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

Marta Figueiredoby Marta Figueiredo

In Batten Disease News.

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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