Gene therapy that targets brain immune cells to remove HIV

Brain myeloid cell-targeted multiplexed gene editing for SIV/HIV eradication

['FUNDING_R01'] · TEXAS BIOMEDICAL RESEARCH INSTITUTE · NIH-11296929

Develops a gene-editing approach delivered to brain immune cells to try to remove hidden HIV in people living with HIV.

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorTEXAS BIOMEDICAL RESEARCH INSTITUTE (nih funded)
Locations1 site (SAN ANTONIO, UNITED STATES)
Trial IDNIH-11296929 on ClinicalTrials.gov

What this research studies

From a patient's perspective, researchers are working on a gene-editing treatment delivered by a harmless virus to reach long-lived brain immune cells (microglia and perivascular macrophages) where HIV can hide. They are improving viral delivery tools so the therapy can cross the blood-brain barrier and specifically enter those myeloid cells. The approach is being tested in animal models (macaques infected with SIV) and builds on earlier AAV-CRISPR work that removed viral DNA in mice and primates. The team aims to find a safe way to clear brain reservoirs without triggering harmful inflammation.

Who could benefit from this research

Good fit: People living with HIV—particularly those on long-term antiretroviral therapy who may have virus hidden in the central nervous system—would be the eventual candidates for related clinical trials.

Not a fit: People without HIV, those whose infection is fully controlled with no evidence of CNS involvement, or those who cannot tolerate gene therapy or have active brain inflammation are unlikely to benefit from this work.

Why it matters

Potential benefit: If successful, this could help remove hidden HIV in the brain, reduce the chance of viral rebound, and move toward a durable cure.

How similar studies have performed: Similar AAV-CRISPR strategies have removed HIV/SIV DNA in humanized mice and nonhuman primates, but reliably targeting brain myeloid cells is a newer and still challenging goal.

Where this research is happening

SAN ANTONIO, 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.

View on NIH RePORTER →

Conditions: Acquired Immune Deficiency Syndrome Virus, Acquired Immunodeficiency Syndrome Virus

Last reviewed 2026-05-15 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.