Using CRISPR to fix genetic defects in the immune system
Official title: CRISPR Editing of Primary Human Cells to Model and Correct Primary Immunodeficiency Mutations
Funded at University of California, San Francisco · NIH-10884398 · Data updated 2026-11-12
This study is looking at how to fix genetic problems that cause primary immunodeficiency (PID), a condition that makes it hard for your immune system to work right, using a special technology called CRISPR to help create new treatments that could help people with PID feel better.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | July 1, 2020 |
| Project end | June 30, 2025 |
| Funding year | 2024 |
| Award (this year) | $198,735 |
| NIH project number | 5K08AI153767-05 |
| Project ID | NIH-10884398 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with primary immunodeficiency diseases caused by specific genetic mutations.
✕Less likely to help
Patients with primary immunodeficiency who do not have identifiable genetic mutations or those with non-genetic causes of immune dysfunction may not benefit from this research.
What this research studies
This research focuses on understanding and correcting genetic mutations that cause primary immunodeficiency (PID), a condition where the immune system fails to function properly. The principal investigator, Dr. David Nguyen, will use advanced CRISPR gene-editing technology to recreate and test these mutations in human immune cells. By identifying the specific genetic changes responsible for PID, the research aims to develop new strategies to replace these mutations in patients' own blood cells, potentially leading to effective treatments. This innovative approach could pave the way for personalized gene therapies for individuals with PID.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking treatments that cure primary immunodeficiency diseases.
How similar studies have gone: Previous research using CRISPR technology has shown promise in correcting genetic defects, indicating that this approach could be successful in treating primary immunodeficiency.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Nguyen, David-Huy Nhu — University of California, San Francisco
- Study coordinator: Nguyen, David-Huy Nhu
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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.