Finding ways to increase titin protein levels to treat heart disease.
Development of strategies to enhance titin (TTN) expression and treat dilated cardiomyopathy caused by TTN haploinsufficiency
This study is looking at how changes in a specific gene can affect heart health in people with dilated cardiomyopathy, and they're using human stem cells to find ways to boost a protein that helps the heart work better, with the hope of discovering new treatments to keep hearts strong and healthy.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11047743 on NIH RePORTER |
What this research studies
This research focuses on dilated cardiomyopathy (DCM), a heart condition caused by mutations in the titin gene, which leads to reduced levels of the titin protein. The researchers are developing a model using human stem cells to better understand how these mutations affect heart function and to identify strategies to enhance titin expression. By studying these cells, they aim to discover potential therapies that could improve heart muscle function and prevent the progression to heart failure.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with dilated cardiomyopathy due to mutations in the titin gene.
Not a fit: Patients with dilated cardiomyopathy caused by factors other than titin mutations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that improve heart function and quality of life for patients with dilated cardiomyopathy.
How similar studies have performed: Previous research has shown promise in using genetic engineering approaches to enhance protein expression in similar cardiac conditions, indicating potential for success in this area.
Where this research is happening
Boston, United States
- Brigham and Women's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Kim, Yuri — Brigham and Women's Hospital
- Study coordinator: Kim, Yuri
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.