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HIV Breakthrough: Researchers Aim To Shut Down Virus With One-Time Gene Therapy

HIV remains incurable but manageable. Johns Hopkins found a way to silence it long-term using a molecule from the virus itself, offering hope for future gene therapy without daily meds.

HIV is no longer a death sentence, but it remains a serious chronic illness given that it has no cure or vaccine. Amid stress on continued research, equitable access to care, and global awareness which are considered to be  crucial to ending the epidemic, researchers at Johns Hopkins Medicine have made a breakthrough in the fight against the virus--a way to potentially silence the pathogen for extended periods using a molecule found within HIV itself.

The study, published in Science Advances, suggests that enhancing the activity of a natural molecule called an “antisense transcript” (AST) could keep the virus in a dormant state, preventing it from reproducing.

HIV infects the body’s immune cells and causes AIDS. While antiretroviral drugs can suppress the virus, they must be taken daily for life and can come with side effects. If the medication is stopped, the virus typically reactivates. However, the Johns Hopkins team hopes their discovery will one day lead to a gene therapy that offers a longer-lasting solution.

“We’re trying to develop a treatment that doesn’t require daily medication—a one-time therapy that could keep HIV inactive for the long term,” said Dr. Rui Li, a postdoctoral researcher and first author of the study.

The study builds on earlier work from Dr. Fabio Romerio, associate professor at the Johns Hopkins University School of Medicine, whose team previously identified AST as a molecule produced by HIV’s own genetic material. AST plays a role in keeping the virus in a “latent” state—a sort of biological sleep where the virus stays hidden and doesn’t replicate.

To test its potential, the scientists modified human immune cells (specifically CD4+ T cells, the type targeted by HIV) to produce higher levels of AST. When they did, they saw that the virus stopped replicating, as shown by a drop in a glowing marker protein used to measure HIV activity.

The team also used advanced laser-based technology to pinpoint which segments of the AST molecule are most responsible for suppressing the virus. By creating different versions of AST and inserting them into infected T cells, they determined which parts were critical for locking the virus in its dormant state.

The researchers confirmed their findings in immune cells taken from 15 people living with HIV.

“This gives us a solid foundation to explore gene therapy as a long-term treatment option,” Dr. Romerio said. “Our ultimate goal is to put HIV into a permanent state of dormancy—essentially shutting it down without having to eliminate it entirely.”

The findings open up a promising new pathway toward a future in which HIV can be controlled without lifelong drug regimens, improving quality of life and reducing health burdens worldwide.

Global HIV Snapshot

  • 39.9 million people living with HIV worldwide at the end of 2023.

  • 1.3 million new infections occurred in 2023 (a 39% drop since 2010), including about 120,000 children.

  • Approximately 630,000 HIV-related deaths happened in 2023—a nearly 51% decrease since 2010.

Regional distribution

  • Sub-Saharan Africa remains the most impacted region, housing ~26 million people with HIV and accounting for 50% of new infections.

  • In Asia-Pacific, around 6.7 million people live with HIV, with 150,000 AIDS-related deaths reported in 2023.

  • Some regions—Eastern Europe, Central Asia, Middle East, North Africa, and parts of Latin America—are seeing rising new infection rates.

Treatment and Progress

  • Globally, 86% of people living with HIV know their status; 77% (about 30.7 million) are on antiretroviral therapy (ART); 72% of those are virally suppressed.

Real-world success

  • Botswana, Zimbabwe, and other countries have achieved the UNAIDS 95‑95‑95 targets—95% know their status, 95% receive treatment, 95% are virally suppressed.

Published At:
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