Scientists cure mice of HIV for first time in groundbreaking study using CRISPR

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The groundbreaking study, published in the journal Nature Communications, revealed that treatment to suppress HIV replication coupled with gene editing therapy can eliminate HIV from infected cells and organs.

Current HIV treatment focuses on the life-long use of antiretroviral therapy (ART), which suppresses HIV replication but does not eliminate the virus. Dr Kamel Khalili, a senior investigator of the study, had found in previous work that by using the gene editing CRISPR-Cas9 technology, large fragments of HIV DNA could be removed from infected cells.

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For the new study, Khalili and his colleagues combined the gene editing system with a recently developed therapeutic strategy known as long-acting slow-effective release (LASER) ART. LASER limits HIV replication to low levels for extended periods of time, potentially allowing time for the gene editing phase of the test to kick in.

The newly modified drug was packaged into nanocrystals, which can be delivered to the HIV infected tissues and slowly release the drug for weeks.

The researchers then used a combination of LASER ART and the CRISPR-Cas9 for treatment on mice specially engineered to produce human cells susceptible to HIV. Remarkably, results showed a complete elimination of HIV DNA in about one third of the HIV-infected mice, according to the team.

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Dr Howard Gendelman, co-lead of the study, said the researchers now have a “clear path to move ahead to trials in non-human primates and possibly clinical trials in human patients within the year.