Simple injection into the eye could help treat blind patients, finds study.
Discovered by a team of University of California, Berkeley, scientists in collaboration with researchers at the University of Munich and University of Washington in Seattle, the chemical temporarily restores some vision to blind mice.
The team is now working on an improved compound that may someday allow people with degenerative blindness to see again.
The approach could eventually help those with retinitis pigmentosa, a genetic disease that is the most common inherited form of blindness, as well as age-related macular degeneration, the most common cause of acquired blindness in the developed world. In both diseases, the light sensitive cells in the retina - the rods and cones - die, leaving the eye without functional photoreceptors.
The chemical, called AAQ, acts by making the remaining, normally "blind" cells in the retina sensitive to light, said lead researcher Richard Kramer, UC Berkeley professor of molecular and cell biology.
AAQ is a photoswitch that binds to protein ion channels on the surface of retinal cells. When switched on by light, AAQ alters the flow of ions through the channels and activates these neurons much the way rods and cones are activated by light.
"This is similar to the way local anesthetics work: they embed themselves in ion channels and stick around for a long time, so that you stay numb for a long time. Our molecule is different in that it's light sensitive, so you can turn it on and off and turn on or off neural activity," Kramer said.
Because the chemical eventually wears off, it may offer a safer alternative to other experimental approaches for restoring sight, such as gene or stem cell therapies, which permanently change the retina. It is also less invasive than implanting light-sensitive chips in the eye.
"The advantage of this approach is that it is a simple chemical, which means that you can change the dosage, you can use it in combination with other therapies, or you can discontinue the therapy if you don't like the results. As improved chemicals become available, you could offer them to patients. You can't do that when you surgically implant a chip or after you genetically modify somebody," Kramer said.
"This is a major advance in the field of vision restoration," said co-author Dr. Russell Van Gelder, an ophthalmologist and chair of the Department of Ophthalmology at the University of Washington, Seattle.
The blind mice in the experiment had genetic mutations that made their rods and cones die within months of birth and inactivated other photopigments in the eye.
After injecting very small amounts of AAQ into the eyes of the blind mice, Kramer and his colleagues confirmed that they had restored light sensitivity because the mice's pupils contracted in bright light, and the mice showed light avoidance, a typical rodent behaviour impossible without the animals being able to see some light.
Kramer is hoping to conduct more sophisticated vision tests in rodents injected with the next generation of the compound.
The findings have been in the journal Neuron.