Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice.

ABSTRACT: 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. However, current approaches of optogenetic treatment rely on opsins that require high intensity light for activation thus adding to the challenge for use as part of a wearable device. Here, we report AAV2 assisted delivery of highly photosensitive multi-characteristic opsin (MCO1) into ON-bipolar cells of mice with retinal degeneration to allow activation by ambient light. Rigorous characterization of delivery efficacy by different doses of AAV2 carrying MCO1 (vMCO1) into targeted cells showed durable expression over 6 months after delivery as measured by r

SUBMITTER: Batabyal S 

PROVIDER: S-EPMC9191254 | biostudies-literature | 2021 Apr

REPOSITORIES: biostudies-literature