UCL Discovery
UCL home » Library Services » Electronic resources » UCL Discovery

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

Ashtari, M; Zhang, H; Cook, PA; Cyckowski, LL; Shindler, KS; Marshall, KA; Aravand, P; ... Bennett, J; + view all (2015) Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosis. Science Translational Medicine , 7 (296) , Article 296ra110. 10.1126/scitranslmed.aaa8791. Green open access

[thumbnail of STM_Submission_April%2020_Final_template_2.pdf]
Preview
Text
STM_Submission_April%2020_Final_template_2.pdf

Download (1MB) | Preview

Abstract

Much of our knowledge of the mechanisms underlying plasticity in the visual cortex in response to visual impairment, vision restoration, and environmental interactions comes from animal studies. We evaluated human brain plasticity in a group of patients with Leber's congenital amaurosis (LCA), who regained vision through gene therapy. Using non-invasive multimodal neuroimaging methods, we demonstrated that reversing blindness with gene therapy promoted long-term structural plasticity in the visual pathways emanating from the treated retina of LCA patients. The data revealed improvements and normalization along the visual fibers corresponding to the site of retinal injection of the gene therapy vector carrying the therapeutic gene in the treated eye compared to the visual pathway for the untreated eye of LCA patients. After gene therapy, the primary visual pathways (for example, geniculostriate fibers) in the treated retina were similar to those of sighted control subjects, whereas the primary visual pathways of the untreated retina continued to deteriorate. Our 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 eye in LCA patients. The interactions between the eye and the brain enabled improved and sustained long-term visual function in patients with LCA after gene therapy.

Type: Article
Title: Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosis.
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1126/scitranslmed.aaa8791
Publisher version: http://dx.doi.org/10.1126/scitranslmed.aaa8791
Language: English
UCL classification: UCL
UCL > Provost and Vice Provost Offices > UCL BEAMS
UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Engineering Science
UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Engineering Science > Dept of Computer Science
URI: https://discovery.ucl.ac.uk/id/eprint/1470156
Downloads since deposit
110Downloads
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item