Neural Mitochondria and Gene Editing
Funded at Harvard University · NIH-11444918 · Data updated 2026-11-12
Aims to understand how mitochondrial DNA mutations affect brain function and how gene editing can study or potentially fix them, focusing on people and models with mitochondrial-related brain issues.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Harvard University NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project start | September 1, 2026 |
| Project end | June 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $595,961 |
| NIH project number | 1R01NS141924-01A1 |
| Project ID | NIH-11444918 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with mitochondrial DNA mutations that affect the brain, or families carrying such mutations, may benefit from future advances stemming from this research.
✕Less likely to help
At present, there may be no direct benefit to most participants while the study progresses in basic mechanistic work.
What this research studies
This grant explores how mitochondrial DNA mutations contribute to brain dysfunction and development, using advanced gene-editing tools to study neural cells. Researchers will examine how different levels of mutant mtDNA (heteroplasmy) and cell types influence disease outcomes, with a focus on conditions like MELAS and related neurodevelopmental issues. By testing mitochondrial-targeted editing strategies, the project seeks to uncover mechanisms that could guide future therapies for patients affected by mitochondrial brain disorders.
Why it matters
Potential benefit: Better understanding of brain-related mitochondrial diseases could lead to new therapeutic approaches for patients.
How similar studies have gone: Emerging evidence shows mtDNA editing in cells is possible, but translating this to patient care remains in early stages.
Where this research is happening
-
Harvard University
Cambridge, United States Active
Researchers
- Principal investigator: Lee, Richard T — Harvard University
- Study coordinator: Lee, Richard T
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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.