Improving gene therapy for Friedreich’s ataxia
Immunomodulation Approaches to Improve Safety And Efficacy of Gene Therapy Treatment in Friedreich’s Ataxia
This study is looking at ways to make gene therapy safer and more effective for people with Friedreich’s ataxia by using a special delivery method to provide the missing gene, while also finding ways to reduce any immune reactions that could cause problems, so patients can benefit from the treatment without side effects.
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 ID | NIH-10925410 on NIH RePORTER |
What this research studies
This research focuses on enhancing the safety and effectiveness of gene therapy for Friedreich’s ataxia, a genetic disorder that affects movement and coordination. The approach involves using adeno-associated viral vectors to deliver the human frataxin gene, which is deficient in patients with this condition. The study aims to address the immune responses that can hinder the success of gene therapy, ensuring that patients can receive the treatment without adverse effects. By investigating immunomodulation strategies, the research seeks to improve patient outcomes and the longevity of the therapeutic effects.
Who could benefit from this research
Good fit: Ideal candidates for this research are children and young adults aged 0-11 years diagnosed with Friedreich’s ataxia.
Not a fit: Patients with Friedreich’s ataxia who are older than 11 years or those with other unrelated medical conditions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a safer and more effective gene therapy option for patients with Friedreich’s ataxia.
How similar studies have performed: Previous research has shown promise in using gene therapy for similar genetic disorders, indicating potential for success in this approach.
Where this research is happening
Gainesville, United States
- University of Florida — Gainesville, United States (Active)
Researchers
- Principal investigator: Corti, Manuela — University of Florida
- Study coordinator: Corti, Manuela
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.