Development of a Retinal Ganglion Cell Specific Gene Therapy Using SIRT1 Signaling for Neuro-Protection (Video)

Loss of retinal ganglion cells (RGC) can occur by trauma, inflammatory, and ischemia leading to irreversible effects upon vision. Sirtuin 1 (SIRT1) deacetylase has demonstrated therapeutic value in multiple models of optic neuropathy with small molecule biologics. The initial use of gene therapy modestly improved visual outcomes in the EAE-induced mouse model of optic neuritis but with limited effects due to a low transduction rate and lack of cell specificity. Here we investigated the therapeutic potential of RGC specific SIRT1 gene augmentation in a mouse model of optic nerve crush (ONC).