Bait-and-switch gene therapy for heart muscle diseases
Official title: The "bait and switch" gene therapy for cardiomyopathies
Funded at Case Western Reserve University · NIH-11343872 · Data updated 2026-11-12
A heart-focused gene therapy approach uses AAV vectors redirected to the heart to treat conditions like hypertrophic cardiomyopathy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Case Western Reserve University NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project start | July 1, 2026 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $1,532,398 |
| NIH project number | 1R01HL185525-01 |
| Project ID | NIH-11343872 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are adults with genetic cardiomyopathies or heart failure due to cardiomyopathy who may be eligible for future gene therapy trials.
✕Less likely to help
Patients without genetic cardiomyopathies or those who cannot undergo gene therapy might not benefit.
What this research studies
This project aims to make gene therapy for heart diseases safer and more effective by rerouting AAV vectors to the heart and reducing liver exposure. Researchers will test how different nanoparticle designs affect delivery to the heart, and they will study whether this approach can reverse disease features in mouse models and assess translatability in pig hearts. If successful, the work could pave the way for a one-day clinical translation that preserves heart function with lower overall vector doses.
Why it matters
Potential benefit: If successful, patients with genetic cardiomyopathies could benefit from more effective gene therapies that require smaller doses and fewer side effects.
How similar studies have gone: Prior work showed preclinical promise for targeted AAV delivery, with ongoing challenges in translating to humans; this project builds on that foundation with a novel delivery strategy.
Where this research is happening
-
Case Western Reserve University
Cleveland, United States Active
Researchers
- Principal investigator: Maiseyeu, Andrei — Case Western Reserve University
- Study coordinator: Maiseyeu, Andrei
Related research
Other NIH-funded projects on the same topics.
-
Inflammatory and Genetic Heart Disease: Using Metabolic Clues to Guide Diagnosis and Therapy
Aims to improve diagnosis and treatment decisions for people with complex heart muscle diseases by combining metabolic data, genetics, and advanced imaging.
-
Biological Basis of cMyBP-C Cardiomyopathies and How Variants Cause Disease
This grant studies how genetic changes in a heart protein called cMyBP-C lead to cardiomyopathy, aiming to connect a person’s specific variant to their risk and potential therapies.
-
How extra calcium in heart cell powerhouses affects mitochondrial heart disease in children
This project looks at whether increased calcium entry into mitochondria helps heart cells make more energy in children with mitochondrial cardiomyopathy.
-
Genetic heart muscle problems: how tiny changes in muscle proteins affect your heart
A research study from the University of Washington aims to understand how genetic changes in heart muscle proteins affect heart function and how to predict which patients might respond to future treatments.
-
How mutations affect heart muscle function at the tiny level
Researchers are studying how heart muscle proteins interact at the smallest scale to understand why certain genetic changes lead to heart muscle disease.
-
Thin-filament heart muscle disease: causes and ways to block it
This project looks at how small changes in heart muscle proteins cause inherited cardiomyopathy and seeks ways to stop those changes from damaging the heart.
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.