Developing a new treatment for a specific immune deficiency using gene editing
Autologous Non-viral CRISPR/Cas Homology-directed Repair for Artemis-deficient Severe Combined Immunodeficiency
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 ID | NIH-11033979 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Artemis-deficient severe combined immunodeficiency.
Not a fit: Patients with other forms of severe combined immunodeficiency or those without Artemis deficiency may not benefit from this research.
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 performed: 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
San Francisco, United States
- 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
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.