Early in 2016, a woman went to a clinic in Dallas to have a genetically modified adeno-associated virus injected into her eye. The woman was blind due to the degenerative disease retinitis pigmentosa, and the virus carried the gene for a light-responsive algae protein called channelrhodopsin. The goal was to induce her retinal ganglion cells—normally only downstream of cells that detect visual input—to make channelrhodopsin and become light-sensitive, giving her a rudimentary visual sense.
That patient is thought to be the first in the world to have received a therapy based on optogenetics, the principle of using genetic modifications and light stimuli to precisely manipulate cells’ behavior. The technique enables researchers to turn the activity of certain genes in specific cells on or off at the flick of a switch, and has been a boon to biology research. Its fine-tunability also makes it an attractive tool for those searching for ...
