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 (green layer), a layer of cells that supports the light-sensing photoreceptor cells.
Researchers from the University of Pennsylvania have developed a gene therapy that successfully treats a form of macular degeneration in a canine model. The work sets the stage for translating the findings into a human therapy for an inherited disease that results in a progressive loss of central vision and which is currently untreatable...
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