The approach, which is still in experimental stages, takes benefits of a rare mutation that makes 1% of individuals of European descent resistant to HIV.
Applying a new “genome editing” tool, scientists are expecting to be capable to put in the mutation into the cells of other individuals – and they have previously shown the basic principles work using induced pluripotent stem cells (iPSCs).
The new genome modifying method is much more accurate than tradition types of genetic engineering, as it places a series of gene into a specific area of the genome, instead of at random areas.
By applying this approach, lead investigator by Yuet Kan from the University of California, have managed to modify the genome of iPSCs, which can switch into any cell in the human body. As expected, when the researchers matured these iPSCs into white blood cells, they were tolerant to HIV. Their study is presented in the Proceedings of the National Academy of Sciences.
The mutation they are insert leads to individuals to carry two copies of a mutated gene for a protein known as CCR5, which the HIV virus has to lock onto before it can get into white blood cells. The mutation stops it from doing that.
The study was motivated by a man known as Timothy Ray Brown, who was famously “cured” after obtaining a bone marrow transplant from a naturally HIV-resistant individual. This new approach would perform in an identical way, but without require someone else’s bone marrow.
However, even though it works in the lab, there is still a long way to go prior to the therapy could be used in human beings.
The scientists now prepare to turn the iPSCs into blood-forming stem cells, which, when replanted into a patient, would give rise to all the various kinds of blood cells – such as the specific white blood cell attacked by HIV.