Investigating brain development in fragile X syndrome using human cell models
Project 1
This study is looking at fragile X syndrome by using brain cells from patients to see how a certain protein affects brain development, with the hope of finding better treatments for those with the condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project ID | NIH-11179532 on NIH RePORTER |
What this research studies
This research focuses on understanding fragile X syndrome (FXS) by using human-derived neural models to study the role of a specific protein involved in brain development. Researchers will create both 2D and 3D models from patients' cells to explore how these cells behave and develop compared to healthy cells. The study aims to identify molecular and cellular changes that occur in FXS, which could lead to better treatment strategies. By validating findings from animal models in human cells, the research seeks to bridge the gap between laboratory findings and clinical applications.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with fragile X syndrome, particularly those over 21 years old.
Not a fit: Patients without a diagnosis of fragile X syndrome or those under 21 years old may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic strategies for individuals with fragile X syndrome.
How similar studies have performed: Previous research using similar human cell models has shown promise in understanding genetic disorders, indicating potential for success in this approach.
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.