A New Scientist article describes a study in which researchers used two cutting-edge technologies to make very specific changes in the DNA of stem cells, cells that are able to turn into any other type of cell, replicating a naturally occurring mutation in a gene that is associated with HIV resistance. While previous gene therapies often made unpredictable changes in the cell’s genetic structure, these researchers showed an efficient method of disrupting a specific gene without making any other changes in DNA, removing unacceptable side-effects.
Previously, it has been shown that a bone marrow transplant from someone with this mutation may be able to cure HIV. The problem is the low chance of finding a transplant match who also happens to have this mutation. Therefore researchers turned to editing the genes of stem cells, which are produced from a patient’s own cells and could produce a large supply of the desired cell type. They used a technique developed by other researchers to change these stem cells into white blood cells, the target of the HIV virus. Like white blood cells from patients with this mutation, white blood cells made from the modified stem cells were resistant to HIV infection.
While this is an exciting step forward in the search for a cure for HIV, this is just one piece of a larger puzzle. There is still a lot of work to do to determine whether white blood cells made from stem cells really behave exactly like regular white blood cells before this type of treatment is available.
Special thanks to Mara Inniss from the Harvard Biological and Biomedical Sciences Program for her expertise and comments on the topic. Edited by Ankita Shastri.