Using non-invasive imaging to evaluate treatments for Leigh Syndrome in mice
Official title: Non-invasive Imaging for Therapeutic Efficacy (NITE) in Ndufs4 Mouse Model of Leigh Syndrome
Funded at University of Pittsburgh at Pittsburgh · NIH-11115024 · Data updated 2026-11-12
This study is looking at new ways to safely track how well treatments work for children with Leigh Syndrome by using special imaging techniques in mice, so we can better understand how different parts of the brain respond to therapy without any painful procedures.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project start | June 1, 2025 |
| Project end | May 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $416,544 |
| NIH project number | 1R21NS142555-01 |
| Project ID | NIH-11115024 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with Leigh Syndrome or those with genetic defects related to mitochondrial dysfunction.
✕Less likely to help
Patients with other forms of neurodegenerative diseases not related to mitochondrial dysfunction may not benefit from this research.
What this research studies
This research focuses on Leigh Syndrome, a severe neurodegenerative disease that primarily affects children. It aims to develop non-invasive imaging techniques to assess the effectiveness of potential therapies in a mouse model of the disease. By avoiding invasive procedures, the study seeks to provide a more accurate and humane way to monitor treatment responses over time. The research will explore how different brain regions respond to therapies, which is crucial given the multi-system nature of Leigh Syndrome.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for Leigh Syndrome, improving outcomes for affected children.
How similar studies have gone: While there have been various studies on Leigh Syndrome, the specific approach of using non-invasive imaging for therapeutic efficacy is relatively novel and has not been extensively tested.
Where this research is happening
-
University of Pittsburgh at Pittsburgh
Pittsburgh, United States Active
Researchers
- Principal investigator: Wu, Yijen Lin — University of Pittsburgh at Pittsburgh
- Study coordinator: Wu, Yijen Lin
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.