Understanding mitochondrial disease using advanced genetic tools
Modeling mtDNA Disease in vivo
['FUNDING_OTHER'] · UNIVERSITY OF TEXAS AT AUSTIN · NIH-10767530
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
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF TEXAS AT AUSTIN (nih funded) |
| Locations | 1 site (AUSTIN, UNITED STATES) |
| Trial ID | NIH-10767530 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with mitochondrial diseases or those exhibiting symptoms related to mitochondrial dysfunction.
Not a fit: Patients with non-mitochondrial diseases or those without any mitochondrial-related symptoms may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the development of effective treatments for mitochondrial diseases.
How similar studies have performed: 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
AUSTIN, UNITED STATES
- 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
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.
Conditions: Animal Disease Models