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Beckwith-Cohen and colleagues report on successful gene augmentation therapy in a naturally occurring large-animal dog model of cone-rod synaptic disorder due to a mutation in CaBP4. Affected dogs exhibit synaptic immaturity and disorganization, which are rescued even in later stages of the disease supporting significant retinal plasticity.
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Gene augmentation therapy induces synaptic ribbon maturation, synaptogenesis and vision restoration in an adult dog model of retinal degeneration.
Molecular Therapy Family of Journals