Using nanoparticles to improve gene therapy for blood stem cells
Official title: CD90-targeted nanoparticles for in vivo hematopoietic stem cell gene therapy
Funded at Fred Hutchinson Cancer Center · NIH-11048496 · Data updated 2026-11-12
This study is exploring a new way to use tiny particles to change your own blood stem cells right in your body, making gene therapy easier and safer for people with genetic blood disorders who struggle to find donors for transplants.
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 start | May 1, 2025 |
| Project end | February 28, 2029 |
| Funding year | 2025 |
| Award (this year) | $814,209 |
| NIH project number | 1R01HL173365-01A1 |
| Project ID | NIH-11048496 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients with genetic blood disorders who do not have a suitable HLA-matched donor for stem cell transplantation.
✕Less likely to help
Patients with non-genetic blood disorders or those who have access to suitable HLA-matched donors may not benefit from this research.
What this research studies
This research focuses on developing a method to genetically modify a patient's own blood stem cells using nanoparticles, which could allow for safer and more accessible gene therapy. Currently, many patients with genetic blood disorders face challenges in finding suitable donors for stem cell transplants, and existing gene therapy methods require complex facilities. By enabling in vivo gene therapy, this approach aims to simplify the process and reduce the need for specialized infrastructure. The research will utilize advanced techniques to ensure that the nanoparticles effectively target and modify the blood stem cells directly within the patient's body.
Why it matters
Potential benefit: If successful, this research could provide a safer and more accessible treatment option for patients with genetic blood diseases.
How similar studies have gone: While gene therapy for blood disorders has shown promise, the specific approach of using nanoparticles for in vivo modification is relatively novel and has not been extensively tested.
Where this research is happening
-
Fred Hutchinson Cancer Center
Seattle, United States Active
Researchers
- Principal investigator: Kiem, Hans-Peter — Fred Hutchinson Cancer Center
- Study coordinator: Kiem, Hans-Peter
Related research
Other NIH-funded projects on the same topics.
-
How brain glia prune neuron fragments in a worm model
Researchers study how brain support cells called glia remove pieces of living neurons in a tiny worm to learn about brain aging and diseases like Alzheimer's.
-
Understanding and Targeting Lineage Plasticity in Prostate Cancer with Genomic-Based Neoadjuvant Therapy
A broad research program at Fred Hutchinson Cancer Center is exploring how aggressive prostate cancers change their cell states in response to therapy and how to block these changes.
-
Personalized cancer drug testing on patient tumor cells
Aiming to test many FDA-approved cancer drugs on a patient’s own tumor cells to identify effective options when standard therapies fail.
-
MOSAAIC: Using health records to track heart disease in Asian American Communities
A big, dedicated effort is testing whether electronic health records can more quickly and accurately track heart and blood vessel problems in Asian American populations.
-
Targeting gut microbes to prevent progression to multiple myeloma
Researchers are testing how specific gut bacteria might trigger immune changes linked to early blood cancer stages and whether targeting these microbes could help prevent progression to multiple myeloma.
-
eDyNAmiC: Understanding extrachromosomal DNA in cancer and how it drives aggressiveness
A cancer research project at Fred Hutchinson Cancer Center is studying abnormal circular DNA pieces (ecDNA) that help cancers grow and resist treatment, with the aim of finding ways to help patients.
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.