Improving before-birth stem cell transplants for sickle cell disease
Targeted conditioning to maximize prenatal HSC engraftment for SCD
This work looks for safer ways to prepare unborn babies at risk of sickle cell disease so donated blood-forming stem cells can take hold and correct the disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Wake Forest University Health Sciences NIH-funded |
| Lab location | 1 site (Winston-Salem, United States) |
| Project ID | NIH-11129924 on NIH RePORTER |
What this research studies
From a patient's point of view, researchers are trying to make stem cell transplants given before birth work better by creating space in the fetal bone marrow, limiting competition from the fetus's own blood stem cells, and managing fetal immune responses. They plan to test non-genotoxic (less harmful) conditioning approaches that could open bone marrow niches without using damaging radiation or chemotherapy. Experiments will use a fetal sheep model that closely mimics human fetal development and pregnancy to see how well adult donor hematopoietic stem cells engraft and produce healthy blood cells. Findings will guide safer strategies that could eventually be offered to human pregnancies diagnosed with severe hemoglobin disorders.
Who could benefit from this research
Good fit: The ideal future candidates would be fetuses diagnosed before birth with severe hemoglobin disorders, such as sickle cell disease, who could be offered in utero stem cell transplantation.
Not a fit: Adults, people already born, or patients whose conditions cannot be corrected by blood-forming stem cell replacement are unlikely to benefit directly from this project.
Why it matters
Potential benefit: If successful, this work could enable safe prenatal stem cell transplants that correct sickle cell disease before symptoms start, preventing lifelong complications.
How similar studies have performed: In utero transplants have previously cured X-linked SCID but have rarely achieved full success for other disorders, so this builds on past success while testing new, experimental low-toxicity conditioning methods.
Where this research is happening
Winston-Salem, United States
- Wake Forest University Health Sciences — Winston-Salem, United States (Active)
Researchers
- Principal investigator: Almeida-Porada, Graca Duarte — Wake Forest University Health Sciences
- Study coordinator: Almeida-Porada, Graca Duarte
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.