Targeting BCL11A to raise fetal hemoglobin
Dissecting Enhancer-Centric Mechanisms for Therapeutic Targeting of BCL11A
This project looks for new ways to lower BCL11A so people with sickle cell disease or β-thalassemia can make more fetal hemoglobin and have milder symptoms.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project ID | NIH-11342564 on NIH RePORTER |
What this research studies
From my perspective as a patient, researchers are examining the genetic switch that keeps fetal hemoglobin turned off in adult red blood cells. They use lab experiments, including CRISPR editing of an enhancer near the BCL11A gene and studies of human blood cells, to learn why enhancer editing so effectively lowers BCL11A. The team aims to uncover specific molecular weaknesses that could be targeted with cheaper, more accessible therapies than current gene-editing treatments. Their goal is to find approaches that could one day be turned into treatments that more people can reach and afford.
Who could benefit from this research
Good fit: Adults with sickle cell disease or β-thalassemia, or healthy adult blood donors willing to provide blood or bone marrow samples for lab studies, would be the most relevant participants.
Not a fit: Children under 21 and people without hemoglobin disorders are unlikely to gain direct benefit from this research.
Why it matters
Potential benefit: If successful, this work could lead to safer and more affordable treatments that raise fetal hemoglobin and reduce complications from sickle cell disease and β-thalassemia.
How similar studies have performed: CRISPR editing of the BCL11A enhancer has already produced transformative clinical results as one of the first CRISPR-based therapies for blood disorders, while this project seeks to explain that success and find alternative ways to achieve it.
Where this research is happening
Memphis, United States
- St. Jude Children's Research Hospital — Memphis, United States (Active)
Researchers
- Principal investigator: Xu, Jian — St. Jude Children's Research Hospital
- Study coordinator: Xu, Jian
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.