Gene therapy could revolutionize HIV treatment, potentially offering a durable, one-time solution for millions of people living with the virus worldwide, a study published in Science Advances by Johns Hopkins University researchers suggests.

Led by Associate Professor Fabio Romerio, the study focuses on a molecule within HIV called the antisense transcript (AST), which makes the virus go dormant and stop replicating.

By amplifying AST’s effects, this innovative approach could suppress HIV indefinitely, reducing or eliminating the need for lifelong antiretroviral therapy (ART), recommended for patients with HIV by the Department of Health and Human Services (DHHS) and the World Health Organization (WHO).

HIV, the virus that causes AIDS, remains a global health challenge, with 630,000 annual deaths, according to the World Health Organization.

Current ART suppresses viral replication but requires daily medication, carries side effects, and risks viral reactivation if interrupted. Gene therapy, however, could address these limitations.

“Our aim is to find a way to provide a lasting, durable treatment for HIV,” said Rui Li, postdoctoral fellow in Romerio’s lab and first author of the paper. By forcing HIV into a permanent dormant state, this approach could transform the lives of those affected, offering a less invasive alternative to daily regimens.

Scientists at Johns Hopkins looked at AST, a molecule made by HIV itself that helps keep the virus asleep (inactive) by sticking to certain proteins in infected cells. They tested this on both lab-grown immune cells and cells from 15 people living with HIV. By adding extra AST with genetic engineering, they made the virus stay asleep even more strongly. They also used a powerful laser method to find exactly which parts of AST attach to these proteins so they could design better treatments in the future.

Though the experiment shows AST’s potential to suppress HIV replication, the DNA degraded after this period, indicating a need for further development to achieve permanent latency. 

The study builds on prior work by Romerio’s team, which first identified AST’s role in HIV latency. By enhancing AST production, the researchers aim to develop a gene therapy that permanently silences HIV in infected cells, eliminating the need for daily ART.

This could significantly improve quality of life for those living with HIV, reducing the physical and psychological burden of lifelong treatment. 

Despite its promise, challenges remain. Sustaining AST production long-term is a critical hurdle, as the virus could reactivate if the therapy’s effects slow down. Additionally, ensuring the safety and efficacy of gene therapy in humans will require extensive testing.

This research is part of a broader wave of HIV innovation in 2025. Other approaches, like CRISPR-Cas9 gene editing and CAR T-cell therapies, are also showing promise in targeting HIV reservoirs. For millions worldwide, this study offers hope for a future where HIV is managed with minimal disruption, paving the way for a new era in HIV care.

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