Treating Best Disease with Gene Therapy (IMAGE)

University of Pennsylvania

Treating Best Disease with Gene Therapy

Caption

Gene therapy successfully treated a canine version of Best disease, a blinding disorder, the effects lasting more than five years. In these images of the retina of untreated (left) and treated (right) dogs, one can see that BEST1 gene expression (in red) was restored following treatment. In addition, the threrapy restored the structure of the RPE (bottom green layer), a layer of cells that supports the light-sensing photoreceptor cells.

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University of Pennsylvania

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