Gérard M.B. Péan
What in the world is optogenetics and what does it have to do with technology?
The answer is a complicated if you’re not in the field but let me give some insight. You see, “Optogenetics is the combination of genetics and optics to control well-defined events within specific cells of living tissue. It includes the discovery and insertion into cells of genes that confer light responsiveness; it also includes the associated technologies for delivering light deep into organisms as complex as freely moving mammals, for targeting light-sensitivity to cells of interest, and for assessing specific readouts, or effects, of this optical control”.
Now there is a start-up company who strongly believe that they possess the skills and know-how to actually cure blindness or restore vision in blind patients with gene therapy. by using newly developed techniques, the company “Retrosense Therapeutics is geared to cure the age old disease of blindness. Scientists have used the technique over the last few years as a research tool to study brain circuits and the neural control of behavior by directing neuron activity with flashes of light. Retrosense and others groups are pushing to bring the technique to patients in clinical trials”.
“The idea behind Retrosense’s experimental therapy is to use optogenetics to treat patients who have lost their vision due to retinal degenerative diseases such as retinitis pigmentosa. Patients with retinitis pigmentosa experience progressive and irreversible vision loss because the rods and cones of their eyes die due to an inherited condition. If the company is successful, the treatment could also help patients with the most common form of macular degeneration, which affects nearly a million people in the United States. The U.S. Food and Drug Administration hasn’t approved any therapies for either condition”.
“Doctors would inject a non-disease causing virus into a patient’s eye. The virus would carry the genetic information needed to produce the light-sensitive channel proteins in the ganglion cells. Normally, rods, cones, and other cells translate light information into a code of neuron-firing patterns that is then transmitted via the ganglion cells into the brain. Since Retrosense’s therapy would bypass that information processing, it may require the brain to learn how to interpret the signals.
So far, Retrosense and its academic collaborators have shown that the treatment can restore some vision-evoked behaviors in rodents. The treatment also seems safe in nonhuman primates. The optogenetically modified ganglion cells of these primates are light-responsive, but behavioral tests aren’t possible, as there are no nonhuman primate models of retinal degeneration, says Retrosense CEO Sean Ainsworth.
Retrosense plans to begin its first clinical trial in 2013 with nine blind retinitis pigmentosa patients”.
I don’t expect this to happen overnight but, it’s a great start at solving the dilemma of curing blindness. For those of you with family members who suffer from lost of sight, this definitely gives hope that one day your loved ones will be able to gain or regain the ability to see. What are your thoughts? Voice your opinion!