Targeting gene modifications to treat Friedreich’s Ataxia
Official title: A CRSIPR/dCas9-Targeted Histone Demethylation Induces GAA repeat contraction
Funded at Florida International University · NIH-10798294 · Data updated 2026-11-12
This study is exploring a new way to help people with Friedreich’s Ataxia by using a special tool called CRISPR to change certain genes, with the hope of boosting the production of a key protein that can improve symptoms and support nerve health.
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) | $73,750 |
| NIH project number | 5R03NS132097-02 |
| Project ID | NIH-10798294 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 whose condition is not linked to the GAA repeat expansion may not benefit from this research.
What this research studies
This research investigates a novel approach to treat Friedreich’s Ataxia (FRDA) by using a CRISPR-based method to target and modify specific genetic sequences responsible for the disease. The researchers aim to induce demethylation of histones associated with the frataxin gene, which is crucial for reducing the expanded GAA repeats that cause FRDA. By utilizing a modified CRISPR system, they will assess whether this technique can effectively upregulate the frataxin gene expression and potentially reverse some of the disease's effects in neural cells. This approach combines genetic engineering with cellular repair mechanisms to address the underlying cause of the disorder.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment for Friedreich’s Ataxia, potentially improving the quality of life for affected patients.
How similar studies have gone: While the use of CRISPR technology in gene editing is well-established, 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
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.