Developing a new method to treat cystic fibrosis caused by specific genetic mutations
Official title: Combinatorial design of nonviral base editing systems for treating cystic fibrosis with nonsense mutations
Funded at University of Toronto · NIH-11158740 · Data updated 2026-11-12
This study is exploring a new way to help people with cystic fibrosis by using special tools to fix genetic mistakes in their cells, and it will test how well this works and how safe it is in mice before considering it for humans.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Toronto NIH-funded |
| Lab location | 1 site (Toronto, Canada) |
| Project start | August 1, 2024 |
| Project end | April 30, 2029 |
| Funding year | 2025 |
| Award (this year) | $243,000 |
| NIH project number | 5R01HL174773-02 |
| Project ID | NIH-11158740 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are cystic fibrosis patients who have been diagnosed with nonsense mutations in the CFTR gene.
✕Less likely to help
Patients with cystic fibrosis who do not have nonsense mutations or those with other types of genetic mutations may not benefit from this research.
What this research studies
This research focuses on treating cystic fibrosis (CF), a genetic disorder affecting the lungs and pancreas, particularly targeting nonsense mutations that lead to non-functional proteins. The approach involves using adenine base editors to correct these mutations, restoring the production of functional proteins. To ensure effective delivery of these editors to lung cells, specially designed lipid nanoparticles will be developed. The research includes testing these methods in a mouse model to evaluate their safety and effectiveness.
Why it matters
Potential benefit: If successful, this research could provide a targeted therapy for cystic fibrosis patients with nonsense mutations, potentially improving their health outcomes significantly.
How similar studies have gone: While the approach of using base editing for genetic disorders is relatively novel, similar methodologies have shown promise in other genetic conditions, indicating potential for success.
Where this research is happening
-
University of Toronto
Toronto, Canada Active
Researchers
- Principal investigator: Li, Bowen — University of Toronto
- Study coordinator: Li, Bowen
Related research
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-
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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.