Restoration of retinal and visual function in RPE65 mutant dogs by subretinal AAV-RPE65 therapy
Dark-adapted ERGs evoked by increasing intensities of blue light stimuli in a control dog (left) are compared with those evoked using the same stimuli in an RPE65 mutant dog (middle). After subretinal AAV-RPE65 therapy (right), the mutant dog shows an improved response, although not to normal levels, as well as an ERG waveform shape that is now similar to that of the control dog.
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Can you stop LCA, a form of blindness caused by a change in a single base pair, simply by injecting a viral vector with the right nucleotide sequence? Unfortunately, gene therapy is not this simple.
