Targeting the frataxin gene to treat Friedreich's ataxia
Official title: Frataxin Gene-Targeted GAA Repeat Contraction Through A Vault Bionanoparticle-Mediated CRISPR/dCas9 System In Friedreich's Ataxia
Funded at Florida International University · NIH-11036612 · Data updated 2026-11-12
This study is exploring a new way to help people with Friedreich's ataxia by using special techniques to fix the gene that causes the condition, which could lead to improvements in their symptoms.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Florida International University NIH-funded |
| Lab location | 1 site (Miami, United States) |
| Project start | March 1, 2023 |
| Project end | February 28, 2026 |
| Funding year | 2024 |
| Award (this year) | $117,615 |
| NIH project number | 3R03NS132097-02S1 |
| Project ID | NIH-11036612 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Friedreich's ataxia who have the genetic markers associated with the disease.
✕Less likely to help
Patients without Friedreich's ataxia or those with other forms of ataxia may not receive any benefit from this research.
What this research studies
This research focuses on developing a novel approach to treat Friedreich's ataxia by targeting the frataxin gene, which is affected by expanded GAA repeats. The team will utilize advanced techniques involving CRISPR technology and vault nanoparticles to promote the contraction of these GAA repeats in neural cells. By inhibiting specific histone modifications, the researchers aim to enhance DNA repair mechanisms and activate the frataxin gene, potentially reversing some of the disease's effects. Patients may benefit from this innovative strategy that seeks to address the underlying genetic cause of the condition.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that addresses the root cause of Friedreich's ataxia, potentially improving patients' symptoms and quality of life.
How similar studies have gone: While the approach of using CRISPR technology for gene editing is gaining traction, this specific application targeting Friedreich's ataxia is novel and has not been extensively tested in clinical settings.
Where this research is happening
-
Florida International University
Miami, United States Active
Researchers
- Principal investigator: Liu, Yuan — Florida International University
- Study coordinator: Liu, Yuan
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A study in adults and children with Friedreich’s ataxia will look at how changes in GAA repeat length inside the FXN gene relate to the disease’s start and progression.
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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.