Gene therapy to treat ALPK3 cardiomyopathy
Official title: Gene Therapy for ALPK3 Cardiomyopathy Using MiniALPK3
Funded at University of California, San Diego · NIH-11134646 · Data updated 2026-11-12
This study is looking at a new gene therapy that uses a harmless virus to deliver a healthy version of the ALPK3 gene into heart cells, with the hope of helping people with ALPK3 cardiomyopathy improve their heart function and slow down the disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project start | July 15, 2024 |
| Project end | May 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $490,883 |
| NIH project number | 5R01HL175344-02 |
| Project ID | NIH-11134646 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with ALPK3 cardiomyopathy, particularly those experiencing early-onset dilated cardiomyopathy or related heart conditions.
✕Less likely to help
Patients with cardiomyopathy caused by other genetic mutations or non-genetic factors may not benefit from this specific research.
What this research studies
This research investigates the use of gene therapy to address ALPK3 cardiomyopathy, a condition caused by mutations in the ALPK3 gene that leads to heart disease. The approach involves using a specific type of virus, known as an adeno-associated virus (AAV), to deliver a healthy copy of the ALPK3 gene directly into heart cells. By studying mouse models that mimic the disease, researchers aim to understand how this therapy can restore normal heart function and potentially prevent the progression of the disease in patients.
Why it matters
Potential benefit: If successful, this research could provide a novel treatment option for patients suffering from ALPK3 cardiomyopathy, potentially improving heart function and quality of life.
How similar studies have gone: Previous research using AAV-based gene therapies has shown promising results in treating various cardiovascular diseases, indicating potential for success in this novel application.
Where this research is happening
-
University of California, San Diego
La Jolla, United States Active
Researchers
- Principal investigator: Chen, Ju — University of California, San Diego
- Study coordinator: Chen, Ju
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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.