Gene therapy for long QT syndrome type 2
Funded at Rhode Island Hospital · NIH-11054685 · Data updated 2026-11-12
This study is testing a new gene therapy to help people with long QT syndrome type 2 (LQT2) by using a special treatment that could fix the genetic issues causing heart rhythm problems, which might help keep your heart beating normally and reduce the risk of serious complications.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Rhode Island Hospital NIH-funded |
| Lab location | 1 site (Providence, United States) |
| Project start | April 1, 2024 |
| Project end | March 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $727,361 |
| NIH project number | 5R01HL166165-02 |
| Project ID | NIH-11054685 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with long QT syndrome type 2, particularly those with mutations in the KCNH2 gene.
✕Less likely to help
Patients with long QT syndrome types other than type 2 or those without a genetic basis for their arrhythmias may not benefit from this research.
What this research studies
This research investigates a novel gene therapy approach for treating long QT syndrome type 2 (LQT2), a genetic condition that can lead to serious heart rhythm problems. Using a specially developed rabbit model that mimics the human condition, the researchers aim to deliver a gene therapy that targets the underlying genetic mutations responsible for LQT2. The therapy involves using a viral vector to introduce a modified gene that can restore normal heart function and prevent dangerous arrhythmias. Patients may benefit from this innovative treatment if it proves effective in restoring normal heart rhythms and reducing the risk of sudden cardiac death.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that significantly reduces the risk of life-threatening arrhythmias in patients with LQT2.
How similar studies have gone: This approach is novel and has not been extensively tested in humans, although gene therapy has shown promise in other genetic conditions.
Where this research is happening
-
Rhode Island Hospital
Providence, United States Active
Researchers
- Principal investigator: Koren, Gideon — Rhode Island Hospital
- Study coordinator: Koren, Gideon
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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.