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Translational Science With Clinical Promise
September 2014

The First International Optogenetic Therapies for Vision SymposiumTranslational Medicine in Progress

Author Affiliations
  • 1Institute of Ophthalmology and Visual Science, Rutgers–New Jersey Medical School, Rutgers University, Newark

Copyright 2014 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA Ophthalmol. 2014;132(9):1043-1044. doi:10.1001/jamaophthalmol.2014.1420

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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