crucial for the proper functioning of neurons in individuals with Fragile X syndrome. By exploring how FMRP influences the surface expression of proteins involved in synaptic communication, the study aims to uncover new mechanisms that may contribute to the symptoms of Fragile X. The approach includes examining the translational regulation of proteins by FMRP, focusing on a protein complex known as AP2. This could lead to a better understanding of the molecular underpinnings of Fragile X syndrome and its associated challenges.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic strategies for improving brain function in patients with Fragile X syndrome.
How similar studies have gone: While the specific focus on FMRP's role in endocytosis is novel, related research has shown promising results in understanding the molecular mechanisms of Fragile X syndrome.
Where this research is happening
-
Emory University
Atlanta, United States Active
Researchers
- Principal investigator: Bassell, Gary J — Emory University
- Study coordinator: Bassell, Gary 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.