Investigating the role of microglia in Friedreich's ataxia and potential therapies
Microgial contribution and therapeutic potential in Friedreich's ataxia
This study is looking at a new way to help people with Friedreich's ataxia by using healthy stem cells to fix the problems caused by the disease, with the hope of finding a treatment that could make life better for patients.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11001193 on NIH RePORTER |
What this research studies
This research focuses on Friedreich's ataxia (FRDA), a genetic disorder that leads to severe neurological and muscular complications. The team is exploring a novel therapy involving the transplantation of healthy hematopoietic stem and progenitor cells (HSPCs) to correct the underlying genetic defect. By studying how these cells can rescue damaged neurons through microglial support, the research aims to develop a potential treatment for this currently untreatable condition. Patients may benefit from insights into new therapeutic strategies that could improve their quality of life.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Friedreich's ataxia, particularly those who are experiencing significant neurological and muscular symptoms.
Not a fit: Patients with Friedreich's ataxia who are not eligible for stem cell transplantation or those with advanced disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment for Friedreich's ataxia, potentially reversing neurological and muscular damage.
How similar studies have performed: While the approach of using HSPC transplantation is promising, the specific application to Friedreich's ataxia is relatively novel and has not been extensively tested in clinical settings.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Cherqui, Stephanie — University of California, San Diego
- Study coordinator: Cherqui, Stephanie
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.