In vivo gene therapy for blood diseases using multiplexed virus-like particles
Official title: In vivo gene therapy for hemoglobinopathies with multiplexed virus-like particles
Funded at Fred Hutchinson Cancer Center · NIH-11364773 · Data updated 2026-11-12
A new approach aims to repair patients’ own blood stem cells inside their bodies to treat genetic 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 start | January 17, 2017 |
| Project end | April 30, 2030 |
| Funding year | 2026 |
| Award (this year) | $905,029 |
| NIH project number | 2R01HL136135-10 |
| Project ID | NIH-11364773 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with genetic blood diseases who lack suitable donor options for stem cell transplant.
✕Less likely to help
Patients without access to specialized in vivo gene therapy facilities or who have conditions not amenable to this approach may not benefit.
What this research studies
This project explores delivering gene-editing methods directly into patients to modify hematopoietic stem cells in vivo, potentially reducing the need for complex hospital-based procedures. It builds on nonhuman primate research to identify the most effective cell targets and delivery strategies. If successful, the approach could lead to portable, less infrastructure-dependent gene therapy for blood disorders.
Why it matters
Potential benefit: If successful, patients could receive safer, more accessible treatment that uses their own cells to correct genetic defects.
How similar studies have gone: Previous work shows promise in gene therapy and HSC targeting, but in vivo delivery remains a key challenge.
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.
-
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.
-
Targeting blood stem cells with tiny fat bubbles to treat inherited blood diseases
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.
-
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.
-
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.
-
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.
-
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.