Multi-Characteristic Opsin Restores Vision In Stargardt Mice Model
Presented by Sanghoon Kim, PhD at SPIE BIOS
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.