Biology-Chemistry Joint Colloquium | Subrata Batabyal
December 1, 2020 @ 6:00 pm - 7:00 pm
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Title: Gene Therapy for Treating Blindness
Speaker: Dr. Subrata Batabyal
Abstract: Retinal disorders such as Retinitis pigmentosa or advanced Dry Age-related Macular Degeneration (Dry-AMD) are degenerative eye diseases that cause severe vision impairment and currently do not have any approved treatment. Such disorders are characterized by degeneration of photoreceptors in the eye, which hinders visual ability due to non-photoactivation of the retina, and the lack of transmission of signals to the visual cortex. 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. In this talk, I will discuss the use of highly sensitive, ambient light activatable multi-characteristic opsin (MCO) for the treatment of visual disorders. I will present the safety and efficacy data for AAV2-MCO based optogenetics therapy for rescuing vision in animal models as well as human subjects. I will also discuss and present data on NextGen non-viral laser-based targeted delivery as a minimally invasive approach to reliably deliver therapeutic molecules/genes into targeted retinal neurons in-vivo for functional recovery of retinal function.