Using mitochondria transfer to improve Leigh Syndrome treatment
Official title: Harnessing mitochondria transfer pathways to ameliorate Leigh Syndrome-like disease
Funded at Washington University · NIH-10980991 · Data updated 2026-11-12
This study is looking at a new way to help kids with Leigh Syndrome by transferring healthy mitochondria into their cells, which could boost their energy and improve brain function, and so far, it’s shown some encouraging results in animal tests.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | July 1, 2024 |
| Project end | June 30, 2029 |
| Funding year | 2024 |
| Award (this year) | $596,783 |
| NIH project number | 1R01NS134932-01A1 |
| Project ID | NIH-10980991 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with Leigh Syndrome or other inherited mitochondrial diseases.
✕Less likely to help
Patients with mitochondrial diseases that do not respond to mitochondrial transfer or those with advanced stages of Leigh Syndrome may not benefit from this research.
What this research studies
This research investigates how transferring mitochondria between cells could potentially treat Leigh Syndrome, a severe inherited mitochondrial disease that primarily affects children. The approach involves administering purified mitochondria to affected cells, which may help restore their energy metabolism and improve neurological function. The researchers have observed promising results in animal models, where the treatment increased lifespan and reduced neurological symptoms. The goal is to explore this innovative method as a new therapeutic strategy for Leigh Syndrome and similar mitochondrial disorders.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment option for children suffering from Leigh Syndrome, potentially improving their quality of life and extending their lifespan.
How similar studies have gone: Other research has shown promising results with similar mitochondrial transfer approaches, indicating potential for success in this novel application.
Where this research is happening
-
Washington University
Saint Louis, United States Active
Researchers
- Principal investigator: Brestoff, Jonathan R — Washington University
- Study coordinator: Brestoff, Jonathan R
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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.