Improving CRISPR Gene Repair in Body Cells
Analysis of homolog-based CRISPR editing in somatic cells
This project aims to make CRISPR gene editing work better in our body's cells to fix genetic problems.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11122272 on NIH RePORTER |
What this research studies
Our bodies have natural ways to repair damaged DNA, but these methods don't always work perfectly in regular body cells when we try to use gene editing tools like CRISPR. This project explores how to guide our cells to use a more precise repair method, called homology directed repair (HDR), which is usually more active in reproductive cells. By understanding and improving this process, we hope to make gene editing a more effective and safer way to correct genetic errors in various conditions. This work could pave the way for new treatments that directly fix the root cause of genetic diseases.
Who could benefit from this research
Good fit: Patients with genetic diseases caused by specific DNA errors, such as those related to CFTR protein function, could potentially benefit from future therapies developed from this foundational work.
Not a fit: Patients seeking immediate treatment or those with conditions not caused by correctable genetic mutations would not directly benefit from this foundational research.
Why it matters
Potential benefit: If successful, this work could lead to more effective and precise gene therapies for a wide range of genetic diseases by improving how CRISPR tools correct DNA in body cells.
How similar studies have performed: While CRISPR systems have shown success in other applications, achieving efficient and precise gene editing in somatic cells using this specific repair pathway remains a significant challenge that this project aims to overcome.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Bier, Ethan — University of California, San Diego
- Study coordinator: Bier, Ethan
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.