Understanding the causes of familial dilated cardiomyopathy to improve treatment options
Unraveling the pathogenesis of familial dilated cardiomyopathy towards precision medicine
This study is looking at a heart condition called familial dilated cardiomyopathy (DCM) and is testing how a protein called ATF4 can help improve heart function, with the hope of finding new treatments for people with DCM, no matter what genetic changes they have.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project ID | NIH-11212025 on NIH RePORTER |
What this research studies
This research investigates familial dilated cardiomyopathy (DCM), a serious heart condition that can lead to heart failure. The team is exploring the role of a specific protein, ATF4, which helps cells respond to stress, and how it can be targeted to develop new therapies. By using patient-derived cells and advanced gene therapy techniques, the researchers aim to find ways to improve heart function in individuals with DCM, regardless of the specific genetic mutations they carry. The study involves both laboratory experiments and potential treatments in living models.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with familial dilated cardiomyopathy, particularly those with specific genetic mutations affecting heart function.
Not a fit: Patients with non-familial forms of dilated cardiomyopathy or those without a genetic basis for their condition 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 familial dilated cardiomyopathy.
How similar studies have performed: Previous research has shown promise in targeting stress response pathways for heart conditions, suggesting that this approach could be effective, although the specific application of ATF4 in DCM is relatively novel.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Karakikes, Ioannis — Stanford University
- Study coordinator: Karakikes, Ioannis
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.