Using a new method to edit genes for treating hypertrophic cardiomyopathy
Official title: Chemically inducible split base editors for precise and controllable in vivo genome editing
Funded at University of Pennsylvania · NIH-11050651 · Data updated 2026-11-12
This study is exploring a new way to fix the gene that causes hypertrophic cardiomyopathy (HCM), a heart condition that affects many adults, using a special tool that could lead to a one-time treatment to tackle the problem at its source instead of just easing the symptoms.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | February 1, 2025 |
| Project end | November 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $667,704 |
| NIH project number | 1R01HL173243-01A1 |
| Project ID | NIH-11050651 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with familial hypertrophic cardiomyopathy due to MYBPC3 mutations.
✕Less likely to help
Patients without genetic mutations in MYBPC3 or those with non-familial forms of hypertrophic cardiomyopathy may not benefit from this research.
What this research studies
This research focuses on developing a precise and controllable method for editing genes associated with hypertrophic cardiomyopathy (HCM), a heart condition affecting many adults. The approach involves using a novel split base editor that can correct mutations in the MYBPC3 gene, which is responsible for familial HCM. By utilizing adeno-associated virus vectors for delivery, the researchers aim to achieve high efficiency in gene editing while minimizing unintended effects. This method could potentially provide a one-time treatment that addresses the root cause of the disease rather than just managing symptoms.
Why it matters
Potential benefit: If successful, this research could lead to a curative treatment for patients with hypertrophic cardiomyopathy caused by specific genetic mutations.
How similar studies have gone: Previous research has shown success with similar gene editing approaches, particularly in animal models, indicating potential for human application.
Where this research is happening
-
University of Pennsylvania
Philadelphia, United States Active
Researchers
- Principal investigator: Gao, Xue — University of Pennsylvania
- Study coordinator: Gao, Xue
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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.