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Gene Ther. 2012 Feb;19(2):169-75. doi: 10.1038/gt.2011.155. Epub 2011 Oct 13.

Optogenetic therapy for retinitis pigmentosa.

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Neural Circuit Laboratories, Friedrich Miescher Institute for Biomedical Research, Basel, Switzerland.


Retinitis pigmentosa (RP) refers to a diverse group of progressive, hereditary diseases of the retina that lead to incurable blindness and affect two million people worldwide. Artificial photoreceptors constructed by gene delivery of light-activated channels or pumps ('optogenetic tools') to surviving cell types in the remaining retinal circuit has been shown to restore photosensitivity in animal models of RP at the level of the retina and cortex as well as behaviorally. The translational potential of this optogenetic approach has been evaluated using in vitro studies involving post-mortem human retinas. Here, we review recent developments in this expanding field and discuss the potential and limitations of optogenetic engineering for the treatment of RP.

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