Improving gene therapy for treating RAG1 immunodeficiency
Official title: Development, Optimization and Preclinical Modeling of Hematopoietic Stem Cell Gene Editing for the Treatment of RAG1 Immunodeficiency
Funded at Boston Children's Hospital · NIH-11103261 · Data updated 2026-11-12
This study is working on a new way to use gene editing with your own stem cells to help treat RAG1 immunodeficiency, a serious condition that affects your immune system, so that people without a matching donor can have a safer and more personalized treatment option.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | June 8, 2021 |
| Project end | May 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $442,500 |
| NIH project number | 5R01AI155796-06 |
| Project ID | NIH-11103261 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with RAG1 immunodeficiency who lack a compatible hematopoietic stem cell donor.
✕Less likely to help
Patients with RAG1 immunodeficiency who are eligible for allogeneic transplantation may not benefit from this research.
What this research studies
This research focuses on developing a new gene editing approach using hematopoietic stem cells to treat RAG1 immunodeficiency, a severe genetic condition affecting the immune system. The team aims to create a safe and effective therapy that can restore the function of the RAG1 gene while minimizing risks associated with traditional gene therapy methods. By using patient-derived stem cells, the research seeks to provide a personalized treatment option for individuals who do not have a compatible donor for stem cell transplantation. The methodology involves optimizing gene editing techniques to ensure precise control over gene expression, which is crucial for the immune system's proper functioning.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment option for patients with RAG1 immunodeficiency, potentially restoring their immune function.
How similar studies have gone: Previous research in hematopoietic stem cell gene therapy has shown clinical benefits, indicating that this approach has potential for success.
Where this research is happening
-
Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Genovese, Pietro — Boston Children's Hospital
- Study coordinator: Genovese, Pietro
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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.