Developing smarter gene therapies that target specific tissues for safer treatment.
Official title: Smarter gene therapies: alternative splicing cassettes for tissue-and self-regulated cargo expression
Funded at University of Florida · NIH-10997419 · Data updated 2026-11-12
This study is looking at new ways to make gene therapy safer by making sure that helpful genes are only active in muscles and not in the heart, which could lead to better treatments for genetic diseases with fewer side effects for patients.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | December 8, 2023 |
| Project end | November 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $457,978 |
| NIH project number | 5R01NS132538-02 |
| Project ID | NIH-10997419 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic disorders that could benefit from targeted gene therapy, particularly those affecting skeletal muscle.
✕Less likely to help
Patients with genetic conditions that do not involve skeletal muscle or those who have contraindications for gene therapy may not benefit from this research.
What this research studies
This research focuses on improving gene therapy techniques by using alternative splicing to control where therapeutic genes are expressed in the body. By creating gene expression cassettes that are regulated based on tissue type, the goal is to minimize potential toxicity in non-target tissues. The research will specifically explore how to restrict gene expression to skeletal muscle while avoiding the heart, which could lead to safer treatments for genetic diseases. Patients may benefit from more effective and targeted gene therapies that reduce side effects.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for patients with genetically defined diseases.
How similar studies have gone: Other research has shown promise in using tissue-specific gene expression strategies, indicating that this approach could be a significant advancement in gene therapy.
Where this research is happening
-
University of Florida
Gainesville, United States Active
Researchers
- Principal investigator: Wang, Eric T — University of Florida
- Study coordinator: Wang, Eric T
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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.