Finding new treatments for cystic fibrosis that don't respond to current therapies
Official title: Pharmacogenomic discovery of therapeutic targets for corrector-refractory cystic fibrosis
Funded at Stanford University · NIH-11047482 · Data updated 2026-11-12
This study is looking into why some people with cystic fibrosis don’t get better with current treatments, using special techniques to find out how their cells are different, so they can discover new ways to help those patients feel better.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project start | December 10, 2024 |
| Project end | November 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $750,672 |
| NIH project number | 1R01HL172845-01A1 |
| Project ID | NIH-11047482 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with cystic fibrosis who have mutations in the CFTR gene that are not responsive to existing corrector therapies.
✕Less likely to help
Patients with cystic fibrosis who respond well to current FDA-approved corrector therapies may not benefit from this research.
What this research studies
This research focuses on understanding why some patients with cystic fibrosis do not respond to existing therapies that aim to correct the malfunctioning CFTR protein. By using advanced CRISPR-based pharmacogenomic techniques, the researchers will investigate the cellular mechanisms that lead to the degradation of CFTR in patients who are unresponsive to current treatments. The goal is to identify new therapeutic targets that could lead to effective treatments for those patients. This work addresses a critical gap in knowledge and aims to develop novel therapies for a significant portion of the cystic fibrosis population.
Why it matters
Potential benefit: If successful, this research could lead to new treatment options for cystic fibrosis patients who currently have no effective therapies available.
How similar studies have gone: While there has been significant progress in developing therapies for cystic fibrosis, this specific approach using CRISPR-based pharmacogenomics is relatively novel and untested.
Where this research is happening
-
Stanford University
Stanford, United States Active
Researchers
- Principal investigator: Kopito, Ron R — Stanford University
- Study coordinator: Kopito, Ron R
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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.