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September 13, 2019 03:30 am

CRISPR Gene-Editing May Offer Path To Cure For HIV, First Published Report Shows

An anonymous reader quotes a report from NPR: Chinese scientists have published the first report in a scientific journal of an attempt to use CRISPR-edited cells in a patient -- a 27-year-old man who is HIV-positive. While the treatment did not rid the man of the AIDS virus, the researchers and others are calling the report promising. That's because it indicates that so far the gene-editing technique seems to safely and effectively make the precise DNA change intended. The case was published in the New England Journal of Medicine. In the new report, the researchers attempted to use CRISPR to recreate the experiences of two men known as the Berlin patient and the London patient. In those cases, HIV-positive men were declared effectively cured after they received stem cell transplants from people born with variations of a gene known as CCR5 that makes people naturally resistant to HIV. The variation disables a molecular gateway HIV uses to enter and destroy key immune system cells. In the new case, [Hongkui Deng, a professor of cell biology at the Peking University] and his colleagues used CRISPR to edit the CCR5 gene on stem cells to recreate the naturally occurring protection against HIV. They then used the edited cells to perform a stem cell transplant for the patient. The man also had acute lymphoblastic leukemia, a form of blood cancer. The transplant appears to have put the patient's leukemia into remission, the researchers reported. He suffered no apparent adverse side effects from the gene-edited cells, which have persisted in his body for more than 19 months, according to the report. "The approach did not reduce levels of HIV in the man's body because only about 5% of his white blood cells carried the edited CCR5 variation," the report adds. "So Deng says his team plans to focus on finding ways to boost that closer to 100%, which is what would be needed to eradicate the virus." Regardless, the findings are important positive outcomes that suggest the approach is safe and could work.

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