How hemoglobin changes from the fetal form to the adult form
Regulation of Hemoglobin Switching
This work looks at how genes like HIC2 and BCL11A control the switch from fetal to adult hemoglobin to help people with sickle cell disease and beta-thalassemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11293396 on NIH RePORTER |
What this research studies
Researchers use laboratory gene-editing screens (CRISPR-Cas9) to find regulators that turn fetal-type hemoglobin off and adult-type hemoglobin on. They compare fetal-like and adult red blood cell precursors and study how HIC2 and other factors control a key BCL11A enhancer that drives the switch. The team tests findings in human cell samples and animal models to confirm which signals are critical. The goal is to map the chain of molecular events so future therapies can boost protective fetal hemoglobin in patients.
Who could benefit from this research
Good fit: Adults with sickle cell disease or beta-thalassemia who are interested in contributing samples or who may be eligible for future therapies would be the most relevant candidates.
Not a fit: People under age 21 or those with anemia from causes unrelated to hemoglobin switching are unlikely to benefit from this work.
Why it matters
Potential benefit: If successful, this could point to new ways to reactivate fetal hemoglobin and reduce symptoms in people with sickle cell disease and beta-thalassemia.
How similar studies have performed: Related approaches targeting BCL11A and reactivating fetal hemoglobin have already shown promise in clinical trials, while this project aims to expand understanding of upstream regulation.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Blobel, Gerd a — Children's Hosp of Philadelphia
- Study coordinator: Blobel, Gerd a
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.