Using gene editing to fix mutations that cause immune disorders
Editing to Create and Correct Gene Variants
This study is exploring how to find and fix genetic mistakes that cause primary immunodeficiency (PID) using cutting-edge tools, with the goal of developing safer treatments that could help your immune system work better without needing a risky donor transplant.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-10929328 on NIH RePORTER |
What this research studies
This research investigates how to identify and correct genetic mutations responsible for primary immunodeficiency (PID) using advanced techniques like CRISPR-Cas9 genome editing. By employing whole genome sequencing, the team aims to uncover both coding and non-coding mutations that contribute to T lymphocyte deficiencies. The approach includes testing how specific genetic sequences influence the development of immune cells from stem cells, ultimately leading to potential therapies that rewrite harmful mutations in patients' blood-forming cells. This could provide a more effective alternative to traditional donor transplants, which often come with significant risks.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with primary immunodeficiency disorders caused by genetic mutations.
Not a fit: Patients with immune disorders not linked to genetic mutations or those who do not have primary immunodeficiency may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to personalized gene therapies that effectively treat or even cure certain immune disorders.
How similar studies have performed: Other research has shown promise in using CRISPR technology for gene editing, indicating potential success for this novel approach.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Marson, Alexander — University of California, San Francisco
- Study coordinator: Marson, Alexander
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.