New peptides targeting mitochondria to treat Barth syndrome
Novel mitochondria-targeting peptides (MTPs) as therapeutic intervention in Barth syndrome
This study is looking at new treatments for Barth syndrome, a genetic condition that affects the heart, by exploring how certain peptides can help fix problems in the mitochondria, which are the energy powerhouses of our cells, to improve heart health for those living with this condition.
Quick facts
| Grant type | R15 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cleveland State University NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project ID | NIH-11042916 on NIH RePORTER |
What this research studies
This research focuses on developing mitochondria-targeting peptides (MTPs) as a potential treatment for Barth syndrome, a serious genetic disorder that primarily affects the heart. The study aims to understand the underlying metabolic processes that lead to heart complications in patients with Barth syndrome, particularly those caused by mutations in the TAZ gene. By addressing the abnormalities in cardiolipin, a crucial component of mitochondrial function, the research seeks to create effective therapies that could improve heart health and overall outcomes for affected individuals.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Barth syndrome, particularly those experiencing cardiac issues.
Not a fit: Patients without Barth syndrome or those whose cardiac issues are unrelated to mitochondrial dysfunction may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve heart function and quality of life for patients with Barth syndrome.
How similar studies have performed: While there is ongoing research into mitochondrial therapies, this specific approach using MTPs for Barth syndrome is novel and has not been extensively tested.
Where this research is happening
Cleveland, United States
- Cleveland State University — Cleveland, United States (Active)
Researchers
- Principal investigator: Sandlers, Yana I — Cleveland State University
- Study coordinator: Sandlers, Yana I
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.