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PEX1

This program is focused on developing and testing a gene therapy construct that may ultimately improve the well-being of patients with a disabling disorder by treating retinal degeneration. Retinal degeneration leading to blindness is a major, untreatable feature of PBD-ZSD. In fact, visual improvement is a critical symptomatic target that can substantially improve quality of life of patients. The collaborators propose to test the gene therapy targeting retinal photoreceptor cells in PEX1 animal models to study recovery of peroxisomal function.