Autologous, non-viral genome editing for ART-SCID (a patient-centered approach)
Official title: Autologous Non-viral CRISPR/Cas Homology-directed Repair for Artemis-deficient Severe Combined Immunodeficiency
Funded at University of California, San Francisco · NIH-11332524 · Data updated 2026-11-12
A pediatric immunology researcher is developing a non-viral genome-editing therapy to repair Artemis-deficient SCID using a patient’s own blood-forming cells.
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 | 2026 |
| Award (this year) | $200,016 |
| NIH project number | 5K08AI187714-02 |
| Project ID | NIH-11332524 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with Artemis-deficient SCID or related IEIs could ultimately benefit if this therapy progresses to clinical testing.
✕Less likely to help
Children or adults with ART-SCID who cannot undergo genome-editing therapies or who are not eligible for future trials may not benefit.
What this research studies
This grant supports a physician-scientist in a five-year plan to learn and apply non-viral genome editing in human hematopoietic stem cells, create preclinical models of ART-SCID, and test delivery and potency of edited cells. The goal is to move toward a safe, effective therapy for ART-SCID that could restore immune function using a patient’s own cells. It emphasizes mentorship from leaders in CRISPR technology and clinical immunology to build translational capabilities. The work involves laboratory studies, preclinical testing, and development of assays that would support future clinical application.
Why it matters
Potential benefit: If successful, the research could lead to a patient’s own cells repairing the genetic defect and restoring immune function.
How similar studies have gone: Preclinical studies using genome editing in hematopoietic cells have shown promise, but ART-SCID-specific translation is still early.
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.