Reducing stress in mitochondria to treat neuromuscular disorders
Official title: Targeting NADH-Reductive Stress in Mitochondrial Disease
Funded at Emory University · NIH-11131135 · Data updated 2026-11-12
This study is exploring a new tool called LOXCAT that could help improve energy production in the cells of people with inherited neuromuscular disorders, like Leigh syndrome, by reducing stress in their mitochondria and helping them feel better.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project start | July 3, 2024 |
| Project end | April 30, 2029 |
| Funding year | 2025 |
| Award (this year) | $405,368 |
| NIH project number | 5R01NS133344-02 |
| Project ID | NIH-11131135 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with inherited mitochondrial disorders, particularly those with mutations affecting the electron transport chain.
✕Less likely to help
Patients with mitochondrial diseases not related to NADH-reductive stress or those with other unrelated conditions may not benefit from this research.
What this research studies
This research focuses on understanding and addressing mitochondrial dysfunction, which is linked to various inherited neuromuscular disorders. The team is investigating a novel enzyme tool called LOXCAT, designed to alleviate a specific type of stress in mitochondria known as NADH-reductive stress. By using this tool, they aim to improve the biochemical balance in affected tissues, potentially leading to better outcomes for patients with conditions like Leigh syndrome. The approach involves both laboratory studies and animal models to evaluate the effectiveness of LOXCAT in reducing harmful metabolites and restoring normal cellular function.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic options for patients suffering from severe mitochondrial diseases.
How similar studies have gone: While targeting NADH-reductive stress is a relatively novel approach, preliminary studies have shown promise in similar biochemical interventions for mitochondrial dysfunction.
Where this research is happening
-
Emory University
Atlanta, United States Active
Researchers
- Principal investigator: Patgiri, Anupam — Emory University
- Study coordinator: Patgiri, Anupam
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.