Understanding mitochondrial disease using advanced genetic tools
Official title: Modeling mtDNA Disease in vivo
Funded at University of Texas at Austin · NIH-10767530 · Data updated 2026-11-12
This study is looking at mitochondrial diseases, which can cause problems in different parts of the body, and it's using special techniques to create animal models that mimic these conditions, so researchers can better understand how changes in mitochondrial DNA lead to the symptoms people experience, with the hope of finding new treatments that could help patients like you.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Texas at Austin NIH-funded |
| Lab location | 1 site (Austin, United States) |
| Project start | September 16, 2024 |
| Project end | August 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $629,508 |
| NIH project number | 1R24OD035577-01 |
| Project ID | NIH-10767530 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with mitochondrial diseases or those exhibiting symptoms related to mitochondrial dysfunction.
✕Less likely to help
Patients with non-mitochondrial diseases or those without any mitochondrial-related symptoms may not benefit from this research.
What this research studies
This research focuses on mitochondrial diseases, which affect various bodily systems and have limited treatment options. By utilizing advanced genetic editing techniques, the study aims to create and analyze animal models that mimic human mitochondrial diseases. This approach will help researchers understand how genetic variations in mitochondrial DNA contribute to these diseases and their diverse symptoms. Patients may benefit from insights gained through this research, potentially leading to new therapeutic strategies.
Why it matters
Potential benefit: If successful, this research could lead to the development of effective treatments for mitochondrial diseases.
How similar studies have gone: Previous research has shown promise in using genetic editing tools for studying mitochondrial diseases, indicating a potential for success in this approach.
Where this research is happening
-
University of Texas at Austin
Austin, United States Active
Researchers
- Principal investigator: Ekker, Stephen Carl — University of Texas at Austin
- Study coordinator: Ekker, Stephen Carl
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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.