RESUMEN

Several preclinical studies have investigated the potential of algal channelrhodopsin and human melanopsin as optogenetic tools for vision restoration. In the present study, we assessed the potentially deleterious effects of long-term expression of these optogenes on the diseased retina in a large animal model of retinal degeneration, the RPE65-deficient Briard dog model of Leber congenital amaurosis. Intravitreal injection of adeno-associated virus vectors expressing channelrhodopsin and melanopsin had no effect on retinal thickness over a 16-month period post injection. Our data support the safety of the optogenetic approach for the treatment of blindness.

Asunto(s)

Channelrhodopsins/fisiología , Retina/metabolismo , Degeneración Retiniana/terapia , Opsinas de Bastones/fisiología , Animales , Channelrhodopsins/genética , Channelrhodopsins/metabolismo , Dependovirus/genética , Modelos Animales de Enfermedad , Perros , Electrorretinografía/métodos , Proteínas del Ojo/genética , Regulación de la Expresión Génica/genética , Técnicas de Transferencia de Gen , Terapia Genética/métodos , Vectores Genéticos/genética , Células HEK293 , Humanos , Amaurosis Congénita de Leber/terapia , Retina/fisiología , Opsinas de Bastones/genética , Opsinas de Bastones/metabolismo , Visión Ocular/fisiología