Curing blood disorders without chemotherapy
Official title: Chemotherapy-free cure of hemoglobin disorders through base editing
Funded at Boston Children's Hospital · NIH-11112520 · Data updated 2026-11-12
This study is looking for a new way to help people with severe blood disorders like sickle cell disease and beta-thalassemia by using gene editing to boost their body's natural ability to make healthy blood, without the need for chemotherapy.
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 | September 1, 2023 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $766,048 |
| NIH project number | 5R01HL170629-03 |
| Project ID | NIH-11112520 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with sickle cell disease or transfusion-dependent beta-thalassemia.
✕Less likely to help
Patients with other types of blood disorders or those who do not have sickle cell disease or beta-thalassemia may not benefit from this research.
What this research studies
This research focuses on developing a new treatment for severe blood disorders like sickle cell disease and beta-thalassemia that avoids the use of chemotherapy. It aims to enhance the body's ability to produce fetal hemoglobin, which can help bypass the defects causing these conditions. The approach involves using advanced gene editing techniques to modify blood stem cells, making them more effective and safer for patients. By combining immunotherapy with gene editing, the researchers hope to create a safer and more effective treatment option for patients.
Why it matters
Potential benefit: If successful, this research could provide a safer, chemotherapy-free cure for patients with sickle cell disease and beta-thalassemia.
How similar studies have gone: Previous research has shown promise in using gene editing and immunotherapy for blood disorders, indicating that this approach could lead to significant advancements.
Where this research is happening
-
Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Bauer, Daniel Evan — Boston Children's Hospital
- Study coordinator: Bauer, Daniel Evan
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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.