Thanks to gene editing, we’ve seen much progress in hard-to-treat conditions. Sufferers of both muscular dystrophy and leukemia are experiencing a new variety of treatment options. Now, thanks to CRISPR, a renowned gene editing tool, researchers have increased HIV resistance in animals.
A minor proportion of people harbor a homozygous mutation in CCR5—a gene that encodes a receptor found on immune cells—that thwarts HIV’s attempts to get inside the cells. In an attempt to mimic this natural resistance, researchers mutated CCR5 in human fetal liver hematopoietic stem/progenitor cells (HSPCs) and showed that the cells could block HIV infection after transplantation into mice.
Don’t let the medical jargon fool you — while the procedure may be complicated, the concept itself is a lot simpler. By replicating a naturally occurring genetic mutation, T-cells become more resistant to viruses. But results were slow, and researchers were patient, to say the least.
“The long-term reconstitution and secondary transplantation were time-consuming. It took us more than one-year monitoring of the mice to confirm the gene editing is robust in long-term HSCs,”
The study may not have been the first to incorporate gene editing, but it is the first to use CRISPR. We may not have engineered a complete cure (after all, we’ve only targeted mice), but finding one wouldn’t seem too improbable.
Moral of the story? Take risks. Sponsor a child genius. Our future depends on them.