Mice cured of HIV in gene breakthrough ‘with human trials within a year’
A breakthrough gene therapy has fully cured mice of HIV, the virus which causes AIDS, and human trials could follow within a year.
The treatment is different to current HIV therapies, which rely on patients taking ART (antiretroviral therapy) pills for the whole of their lives.
Mice had the virus eliminated from their cells using a revolutionary gene-editing tool, along with drugs which target ‘reservoirs’ of HIV in the body..
Senior investigator Professor Kamel Khalili, an AIDS expert at Temple University in Philadelphia, said: ‘Our study shows treatment to suppress HIV replication and gene editing therapy, when given sequentially, can eliminate HIV from cells and organs of infected animals.’
His team used a technique called CRISPR-Cas9 that can snip faulty DNA - which has been likened to a pair of 'molecular scissors'.
It combines this with a recently developed therapeutic strategy known as LASER (long-acting slow-effective release) ART.
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It targets viral 'sanctuaries' - maintaining HIV replication at low levels for extended periods of time. This also reduces the frequency of treatments.
The long-lasting medications were made possible by pharmacological changes in the chemical structure of antiretroviral drugs.
HIV rebound is directly attributed to the ability of the virus to integrate its DNA sequence into the genomes of cells of the immune system, where it lies dormant and beyond the reach of antiretroviral drugs.
In the study published in Nature Communications the modified drug was packaged into nanocrystals, which readily distribute to tissues where HIV is likely to be lying dormant.
From there, the nanocrystals, stored within cells for weeks, slowly release the drug.
Dr Khalili explained: 'We wanted to see whether LASER ART could suppress HIV replication long enough for CRISPR-Cas9 to completely rid cells of viral DNA.'
'The big message of this work is that it takes both CRISPR-Cas9 and virus suppression through a method such as LASER ART, administered together, to produce a cure for HIV infection.
'We now have a clear path to move ahead to trials in non-human primates and possibly clinical trials in human patients within the year.'
