Next-Generation Genome Engineering & Chemo-genetic Platform Technologies for Gene Therapy of Ocular Disorders

Next-Generation Genome Engineering & Chemo-genetic Platform Technologies for Gene Therapy of Ocular Disorders (Prof. JG Rao, Biological sciences and Bioengineering)

A multidisciplinary team from the department of Biological Sciences & Bioengineering will be utilizing chemogenetic receptor approach in combination with AAV based gene replacement or gene-editing therapy to treat LCA2. The research team anticipates that this will involve deciphering the novel molecular regulators of visual cycle where a combination of molecular and structural biology approach is required to validate the findings. Furthermore, it is expected the development of novel AAV based technologies for gene replacement/ genome engineering and optimal non-invasive delivery system for chemo-genetic triggers as part of these efforts.