Vertebrate opsins for vision restoration

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Our most cherished sense, vision, begins with the process of phototransduction, a process performed by the highly specialized photoreceptor cells of the retina. Seven transmembrane proteins are responsible for light capture and they have evolved into many different forms through evolution. In majority of inherited blinding diseases photoreceptor cells are altered losing the ability to capture light. To re-animate retinas that have lost their endogenous opsins it has been suggested that simple one-component microbial opsins can be expressed in these dormant photoreceptors. This has lead to elegant proof-of-concept studies in rodents showing that it is possible to restore visual function. However, the major drawback with these microbial opsin systems is their low light sensitivity, their potential immunogenicity in humans and difficulty of their expression in higher primates. Here, we propose to use human cone opsin systems specifically designed to work in remaining retinal neurons in rod-cone dystrophies. These vertebrate cone opsins including their modulated ion channels in combination with new targeting strategies to cones and ganglion cells will circumvent the shortcomings associated with one-component microbial opsins, and offer a highly therapeutically relevant solution to blindness, applicable in the clinic.