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Volume 28 Issue 3-4, April 2021

Cover Credit:Intravitreal injection of AAV-carried Ambient-light activatable Multi-Characteristic Opsin (MCO) led to ON-bipolar cell (green) specific expression (red, reporter mCherry) in rd10 mice retina with complete loss of natural photoreceptors.Gene therapy-based treatment such as optogenetics offers a potentially powerful way to bypass damaged photoreceptors in retinal degenerative diseases and use the remaining retinal cells for functionalization to achieve photosensitivity.This issue of Gene Therapy describes the use of ambient-light activatable Broadband Multi-Characteristic Opsin (MCO) for photosensitizing ON Bipolar cells to restore vision in mice with severe photoreceptor degeneration, mimicking human disease of severe Retinitis Pigmentosa (RP). Theimageshows robust expression of MCO in soma and axonal terminals of bipolar cells of the retina,16 weeks after intravitreal injection of AAV-carriedMCO.See paper by Batabyal et al. in this issue of Gene Therapy.

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