Gene Therapy Directly to Brain Helps Preserve Vision in CLN6 Mouse Model

Gene Therapy Directly to Brain Helps Preserve Vision in CLN6 Mouse Model

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Gene Therapy Directly to Brain Helps Preserve Vision in CLN6 Mouse Model

A single injection into the brain of an investigational gene therapy now in clinical trials partly prevented retinal degeneration and vision loss in a mouse model of CLN6 Batten disease, a study shows. These findings mark the first report of a brain-delivered gene therapy reaching both the brain and eye, and may help optimize gene therapies for CLN6 disease and other neurodegenerative diseases affecting the retina — the eye layer that sends vision signals to the…

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