Spatially targeted non-viral optical delivery of opsin-encoding genes into primate retina

Degeneration of photoreceptors, retinal cells including retinal pigment epithelium, and retinal ganglion cells characterizes visual disorders involving retinal dysfunction. The efficient and targeted gene delivery into retinal cells is critical for treatment of a variety of visual disorders. Here, we report use of an OCT guided, spatially targeted, near-infrared laser microirradiation platform to successfully deliver genes encoding ambient-light activatable multi-characteristic opsin (MCO) to retinal cells in non-human primates. The efficacy of gene delivery was evaluated by fundus imaging of the reporter (mCherry) fluorescence, and immunohistochemistry. Functional improvement was measured by red-ERG which was attributed to MCO’s broadband activation spectrum.