Engineered AAV gene therapy using Brec1 to remove hidden HIV
Capsid-engineered AAV vectors with Brec1-based gene therapeutics for inactivating the HIV reservoir
This project uses a targeted gene therapy that delivers a precise HIV-cutting enzyme called Brec1 to try to remove hidden HIV DNA from infected cells in people living with HIV.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Yale University NIH-funded |
| Lab location | 1 site (New Haven, United States) |
| Project ID | NIH-11139387 on NIH RePORTER |
What this research studies
If you are living with HIV, this work aims to create a one-time gene treatment that can find and remove the HIV DNA stuck in your cells. Researchers plan to package a designer enzyme called Brec1 into specially modified AAV delivery vehicles that are redesigned to reach the cells that hide the virus. The team will test these engineered capsids and the Brec1 enzyme in lab studies and animal models to check safety and how well they remove proviral DNA. Successful preclinical results would be the step before any human testing at research centers.
Who could benefit from this research
Good fit: Ideal candidates in future trials would likely be adults living with HIV who have suppressed viral loads on antiretroviral therapy and whose virus carries the specific Brec1 target sequence.
Not a fit: People whose HIV lacks the specific Brec1 target sequence, children, pregnant people, or those with medical conditions that rule out experimental gene therapies may not benefit from this approach.
Why it matters
Potential benefit: If successful, this approach could reduce or eliminate the HIV reservoir and potentially lead to durable remission or a cure for some people living with HIV.
How similar studies have performed: AAV delivery has been successful for some genetic disorders and gene-editing strategies for HIV are being explored, but using a Brec1 recombinase delivered by engineered AAV to excise HIV is novel and has not yet been tested in humans.
Where this research is happening
New Haven, United States
- Yale University — New Haven, United States (Active)
Researchers
- Principal investigator: Kumar, Priti — Yale University
- Study coordinator: Kumar, Priti
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.