In the current Phase 1 trial, which is ongoing, scientists used three of the four factors to perform a modified reprogramming of diseased retinal ganglion cells in the eye in people with a form of glaucoma—not returning the cells all the way back to their embryonic-like state, but to a point before they stopped functioning properly. Retinal ganglion cells have long arms that connect them from the retina in the eye to the optic nerve, which transmits visual signals to the brain. In some people, over time, fluid in the eye fails to drain properly, putting pressure on the retinal ganglion cells, which lose their delicate connections to the optic nerve, causing loss of vision. While the cells don’t necessarily die, they become dramatically less able to do their job of transmitting critical visual signals. To try to restore their function, in the trial, scientists injected specially designed proteins, or transcription factors, coding for three of the genes into the vitreous: the gel-like fluid cavity at the back of the eye near the retina. It’s an advantageous location to test such a therapy, since it’s protected from the body’s immune defenses which could attack the newly made proteins...
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