Single-use gene therapy may control HIV

Researchers at UCLA say they've gathered solid clinical data that supports the potential single use of gene therapy to combat HIV.

In a blinded study that compared the gene therapy with a dummy therapy, a group of patients were given blood stem cells that were modified with the OZ1 molecule. Following 48 weeks of treatment, the researchers found no difference in HIV levels between the two groups. After 100 weeks, though, the gene therapy group had higher levels of CD4+ immune cells, which are destroyed by HIV.

The research team was particularly excited about the possibility of offering a new treatment that could be delivered only once or a handful of times. While currently used drug cocktails have been very effective at controlling HIV, patients often build up immunity or experience adverse reactions.

"While this treatment is far from being perfected, it is not yet as effective or as complete as current antiretroviral therapy in controlling HIV, the study did show proof of concept that inserting and administering a single anti-HIV gene in the patients' own blood stem cells and giving it back to them could reduce viral replication to some degree when anti-HIV medications are stopped," said Professor Ronald Mitsuyasu.

- read the story from the BBC

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