Understanding how a protein affects brain function in Fragile X syndrome
Fragile X messenger ribonucleoprotein-1 mediated regulation of the Clathrin-Associated Adaptor Complex Protein-2 (AP2)
['FUNDING_R03'] · EMORY UNIVERSITY · NIH-10817391
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
| Phase | ['FUNDING_R03'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | EMORY UNIVERSITY (nih funded) |
| Locations | 1 site (ATLANTA, UNITED STATES) |
| Trial ID | NIH-10817391 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Fragile X syndrome or related intellectual disabilities.
Not a fit: Patients without Fragile X syndrome or those with other unrelated neurological conditions may not benefit from this research.
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 performed: 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
ATLANTA, UNITED STATES
- EMORY UNIVERSITY — ATLANTA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BASSELL, GARY J — EMORY UNIVERSITY
- Study coordinator: BASSELL, GARY J
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.
Conditions: Brain Disorders, Encephalon Diseases, Intracranial CNS Disorders, Intracranial Central Nervous System Disorders, Brain Diseases