Using a new method to edit genes for treating hypertrophic cardiomyopathy
Chemically inducible split base editors for precise and controllable in vivo genome editing
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 ID | NIH-11050651 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with familial hypertrophic cardiomyopathy due to MYBPC3 mutations.
Not a fit: Patients without genetic mutations in MYBPC3 or those with non-familial forms of hypertrophic cardiomyopathy may not benefit from this research.
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 performed: Previous research has shown success with similar gene editing approaches, particularly in animal models, indicating potential for human application.
Where this research is happening
Philadelphia, United States
- University of Pennsylvania — Philadelphia, United States (Active)
Researchers
- Principal investigator: Gao, Xue — University of Pennsylvania
- Study coordinator: Gao, Xue
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.