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OXR1 Gene Therapy

We have constructed AAV8-OXR1 vectors to deliver the OXR1 gene to photoreceptor cells, which are specialized neurons that respond to light and transmit electrical signals to the brain via intermediary neurons and the optic nerve. Photoreceptors are highly oxidizing neurons that are terminally differentiated and once lost cannot be replaced resulting in blindness. In most neurodegenerative diseases, oxidative stress induced cell death is a major contributing factor to neurodegeneration. OXR1appears to function by regulating many genes that protect cells from oxidative stress. Increasing OXR1 levels in cells increases stress resistance to oxidative stress and provides increased protection from oxidative stress induced cell death. To date we have used the retinal degeneration 1 (rd1) mutant mouse model and treated it by sub-retinal injection of AAV-OXR1. rd1 mice normally lose all visual function by 4 weeks of age. We find that after treatment visual function remains active up to 10 weeks of age. OXR1 gene therapy is not a gene specific, therefore, we expect it to prevent or retard neurodegeneration in multiple diseases. Our current work is examining the ability of OXR1 to retard neurodegeneration in multiple disease models.