Gene therapy to remove hidden HIV from brain immune cells

Long-term microglia-targeted endogenous retrovirus-like particle (ERVLP) delivery of Cas12f editor to cure HIV

NIH-funded research Virginia Commonwealth University · NIH-11131076

A new gene-editing delivery aims to clear hidden HIV from brain immune cells in people living with HIV.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionVirginia Commonwealth University NIH-funded
Lab location1 site (Richmond, United States)
Project IDNIH-11131076 on NIH RePORTER

What this research studies

This project is developing a way to deliver a very small gene editor (Cas12f) into brain immune cells called microglia, which can hide HIV even when blood virus is controlled. The team will use engineered AAV viruses together with a natural particle-making system (PEG10-based ERVLPs) to carry the editor across the blood-brain barrier and into microglia. Work includes lab and animal tests to improve delivery, measure how well the editor removes HIV from brain cells, and check safety. The goal is a long-lasting, targeted approach that could one day be tested in people.

Who could benefit from this research

Good fit: People living with HIV, especially adults on stable antiretroviral therapy who have suspected brain reservoirs or HIV-associated cognitive symptoms, would be the population for future trials.

Not a fit: People without HIV, or those with medical conditions that make them ineligible for gene therapies, would not benefit from this work.

Why it matters

Potential benefit: If successful, this approach could remove HIV reservoirs in the brain, reduce chronic neuroinflammation and cognitive problems, and move toward a functional cure.

How similar studies have performed: Other CRISPR-based HIV approaches have shown early promise and entered phase I testing, but using Cas12f delivered via PEG10-based ERVLPs to target microglia is a novel strategy.

Where this research is happening

Richmond, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions Acquired Immune Deficiency Syndrome VirusAcquired Immunodeficiency Syndrome Virus
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.