Finding ways to increase titin protein levels to treat heart disease.
Official title: Development of strategies to enhance titin (TTN) expression and treat dilated cardiomyopathy caused by TTN haploinsufficiency
Funded at Brigham and Women's Hospital · NIH-11047743 · Data updated 2026-11-12
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 start | April 1, 2023 |
| Project end | March 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $169,020 |
| NIH project number | 5K08HL164885-03 |
| Project ID | NIH-11047743 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with dilated cardiomyopathy due to mutations in the titin gene.
✕Less likely to help
Patients with dilated cardiomyopathy caused by factors other than titin mutations may not benefit from this research.
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.
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 gone: 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
-
Brigham and Women's Hospital
Boston, United States Active
Researchers
- Principal investigator: Kim, Yuri — Brigham and Women's Hospital
- Study coordinator: Kim, Yuri
Related research
Other NIH-funded projects on the same topics.
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Discovery of rare genetic variants linked to dilated cardiomyopathy using big biobank data
A large-scale genetic study will look for rare gene variants that affect dilated cardiomyopathy to improve how we diagnose and manage the condition.
-
Understanding how muscle ribosome mutations cause severe neonatal dilated cardiomyopathy and ways to treat it
This grant focuses on a rare genetic heart condition in newborns caused by mutations in a muscle-specific ribosomal protein, with hopes to find new ways to diagnose and treat it.
-
Unlocking how rare genetic changes cause dilated cardiomyopathy
A large study will look at genetic data from many people to understand which rare variants affect dilated cardiomyopathy and how this changes risk and treatment.
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Role of Lmod2 in heart muscle and dilated cardiomyopathy
This NIH grant explores how a heart-specific protein (Lmod2) helps heart cells contract properly and how its problems can cause dilated cardiomyopathy in infants and children.
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Targeting DNA Damage Pathways in LMNA-Related Heart Disease
A study to understand how damaged DNA and inflammatory signals contribute to dilated cardiomyopathy caused by LMNA mutations and explore therapies that can protect the heart.
-
Using miRNA to find new treatment paths for dilated cardiomyopathy
Researchers are studying heart-muscle disease (dilated cardiomyopathy) to discover miRNA-based clues that could lead to targeted therapies for patients.
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.