Gene therapy for FLNC-related heart disease
Official title: Gene therapy for FLNC haploinsufficiency cardiomyopathy
Funded at University of California, San Diego · NIH-11427270 · Data updated 2026-11-12
A patient-focused overview of testing gene-delivery methods to restore FLNC protein in people with FLNC-related cardiomyopathy.
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 | August 25, 2026 |
| Project end | May 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $795,900 |
| NIH project number | 1R01HL187977-01 |
| Project ID | NIH-11427270 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with FLNC haploinsufficiency-related cardiomyopathy or related inherited dilated/arrhythmogenic cardiomyopathies.
✕Less likely to help
People without FLNC haploinsufficiency or those not eligible for gene-delivery strategies may not benefit.
What this research studies
This project explores delivering a large FLNC gene reliably to heart cells using advanced AAV-based strategies, aiming to restore the missing protein in adults with FLNC-related cardiomyopathy. Researchers will compare multi-vector delivery approaches to reconstitute the full FLNC gene and evaluate how well the restored protein supports heart cell structure and function. The study will focus on identifying safe and effective ways to get a functional FLNC protein into heart tissue, with attention to potential arrhythmia risk reduction and disease progression. The work is designed to build toward therapies that could complement current treatments and address the underlying genetic cause.
Why it matters
Potential benefit: If successful, patients with FLNC haploinsufficiency could benefit from a therapy that replenishes FLNC protein and slows or halts disease progression.
How similar studies have gone: Prior approaches have shown progress with multi-vector delivery, but translating to safe, effective heart delivery remains challenging.
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.