Gene Editing Tool Can Erase HIV?
Researchers at University of California, San Francisco may have found a method of genetic engineering that can cure HIV.
A miniscule percentage of people have a genetic mutation that makes them resistant to HIV. These individuals' bone marrow has been used to cure HIV before, famously in the case of Timothy Ray Brown, who was cured of HIV after a bone marrow transplant in which the donor had this mutation. But the potential donors are far too scarce, and a bone marrow transplant too invasive, for this method to serve as a practical cure for HIV.
As a result, researchers have turned to induced pluripotent stem cells (iPSCs) in order to give HIV sufferers this genetic mutation. In order to alter these stem cells to have the genetic mutation, Yuet Kan et al used a particularly precise method of genetic editing. This method is inspired by a bacterial immune system, in which the system splices fragments of the viruses' DNA into their own in order to "flag" them as intruders. The method used by the researchers, in which a specific DNA sequence is replaced by another, is much more efficient than other types of genetic engineering, in which fragments of the foreign DNA are introduced essentially at random.
After the genetic editing, the iPSCs grow into white blood cells that are immune to the ravages of HIV. By using DNA from a specific patient, this method could allow for personalized therapy for and even cure of HIV.
Kan's research method is similar to that of the researchers behind the Resilience Project, which studies healthy genetic mutants to cure diseases with genetic components.
Kan warns that we are still a long way off from a widely distributed treatment, as he still has not grown the pluripotent cells into the specific cells that are decimated by HIV. Kan may be able to transplant the cells in such a way that they would grow into all different kinds of cells in the body, but then, Kan explains, "One of the problems is converting iPSCs into a type of cell that is transplantable." Many hurdles will need to be overcome in order to prove this treatment effective and safe, but this research is a huge step towards finding a cure for a disease that long been considered to be incurable.