Improving prenatal stem cell transplants for genetic disorders
Official title: Targeted conditioning to maximize prenatal HSC engraftment for SCD
Funded at Wake Forest University Health Sciences · NIH-10867474 · Data updated 2026-11-12
This study is looking at ways to make it easier for donated stem cells to successfully grow in unborn babies to help treat genetic disorders, like blood diseases, using fetal sheep to learn how to overcome challenges in the process.
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 start | July 1, 2023 |
| Project end | June 30, 2027 |
| Funding year | 2024 |
| Award (this year) | $662,949 |
| NIH project number | 5R01HL165247-02 |
| Project ID | NIH-10867474 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are pregnant individuals carrying fetuses diagnosed with genetic disorders that could benefit from stem cell transplantation.
✕Less likely to help
Patients with genetic disorders that are not amenable to treatment via in utero transplantation may not benefit from this research.
What this research studies
This research investigates how to enhance the success of in utero transplantation (IUTx) of hematopoietic stem cells (HSC) to treat genetic disorders. By using fetal sheep as a model, the study aims to identify and overcome barriers that prevent effective engraftment of donor stem cells in the developing fetus. The researchers will explore factors such as host stem cell competition, fetal immune responses, and the readiness of the bone marrow environment to accept new cells. The ultimate goal is to develop strategies that could improve outcomes for patients with conditions like hemoglobinopathies.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for genetic disorders through improved prenatal stem cell transplantation.
How similar studies have gone: While in utero transplantation has been performed successfully in some cases, this specific approach to enhancing engraftment is novel and has not been extensively tested.
Where this research is happening
-
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
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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.