Research ArticleGene Therapy

Plasticity of the human visual system after retinal gene therapy in patients with Leber’s congenital amaurosis

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Science Translational Medicine  15 Jul 2015:
Vol. 7, Issue 296, pp. 296ra110
DOI: 10.1126/scitranslmed.aaa8791

Vision restoration shines a light on human brain plasticity

Much of our understanding about the plasticity of the human visual system comes from animal studies. Owing to the success of retinal gene therapy, it is now possible to study this process in humans with Leber’s congenital amaurosis (LCA) after their vision has been restored. Using a multimodal imaging approach, Ashtari et al. assessed both functional and structural changes in the visual pathways of a group of patients who underwent retinal gene therapy for this blinding disease in comparison with a group of sighted controls. Their results suggest that visual experience, enhanced by gene therapy, may be responsible for the reorganization and maturation of synaptic connectivity in the visual pathways of the treated compared to the untreated eye of LCA patients.

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