Until now, gene editing has relied on cell division to propagate modifications made with techniques like CRISPR Cas9. Researchers at the Salk Institute have devised a new method that can modify the genes of non-dividing cells (the majority of adult cells). They demonstrated the method’s potential by inserting missing genes into the brains of young mice that were blind due to retinitis pigmentosa. After the team inserted fully functional copies of the damaged gene responsible for the condition into the relevant visual neurons, the mice experience rudimentary vision.
Team leader Izpisua Belmonte says of the new method, homology-independent targeted integration (HITI), “We now have a technology that allows us to modify the DNA of non-dividing cells, to fix broken genes in the brain, heart and liver. It allows us for the first time to be able to dream of curing diseases that we couldn’t before, which is exciting.”
While the team, naturally and appropriately, envisions therapeutic uses, could this method be used to modify brain function non-therapeutically, to improve normal functioning, for example?