Developing new gene editing therapies for cystic fibrosis
Official title: Developing Gene Editing Therapeutics, Biodegradable Polymeric Delivery Vehicles, and High-throughput Platforms for the Treatment of Cystic Fibrosis- Supplement
Funded at University of Michigan at Ann Arbor · NIH-11265024 · Data updated 2026-11-12
This study is working on new gene editing treatments for people with cystic fibrosis who have rare gene changes, using special materials to deliver medicine directly to the lungs and testing these treatments in the lab to help improve care for CF and similar conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project start | July 1, 2023 |
| Project end | May 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $109,200 |
| NIH project number | 3R00HL151806-04S1 |
| Project ID | NIH-11265024 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with cystic fibrosis who have rare CFTR gene variants and are not responding to existing therapies.
✕Less likely to help
Patients with common CFTR variants who are already benefiting from existing highly effective modulator therapies may not receive additional benefits from this research.
What this research studies
This research focuses on creating innovative gene editing therapies specifically for patients with cystic fibrosis (CF) who have rare genetic variants. The team will develop biodegradable polymeric materials to effectively deliver therapeutic nucleic acids to the lungs, addressing a significant barrier in current treatments. Additionally, they will utilize advanced 3D culture models to screen and assess the efficacy of these therapies in a laboratory setting. This approach aims to enhance the clinical translation of promising treatments for CF and other genetic disorders.
Why it matters
Potential benefit: If successful, this research could provide new treatment options for cystic fibrosis patients who currently have limited or no effective therapies.
How similar studies have gone: Other research has shown promise in using gene editing and novel delivery systems for genetic disorders, indicating potential success for this approach.
Where this research is happening
-
University of Michigan at Ann Arbor
Ann Arbor, United States Active
Researchers
- Principal investigator: Piotrowski-Daspit, Alexandra Sarah Annukka — University of Michigan at Ann Arbor
- Study coordinator: Piotrowski-Daspit, Alexandra Sarah Annukka
Related research
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-
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-
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Gut microbiome in cystic fibrosis and colorectal cancer risk
Researchers are studying how the gut bacteria in adults with cystic fibrosis influence the development of colorectal cancer.
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.