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Viewpoint | Translational Science With Clinical Promise

The First International Optogenetic Therapies for Vision Symposium Translational Medicine in Progress

Marco A. Zarbin, MD, PhD1
[+] Author Affiliations
1Institute of Ophthalmology and Visual Science, Rutgers–New Jersey Medical School, Rutgers University, Newark
JAMA Ophthalmol. 2014;132(9):1043-1044. doi:10.1001/jamaophthalmol.2014.1420.
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Optogenetics is a technology that uses gene therapy to induce the expression of light-activated ion channels in cells, which renders the cells light-sensitive. When these cells are illuminated with light of the proper wavelength, membrane depolarization (or hyperpolarization) is induced by the light-sensitive ion channels, and if the cells are neurons, synaptic transmission can be initiated (or inhibited). Optogenetics has the potential for minimally invasive neuronal stimulation with high spatial resolution. By proper selection of promoters, the expression of these light-sensitive ion channels can be limited to specific subpopulations of neurons. One might, for example, transfect ganglion cells with light-sensitive ion channels by an intravitreal injection of an adeno-associated virus vector containing a naturally occurring or man-made ion channel coupled to a promoter that limits expression to retinal ganglion cells.

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