Targeting blood stem cells with tiny fat bubbles to treat inherited blood diseases
Official title: Targeting hematopoietic stem cells by lipid nanoparticles to treat hemoglobinopathies
Funded at Children's Hosp of Philadelphia · NIH-11422866 · Data updated 2026-11-12
A new approach uses targeted lipid nanoparticles to temporarily reduce certain blood-forming stem cells, paving the way for safer stem cell transplants for conditions like sickle cell and thalassemia.
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-11422866 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with sickle cell disease or thalassemia who may need stem cell transplantation in the future and could participate in related human studies or trials.
✕Less likely to help
Patients not eligible for stem cell transplantation or those not suited for lipid nanoparticle–based approaches might not benefit.
What this research studies
This grant focuses on a novel, targeted therapy that delivers pro-apoptotic messages to hematopoietic stem cells using lipid nanoparticles, with the aim of enabling donor engraftment while reducing toxicity. The work includes preclinical studies in models of human blood diseases to test fertility-preserving strategies and selective depletion of diseased stem cells. If successful, this could lead to less damaging treatments for patients who need stem cell transplantation. The research is conducted at Children’s Hospital of Philadelphia under the leadership of Dr. Stefano Rivella.
Why it matters
Potential benefit: If successful, patients could receive stem cell transplants with fewer toxic side effects and better chances of cure for sickle cell disease and thalassemias.
How similar studies have gone: Preclinical studies have shown partial engraftment improvements and disease phenotype correction in mouse models, suggesting potential but still early-stage.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Rivella, Stefano — Children's Hosp of Philadelphia
- Study coordinator: Rivella, Stefano
Related research
Other NIH-funded projects on the same topics.
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In utero gene editing for sickle cell disease and alpha-thalassemia using lipid nanoparticles
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.
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In vivo gene therapy for blood diseases using multiplexed virus-like particles
A new approach aims to repair patients’ own blood stem cells inside their bodies to treat genetic blood diseases.
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Oral protein-targeting drugs to raise fetal hemoglobin for sickle cell disease
Testing new oral medicines that remove proteins blocking fetal hemoglobin to help adults with sickle cell disease reduce red blood cell sickling.
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Developing new treatments to increase fetal hemoglobin for sickle cell disease
This study is working on new medications that can help increase a type of hemoglobin in your blood to better manage Sickle Cell Disease, aiming to provide a more reliable and safer treatment option than what’s currently available.
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Using nurses to provide genetic counseling for sickle cell disease in primary care.
This study is looking at how training nurses to provide genetic counseling and carrier screening for sickle cell disease can help families in Sierra Leone make informed choices about having children, especially since there aren't many genetic counselors available in the area.
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HOPE Biobank: connecting blood disease patients through stem cell transplant and gene therapy samples
This study will collect blood and other biospecimens along with clinical data from people undergoing stem cell transplant or gene therapy for non-cancer blood diseases to build a large biobank for future research.
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.