Using a special antibody to improve gene therapy for sickle cell disease
Development of 211Astatine-Conjugated Anti-CD45 Antibody-Based Conditioning for Hematopoietic Stem Cell Gene Therapy and Editing
This study is testing a new, safer way to improve gene therapy for sickle cell disease by using a special antibody to help prepare patients' blood cells for treatment, making it easier to fix the genetic issues that cause the disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Fred Hutchinson Cancer Center NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project ID | NIH-10687021 on NIH RePORTER |
What this research studies
This research focuses on developing a new method to enhance gene therapy for sickle cell disease by using a specific antibody conjugated with astatine. The approach aims to condition patients' hematopoietic stem and progenitor cells (HSPCs) to improve the success of gene editing techniques that can correct the genetic defects causing the disease. By potentially replacing the need for traditional conditioning methods that carry significant risks, this study seeks to provide a safer and more effective treatment option for patients. The research will involve both laboratory work and clinical applications to ensure the method is viable for patient use.
Who could benefit from this research
Good fit: Ideal candidates for this research are children and young adults with sickle cell disease who may benefit from gene therapy.
Not a fit: Patients with sickle cell disease who are not eligible for gene therapy or those with severe comorbidities may not receive benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for patients with sickle cell disease.
How similar studies have performed: Other research has shown promise in using gene editing techniques for sickle cell disease, but this specific approach is novel.
Where this research is happening
Seattle, United States
- Fred Hutchinson Cancer Center — Seattle, United States (Active)
Researchers
- Principal investigator: Walter, Roland Bruno — Fred Hutchinson Cancer Center
- Study coordinator: Walter, Roland Bruno
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.