A study published in Nature may point to a promising new path in the fight against pediatric HIV. Led by Mauricio A. Martins, PhD, with key work conducted at the California National Primate Research Center and contributions from Tulane National Biomedical Research Center, researchers showed that a single gene therapy injection given at birth could provide long-term protection against HIV by taking advantage of a unique window when a newborn’s immune system is more tolerant.

The study used an adeno-associated virus, or AAV, to deliver genetic instructions for producing broadly neutralizing antibodies, which are powerful antibodies capable of recognizing multiple strains of HIV. The therapy essentially turns muscle cells into antibody-producing “micro-factories,” offering a potential way to provide lasting protection without repeated treatments or boosters.

Timing proved critical. Infant rhesus macaques treated at birth maintained antibody expression for more than three years and were protected in models that mimic HIV transmission through breastfeeding and sexual exposure. Older recipients were less protected, underscoring the importance of early intervention and the role of the newborn immune system’s natural window of tolerance.

This approach could help address mother-to-child HIV transmission, which remains a major global health challenge, especially in regions where access to follow-up care and long-term treatment can be limited. A one-time intervention given at birth could offer a more practical and scalable prevention strategy for infants at risk.

More research is needed before this approach can be tested in humans, including studies to determine how it may work against different HIV strains. Still, the findings offer hope for a cost-effective, long-lasting strategy to help reduce pediatric HIV and may eventually inform similar approaches for other infectious diseases, including malaria.