Restoring vision to blind mice (and humans with RP or AMD?) with a photoswitch.

Age-related macular degeneration (AMD) and retinitis pigmentosa (RP) affect millions of people around the world and in their advanced stages lead to blindness. Studies in mouse models of these diseases have shown some promise in restoring vision but are either invasive (i.e., implantation of electronic chips) or irreversible (i.e., transplantation of photoreceptor progenitors or viral expression of optogenetic tools). Tochitsky et al. (click on the link to see the authors' fancy PR video of the work) have now performed introcular injection of a synthetic small molecule called DENAQ which is a red-shifted K+ channel photoswitch that exhibits trans to cis photoisomerization with visible light (450–550 nm) and relaxes rapidly to the trans configuration in the dark A single injection photosensitizes blind retinas with no photoreceptors to daylight intensity white light for a period of days with no toxicity. It restores light-elicited behavior and enables visual learning in blind mice. It is a prime drug candidate for vision restoration in patients with end-stage RP and AMD.

Figure of DENAQ from Mourot et al., ACS Chem. Neurosci., 2011, 2 (9), pp 536–543AMD.