Targeting a protein to increase fetal hemoglobin production in patients with blood disorders
DEGRADATION OF BCL11A PROTEIN FOR HbF REACTIVATION
This study is looking at a new way to boost the production of fetal hemoglobin in adults with beta-thalassemia and sickle cell disease by targeting a specific protein, which could lead to better treatments without needing harsh preparations.
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 ID | NIH-10900650 on NIH RePORTER |
What this research studies
This research focuses on reactivating fetal hemoglobin (HbF) in adult patients with beta-thalassemia and sickle cell disease by targeting the BCL11A protein, which inhibits HbF production. The approach utilizes small molecule therapeutics and targeted protein degradation techniques to safely and effectively increase HbF levels without the need for intensive preconditioning treatments. By analyzing erythroid cells at different stages of the cell cycle, the research aims to understand how and when HbF can be reactivated, potentially leading to new treatment options for these blood disorders.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults and young adults diagnosed with beta-thalassemia or sickle cell disease.
Not a fit: Patients who do not have beta-thalassemia or sickle cell disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a safer and more accessible treatment option for patients with beta-thalassemia and sickle cell disease, improving their quality of life.
How similar studies have performed: Previous research has shown promise in using similar approaches to reactivate fetal hemoglobin, indicating potential for success in this novel application.
Where this research is happening
Boston, United States
- Boston Children's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Orkin, Stuart H — Boston Children's Hospital
- Study coordinator: Orkin, Stuart H
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.