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Optogenetics for retinal disease

Allen C. Ho, MD, Director of Retina Research at Wills Eye Hospital, discusses how optogenetics, a mutation-agnostic gene therapy approach offers new possibilities for patients with advanced retinal disease. Optogenetics uses gene therapy strategies to introduce light-sensitive transmembrane proteins called opsins into the retina. Unlike gene replacement therapies targeting specific genetic mutations, optogenetics is both mutation- and disease-agnostic. Optogenetic strategies use remaining retinal cells (eg, bipolar and ganglion cells) to regain light sensitivity and restore a degree of functional vision,9 opening new possibilities for individuals with severe vision loss and potentially transforming the management of conditions such as RP and Stargardt disease.

View the article online in Modern Retina.