Improving gene therapy for treating RAG1 immunodeficiency
Development, Optimization and Preclinical Modeling of Hematopoietic Stem Cell Gene Editing for the Treatment of RAG1 Immunodeficiency
['FUNDING_R01'] · BOSTON CHILDREN'S HOSPITAL · NIH-11103261
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
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BOSTON CHILDREN'S HOSPITAL (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11103261 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients diagnosed with RAG1 immunodeficiency who lack a compatible hematopoietic stem cell donor.
Not a fit: Patients with RAG1 immunodeficiency who are eligible for allogeneic transplantation may not benefit from this research.
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 performed: 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, UNITED STATES
- BOSTON CHILDREN'S HOSPITAL — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: GENOVESE, PIETRO — BOSTON CHILDREN'S HOSPITAL
- Study coordinator: GENOVESE, PIETRO
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.