Gene therapy of HIV infection
Experimental treatment defeated HIV for the first time
A group of molecular biologists led by Kamel Khalili from Temple University, using CRISPR/Cas9 gene editing technology, for the first time managed to successfully cut a segment of DNA belonging to HIV from the genome of living animals. The results of the work are published in the journal Gene Therapy (Kaminski et al., Excision of HIV-1 DNA by gene editing: a proof-of-concept in vivo study – VM). A press release by Researchers from Lewis Katz School of Medicine at Temple University Successfully Excise HIV DNA from Animals is published on the university's website.
In their early studies, scientists were able to show that the CRISPR/Cas9 system is able to remove the virus genes embedded in the genome of the host cell without adversely affecting the latter. However, the tests were carried out on cell cultures taken from patients infected with HIV, and so far there has been no evidence that the method will work on a living organism.
In the latest work, biologists have tested whether gene editing technology can eliminate HIV from the body of transgenic rats and mice in which the virus genes have been implanted in every cell of every organ. To introduce a CRISPR system modified to remove virus genes into animals, scientists used a recombinant adeno-associated virus that contained the necessary nucleotide sequences, as well as the genes of the Cas protein.
Two weeks after the start of the experiment, the researchers analyzed the genomes of the animals. The target segment of HIV was cut from DNA in every tissue, including brain, kidney, liver, lung, spleen and blood cells. The analysis of viral RNA showed that its amount decreased significantly in lymphocyte cells, as well as in lymph nodes.
HIV treatment is usually carried out with the help of antiretroviral drugs that suppress the reproduction of the virus, but do not have the ability to remove it from infected cells. HIV is able to embed its genes into the cells of the immune system, forcing them to produce their own copies.
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