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Researchers Harness CRISPR to Remove HIV from Human Immune Cells

Bykimhangeditor

Jul 4, 2025 #HHằng Nick 1

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While today’s HIV treatments merely suppress the virus, this new strategy aims to eliminate it altogether. At the University of Amsterdam, scientists successfully demonstrated proof-of-concept results in lab conditions, while Excision BioTherapeutics has advanced the same idea to early human trials. Despite these encouraging developments, experts caution that CRISPR-based therapies for HIV remain in early stages, requiring much more research to confirm their safety and effectiveness before they could be used in patients.

How CRISPR Targets HIV

CRISPR works like microscopic scissors, cutting DNA precisely to remove harmful sequences. This Nobel Prize–winning tool can seek out and destroy HIV DNA embedded within immune cells with extraordinary accuracy. By programming CRISPR to identify viral genetic code, scientists can target HIV’s hiding spots while sparing healthy cell functions. This differs from current antiretroviral therapies, which block the virus from multiplying but do not remove it from the body. CRISPR’s precision offers a way to attack the virus even in places where standard drugs cannot reach.

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Amsterdam Team Demonstrates Lab-Scale Success

At the University of Amsterdam, researchers presented promising lab results at the ECCMID 2024 conference, showing they could remove HIV DNA from infected cells using advanced CRISPR methods. However, they stressed these findings remain proof-of-concept, not an immediate cure. Dr. James Dixon from the University of Nottingham noted that while lab data is promising, further validation is essential before trying these approaches on humans. These results are only a first step on the path toward gene-editing treatments for HIV.

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Excision BioTherapeutics Pushes into Human Trials

Meanwhile, Excision BioTherapeutics, a San Francisco–based company, has begun the first human trials of a CRISPR-based HIV therapy called EBT-101. About a year ago, they treated three volunteers with an intravenous dose of the gene-editing drug, aiming to eradicate hidden HIV DNA permanently. Their candidate targets HIV-1, the strain responsible for roughly 95% of global HIV cases.

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Early Clinical Data: Safe but Limited Results

After 48 weeks, data showed no serious side effects or dose-limiting toxicities among the first three trial participants. EBT-101 successfully reached the bloodstream, but unfortunately, the virus reemerged once participants stopped their regular antiretroviral drugs. While the therapy proved safe and well-tolerated, it did not meet its primary goal of permanently suppressing HIV, though researchers believe these results will guide improvements in future gene-editing treatments.

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Scientific Hurdles and Safety Concerns

Dr. Jonathan Stoye of the Francis Crick Institute highlighted the immense difficulty of targeting every HIV-infected cell in the body. One key risk is unintended genetic alterations with unpredictable long-term consequences. Because HIV can hide in different tissue reservoirs, reliably delivering CRISPR to all infected cells is technically challenging. Experts believe it will likely take years of further research before these therapies could enter mainstream medical practice, as thorough safety checks are still needed to prevent harming healthy cells.

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Why a Cure Remains Difficult

HIV specifically attacks immune cells and embeds its genetic material inside them, allowing it to go dormant and later reactivate. Even the best current antiretroviral treatments cannot fully remove these hidden infected cells. That is why researchers are trying to use CRISPR to delete HIV’s genetic code permanently rather than just suppressing it.

Today’s HIV Treatments Still Offer Excellent Outcomes

Modern antiretroviral therapy enables people with HIV to live normal, healthy lives if they take their medication consistently. These drugs reduce viral loads to undetectable levels, stopping transmission to sexual partners. Additionally, preventive measures like PrEP offer highly effective protection for those at risk, and are available for free through UK sexual health services. While most HIV patients will continue lifelong treatment, these medical advances have transformed HIV from a deadly illness into a manageable chronic condition.

Next Steps for CRISPR HIV Therapy

Excision BioTherapeutics plans to test higher EBT-101 doses in its second participant group and explore improved delivery systems, including lipid nanoparticles similar to those used in COVID-19 vaccines. Richard Angell from the Terrence Higgins Trust noted that gene-editing HIV cures are still years away but represent meaningful progress. With these scientific advances — alongside strong prevention and treatment programs — experts remain hopeful that the UK can end new HIV transmissions by 2030.