Using CRISPR technology to fix genetic mutations causing cystic fibrosis
Official title: Leveraging a 'sliding-window' Type I CRISPR base editing platform to correct CFTR null mutations
Funded at University of Michigan at Ann Arbor · NIH-11066610 · Data updated 2026-11-12
This study is testing a new way to fix a specific gene mutation that causes cystic fibrosis, aiming to help patients who don’t respond to current treatments by offering a long-lasting solution to their condition.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| 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 | January 1, 2025 |
| Project end | December 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $43,025 |
| NIH project number | 1F31HL177911-01 |
| Project ID | NIH-11066610 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with cystic fibrosis who carry the CFTR-G542X mutation.
✕Less likely to help
Patients with cystic fibrosis who do not have the CFTR-G542X mutation may not benefit from this specific research.
What this research studies
This research focuses on developing a novel gene editing approach using a Type I CRISPR base editing platform to correct specific mutations in the CFTR gene responsible for cystic fibrosis (CF). The project aims to target the CFTR-G542X mutation, which affects a significant portion of CF patients who do not respond to existing treatments. By correcting this mutation, the research seeks to provide a permanent solution rather than temporary relief from symptoms. Patients may benefit from a more effective and lasting treatment option that addresses the root cause of their condition.
Why it matters
Potential benefit: If successful, this research could lead to a permanent cure for cystic fibrosis in patients with specific genetic mutations.
How similar studies have gone: Previous research using CRISPR technology has shown promise in correcting genetic mutations, 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: Myers, Mason T — University of Michigan at Ann Arbor
- Study coordinator: Myers, Mason T
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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.