Improving gene editing techniques for treating genetic disorders like cystic fibrosis
Official title: Develop High-Precision and Multiplex Base Editing Approaches for Therapeutic Applications
Funded at University of Pennsylvania · NIH-11039527 · Data updated 2026-11-12
This study is looking at new ways to fix the gene that causes cystic fibrosis, using a safe and precise method that could lead to better treatments for people with this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | April 1, 2024 |
| Project end | March 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $455,893 |
| NIH project number | 7R01HL157714-04 |
| Project ID | NIH-11039527 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with cystic fibrosis, particularly those with mutations that cannot be treated with existing therapies.
✕Less likely to help
Patients with genetic disorders not related to cystic fibrosis or those whose mutations are not targeted by the new gene editing techniques may not benefit from this research.
What this research studies
This research focuses on developing advanced gene editing techniques to address genetic disorders, particularly cystic fibrosis, which is caused by mutations in the CFTR gene. The approach utilizes base editing, a method that allows for precise changes to DNA without causing double-strand breaks, thereby reducing the risk of unwanted mutations. By improving the accuracy and safety of gene editing, this research aims to provide a more effective treatment option for patients suffering from genetic diseases. The study will explore the application of these techniques in cultured cell lines to evaluate their therapeutic potential.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for patients with cystic fibrosis and other genetic disorders.
How similar studies have gone: Previous research has shown promise in using gene editing techniques for genetic disorders, but this specific approach using base editing is relatively novel and untested in clinical applications.
Where this research is happening
-
University of Pennsylvania
Philadelphia, United States Active
Researchers
- Principal investigator: Gao, Xue — University of Pennsylvania
- Study coordinator: Gao, Xue
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.