Improving treatments for rare cystic fibrosis mutations
Official title: Rational optimization of combinatorial therapies for the treatment of rare cystic fibrosis variants
Funded at Purdue University · NIH-11094045 · Data updated 2026-11-12
This study is looking for the best combinations of treatments for people with rare types of cystic fibrosis, so they can have more options to help improve their lung health.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Purdue University NIH-funded |
| Lab location | 1 site (West Lafayette, United States) |
| Project start | September 1, 2023 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $686,508 |
| NIH project number | 5R01HL167046-03 |
| Project ID | NIH-11094045 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with cystic fibrosis who have rare CFTR mutations.
✕Less likely to help
Patients with the most common CFTR mutations, such as the ΔF508 variant, may not benefit directly from this research.
What this research studies
This research focuses on developing optimized combinations of therapies for patients with rare mutations of cystic fibrosis (CF). By utilizing advanced techniques, the study aims to identify which specific drug combinations can effectively target and treat these less common CF variants. The approach includes testing various small molecule correctors and potentiators to enhance the function of the CFTR protein, which is crucial for lung health. This research seeks to expand treatment options for patients who currently have limited therapeutic choices due to their unique genetic profiles.
Why it matters
Potential benefit: If successful, this research could significantly improve treatment outcomes for patients with rare cystic fibrosis mutations, potentially leading to better lung function and quality of life.
How similar studies have gone: Previous research has shown success in optimizing therapies for common CF mutations, but this approach for rare variants is relatively novel.
Where this research is happening
-
Purdue University
West Lafayette, United States Active
Researchers
- Principal investigator: Schlebach, Jonathan Patrick — Purdue University
- Study coordinator: Schlebach, Jonathan Patrick
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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.