Developing a new treatment for a specific immune deficiency using gene editing
Official title: Autologous Non-viral CRISPR/Cas Homology-directed Repair for Artemis-deficient Severe Combined Immunodeficiency
Funded at University of California, San Francisco · NIH-11033979 · Data updated 2026-11-12
This study is exploring a new treatment for people with Artemis-deficient severe combined immunodeficiency (ART-SCID) by using a special gene-editing method to fix the genetic problem in their own blood stem cells, with the goal of helping their immune systems work 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 | May 8, 2025 |
| Project end | April 30, 2030 |
| Funding year | 2025 |
| Award (this year) | $200,016 |
| NIH project number | 1K08AI187714-01 |
| Project ID | NIH-11033979 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Artemis-deficient severe combined immunodeficiency.
✕Less likely to help
Patients with other forms of severe combined immunodeficiency or those without Artemis deficiency may not benefit from this research.
What this research studies
This research focuses on creating a novel therapy for patients with Artemis-deficient severe combined immunodeficiency (ART-SCID) using non-viral CRISPR/Cas technology. The approach involves editing the genes of a patient's own blood stem cells to correct the underlying genetic defect. By utilizing advanced techniques in genome editing, the research aims to improve the immune system function in affected individuals. The project includes preclinical modeling and testing to ensure the safety and effectiveness of the treatment before it can be applied in clinical settings.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients suffering from ART-SCID, potentially restoring their immune function.
How similar studies have gone: Previous research has shown promise in using gene editing techniques for similar genetic disorders, indicating a potential for success in this novel approach.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Kan, Matthew J — University of California, San Francisco
- Study coordinator: Kan, Matthew J
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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.