Gene editing to treat Friedreich's Ataxia
Project 2: Therapeutic Gene Editing for Friedreich's Ataxia
['FUNDING_OTHER'] · JACKSON LABORATORY · NIH-11089354
This study is exploring a new way to boost a protein called frataxin in people with Friedreich's Ataxia, a condition that affects the nervous system, by using a special delivery method to help improve their health and possibly slow down or even reverse the disease.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | JACKSON LABORATORY (nih funded) |
| Locations | 1 site (BAR HARBOR, UNITED STATES) |
| Trial ID | NIH-11089354 on ClinicalTrials.gov |
What this research studies
This research focuses on developing a gene editing approach to increase the levels of frataxin protein in patients with Friedreich's Ataxia (FRDA), a genetic disorder that leads to progressive damage to the nervous system and other organs. The project aims to utilize adeno-associated viral vectors to deliver the necessary genetic material to affected tissues, potentially halting or reversing disease progression. By targeting the underlying genetic cause of FRDA, the research seeks to provide a more effective treatment option than current therapies, which only address symptoms.
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 early symptoms of the disease.
Not a fit: Patients with Friedreich's Ataxia who are in advanced stages of the disease or have atypical presentations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could significantly improve the quality of life and longevity for patients with Friedreich's Ataxia by addressing the root cause of the disease.
How similar studies have performed: Previous research has shown promise in using gene editing techniques for other genetic disorders, indicating potential for success in this novel approach for Friedreich's Ataxia.
Where this research is happening
BAR HARBOR, UNITED STATES
- JACKSON LABORATORY — BAR HARBOR, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LIU, DAVID R — JACKSON LABORATORY
- Study coordinator: LIU, DAVID R
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.