Their report was published the journal Cell Stem Cell and states that genome editing could prove to be an efficient way of prevent HIV replication.
Using gene-editing technology CRISPR/Cas, researchers were able to “edit” relevant genes out of the DNA of stem and T-cells that would block HIV from invading the T cell. In theory, the edited cells would be reintroduced to an HIV patient via a bone marrow transplant that would create an immune system resistant to HIV.
The gene therapy targets the cell receptor CCR5, which acts as a doorway to the cell. Once in the cell, HIV replicates and kills the host cell, leaving suffers open to opportunistic infections. By removing the CCR5, researchers believe they can prevent HIV from entering the cell and replicating.
“We showed that you can knock out CCR5 very efficaciously, we showed that the cells are still functional, and we did very, very deep sequencing analysis to show that there were no unwanted mutations, so it appears to be safe,” Chad Cowan, one of the head researchers of the study, said, “there is obviously much more work to do."
The therapy isn’t too different from the treatment Timothy Ray Brown, aka the Berlin Patient, received. Brown was suffering from both HIV and leukemia when doctors gave him a bone marrow transplant from a donor with a genetic defect that left the donor free of CCR5 receptors. Brown remains HIV “free” six years after the transplant.
The next step in testing the therapy will be animal trials and human trials could begin in as little as five years, though researchers remain cautious in their optimism.