Multi-characteristic opsin restores vision in Stargardt mice model

Subrata Batabyal; Adnan Dibas; Houssam Al-Saad; Sanghoon Kim; Samarendra K. Mohanty

doi:10.1117/12.2614730

7 March 2022 Multi-characteristic opsin restores vision in Stargardt mice model

Subrata Batabyal, Adnan Dibas, Houssam Al-Saad, Sanghoon Kim, Samarendra K. Mohanty

Proceedings Volume PC11947, Optogenetics and Optical Manipulation 2022; PC119470B (2022) https://doi.org/10.1117/12.2614730
Event: SPIE BiOS, 2022, San Francisco, California, United States

Abstract

Stargardt Disease is an inherited macular degeneration caused by mutation of genes, primarily ABCA4 in the photoreceptor cells leading to their dysfunction and degeneration in the macula. Here, we report results of ambient-light vision restoration in Stargardt mice (Abca4tm1Ght/J) upon intravitreal delivery of AAV-carried MCO (vMCO) as evaluated by electrophysiology, Optical Coherence Tomography (OCT) and visually guided behavior. Further, safety of MCO-enabled vision restoration therapy was evaluated by measurement of Intraocular Pressure, OCT and Immunohistochemistry. The vMCO based ambient light activatable optogenetic therapy has potential to be a disease-agnostic therapy to address the unmet need of inherited retinal degenerative diseases.

Conference Presentation

Citation Download Citation

Subrata Batabyal, Adnan Dibas, Houssam Al-Saad, Sanghoon Kim, and Samarendra K. Mohanty "Multi-characteristic opsin restores vision in Stargardt mice model", Proc. SPIE PC11947, Optogenetics and Optical Manipulation 2022, PC119470B (7 March 2022); https://doi.org/10.1117/12.2614730

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