Nanoparticles that target blood-forming stem cells to enable in-body gene correction
CD90-targeted nanoparticles for in vivo hematopoietic stem cell gene therapy
Tiny particles are being developed to carry gene fixes directly into a person’s blood-forming stem cells to help people with inherited blood diseases.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Fred Hutchinson Cancer Center NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project ID | NIH-11325697 on NIH RePORTER |
What this research studies
You would get very small, targeted particles designed to stick to a marker called CD90 on your blood-forming stem cells and deliver corrective genes without removing the cells from your body. The team will study where the particles go, how effectively they deliver genetic material, and whether they are safe using lab tests and nonhuman primate models. The plan is to replace complex, hospital-based stem cell transplants with a simpler in-body treatment that could work without specialized transplant infrastructure. This could make gene therapy faster and more available, especially in places without advanced transplant centers.
Who could benefit from this research
Good fit: People with inherited blood diseases (for example sickle cell disease, thalassemia, or certain bone marrow failure syndromes) who need hematopoietic stem cell gene correction would be the eventual candidates.
Not a fit: Patients with blood problems that do not stem from a genetic defect in hematopoietic stem cells, or those needing immediate treatment, may not benefit, and this project is currently at a preclinical stage so it does not yet provide direct patient treatment.
Why it matters
Potential benefit: If successful, this could allow one-time in-body correction of genetic blood disorders without donor transplants or the risk of graft-versus-host disease.
How similar studies have performed: Ex vivo gene therapies for blood disorders have helped patients, but direct in-body delivery to hematopoietic stem cells is largely new with encouraging animal-model results and no established human successes yet.
Where this research is happening
Seattle, United States
- Fred Hutchinson Cancer Center — Seattle, United States (Active)
Researchers
- Principal investigator: Kiem, Hans-Peter — Fred Hutchinson Cancer Center
- Study coordinator: Kiem, Hans-Peter
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.