Delivering healthy mitochondria to help people with Leigh syndrome
Harnessing mitochondria transfer pathways to ameliorate Leigh Syndrome-like disease
This project is seeing if giving healthy mitochondria can reduce brain damage and improve outcomes for people with Leigh syndrome.
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 ID | NIH-11127762 on NIH RePORTER |
What this research studies
Researchers are using a mouse model that mimics Leigh syndrome and giving purified healthy mitochondria into the bloodstream to see whether damaged brain cells and immune cells take them up and recover. Early lab work showed treated mice lived longer and had much less neurodegeneration, so the team will map how mitochondria move between cells, which cell types capture them, and where they travel in the body. The work will test different delivery schedules and analyze changes in cell metabolism and behavior in the brain and immune system. Those findings will guide whether this approach could move toward human testing for Leigh syndrome and related mitochondrial diseases.
Who could benefit from this research
Good fit: Future trials would most likely enroll people with genetically confirmed Leigh syndrome or closely related inherited mitochondrial complex I disorders.
Not a fit: People with unrelated neurological diseases, irreversible late-stage brain injury, or conditions that prevent mitochondrial uptake may not benefit from this approach.
Why it matters
Potential benefit: If successful, this could point to a new therapy that restores cellular energy and slows or prevents brain damage in children with Leigh syndrome.
How similar studies have performed: Related preclinical work, including the investigators' own mouse studies, has shown promising rescue of cell metabolism and improved survival, but human testing has not yet been done.
Where this research is happening
Saint Louis, United States
- Washington University — Saint Louis, United States (Active)
Researchers
- Principal investigator: Brestoff, Jonathan R — Washington University
- Study coordinator: Brestoff, Jonathan R
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 below.