In utero gene editing for sickle cell disease and alpha-thalassemia using lipid nanoparticles
Official title: In utero gene editing via LNP delivery for hemoglobinopathies
Funded at Children's Hosp of Philadelphia · NIH-11422868 · Data updated 2026-11-12
A prenatal therapy using specially designed lipid nanoparticles to edit genes in fetal blood-forming cells could prevent sickle cell disease and alpha-thalassemia before symptoms start.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 1, 2026 |
| Project end | May 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $452,150 |
| NIH project number | 1P01HL187309-01 |
| Project ID | NIH-11422868 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be pregnant individuals carrying a fetus at risk for SCD or AT where in utero therapy could be ethically and medically appropriate and accessible.
✕Less likely to help
Pregnancies with contraindications to fetal interventions or where maternal-fetal risk outweighs potential benefit may not benefit.
What this research studies
This project explores delivering gene-editing tools to a fetus during pregnancy to fix disease-causing genes in blood-forming cells. Researchers will optimize lipid nanoparticles to carry gene-editing mRNA and test targeting strategies in animal models that mimic human sickle cell disease and alpha-thalassemia. The work aims to achieve lasting correction in fetal hematopoietic stem cells with minimal invasive procedures and without the need for harsh conditioning. If successful, it could offer a one-time intervention before birth to prevent severe disease progression.
Why it matters
Potential benefit: If successful, this approach could prevent or lessen the impact of SCD and AT before birth, potentially reducing early illness and improving long-term health.
How similar studies have gone: Early animal studies show promising delivery and editing to fetal hematopoietic cells, but this approach is still in preclinical development.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Peranteau, William H. — Children's Hosp of Philadelphia
- Study coordinator: Peranteau, William H.
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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.