Understanding how frataxin is turned back on in Friedreich’s ataxia
Official title: Probing mechanisms of frataxin repression in Friedreich’s ataxia
Funded at St. Jude Children's Research Hospital · NIH-11376716 · Data updated 2026-11-12
A patient-friendly look at a Promising approach that aims to turn on the frataxin gene in people with Friedreich’s ataxia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project start | April 1, 2018 |
| Project end | March 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $696,710 |
| NIH project number | 2R01NS108376-12 |
| Project ID | NIH-11376716 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with Friedreich’s ataxia or cells derived from such patients.
✕Less likely to help
Healthy individuals without FRDA are unlikely to benefit directly.
What this research studies
This project explores genetic tools called SynGRs that may reactivate the frataxin gene in patient-derived cells, with the goal of restoring normal protein levels and reducing disease impact. Researchers will study how these gene regulators change the cell’s epigenetic landscape and DNA structures to overcome gene silencing. By testing next-generation SynGRs in disease-relevant cell types like cardiac muscle cells and neurons, the work aims to move toward precision, customized therapies for FRDA. The research is conducted using cells from patients to ensure findings are relevant to those living with the condition.
Why it matters
Potential benefit: If successful, this work could lead to gene-based therapies that restore frataxin levels and potentially lessen neurological and cardiac symptoms.
How similar studies have gone: Previous work shows that targeted gene regulators can reactivate silenced genes in cell models, but translating to FRDA is still early and promising.
Where this research is happening
-
St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Ansari, Aseem Z — St. Jude Children's Research Hospital
- Study coordinator: Ansari, Aseem Z
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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.