New drug targets for people with cystic fibrosis who don't respond to current corrector medicines
Pharmacogenomic discovery of therapeutic targets for corrector-refractory cystic fibrosis
Researchers are using genetic screening to find new protein targets and drug strategies for people with cystic fibrosis whose disease doesn't improve with current corrector drugs.
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 ID | NIH-11245760 on NIH RePORTER |
What this research studies
This project focuses on people with cystic fibrosis who do not get help from existing CFTR corrector medicines. Scientists will use CRISPR-based genetic screens in cells and human-derived samples to map the protein quality control systems that cause faulty CFTR to be destroyed. By identifying the specific proteins and pathways that block CFTR from reaching the cell surface, they aim to point to new targets for drugs or drug combinations. The ultimate goal is to find approaches that could rescue CFTR function for people with rare or corrector-refractory variants.
Who could benefit from this research
Good fit: Ideal candidates are people with cystic fibrosis who do not respond to FDA-approved CFTR corrector therapies, including those with rare or 'corrector-refractory' CFTR variants.
Not a fit: People with CF who already have good results from current corrector therapies, or those without CF, are unlikely to benefit directly from this early-stage research.
Why it matters
Potential benefit: If successful, this work could lead to new treatments or drug combinations that help people with CF who don't benefit from existing corrector medicines.
How similar studies have performed: Related CRISPR and pharmacogenomic approaches have identified disease-modifying targets in lab models and some CF research, but targeting the protein-quality-control pathways for corrector-refractory CF is relatively new.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Kopito, Ron R — Stanford University
- Study coordinator: Kopito, Ron R
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 below.