ISTOCK, COSMIN4000Early 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 more-effective treatments for blindness and other conditions.
“I believe restoring vision probably will be one of the most promising applications for optogenetics,” says Zhuo-Hua Pan, a vision researcher at Wayne State University. Pan first learned about channelrhodopsins in a 2003 paper by another group, and soon afterward, his lab ...
