A “promising cure” for HIV and AIDS has been discovered, according to scientists who managed to almost entirely eliminate the devastating immune disease from infected mice.
The researchers said they had demonstrated the “feasibility and efficiency” of removing the HIV-1 provirus using a gene-editing technique called CRISPR.
They admitted there were still some practical problems to be overcome, but suggested their work was a “significant step” towards carrying out clinical trials of the technique in humans.
Writing in the journal Molecular Therapy, the scientists described how some of the mice had been ‘humanised’ by being given some of our immune cells.
In these animals, “successful proviral excision was detected … in the spleen, lungs, heart, colon, and brain after a single intravenous injection” of the gene-editing protein.
Apparent breakthroughs in animal models often encounter problems later in the process of developing a treatment for humans.
Nonetheless, the researchers, from Temple University and Pittsburgh University in the US, wrote in the journal that this type of genome editing "provides a promising cure for HIV-1/AIDS”.
“Here, we demonstrate the feasibility and efficiency of excising the HIV-1 provirus in three different animal models,” they wrote.
“Excision of HIV-1 proviral DNA by [this method in a living animal] in solid tissues/organs can be achieved … [in] a significant step toward human clinical trials.
"To our knowledge, this study is the first to demonstrate the effective excision of HIV-1 proviral DNA from the host genome in pre-clinical animal models [using this method]."
However, they added that “gene delivery efficiency … remains an obstacle to overcome” in a living animal.
The researchers told The Daily Mail that the next step would be to repeat the study in primates, described as a “more suitable animal model where HIV infection induces disease” before the “eventual goal” of human clinical trials.
Dr Wenhui Hu, of Temple University, told the Mail the new study built on earlier research but was “more comprehensive”.
“We confirmed the data from our previous work and have improved the efficiency of our gene editing strategy,” Dr Hu said.
“We also show that the strategy is effective in two additional mouse models, one representing acute infection in mouse cells and the other representing chronic, or latent, infection in human cells.”
