IV-delivered blood stem cell gene therapy to cure HIV
In vivo HSC gene therapy using a multi-modular HDAd vector for HIV cure
This project develops a one-time IV gene therapy that changes your blood stem cells to try to eliminate HIV for people living with the virus.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Washington NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project ID | NIH-11283964 on NIH RePORTER |
What this research studies
The team mobilizes blood-forming stem cells out of the bone marrow into the bloodstream and then gives a single IV injection of a helper-dependent adenovirus vector that delivers protective genes to those stem cells. The modified stem cells travel back to the bone marrow, persist long-term, and produce blood cells that resist or clear HIV. The approach is designed to be delivered as an outpatient IV treatment without the high-dose chemotherapy and complex lab transplant steps used in current HSC therapies. The overall aim is durable prevention or eradication of HIV in target and reservoir cells.
Who could benefit from this research
Good fit: Ideal candidates would be adults living with HIV who are medically stable and able to undergo stem cell mobilization and clinic-based IV treatment.
Not a fit: People without HIV, those who cannot tolerate stem cell mobilization or who have serious medical conditions that make gene therapy unsafe, and likely pregnant individuals would not be expected to benefit.
Why it matters
Potential benefit: If successful, this could offer a one-time outpatient therapy that eliminates HIV from the body and removes the need for lifelong antiretroviral drugs.
How similar studies have performed: A few people were cured of HIV after bone marrow transplants from resistant donors and ex vivo HSC gene therapies work for some blood disorders, but direct IV in vivo stem cell gene therapy for HIV is novel and not yet proven in humans.
Where this research is happening
Seattle, United States
- University of Washington — Seattle, United States (Active)
Researchers
- Principal investigator: Lieber, Andre Michael — University of Washington
- Study coordinator: Lieber, Andre Michael
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.