{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T02:04:03Z","timestamp":1773281043683,"version":"3.50.1"},"reference-count":115,"publisher":"Wiley","license":[{"start":{"date-parts":[[2018,1,1]],"date-time":"2018-01-01T00:00:00Z","timestamp":1514764800000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Jukes Glaucoma Research Fund"},{"name":"Cambridge Eye Trust"},{"DOI":"10.13039\/100002089","name":"Fight for Sight","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002089","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Neural Plasticity"],"published-print":{"date-parts":[[2018]]},"abstract":"<jats:p>The eye is at the forefront of the application of gene therapy techniques to medicine. In the United States, a gene therapy treatment for Leber\u2019s congenital amaurosis, a rare inherited retinal disease, recently became the first gene therapy to be approved by the FDA for the treatment of disease caused by mutations in a specific gene. Phase III clinical trials of gene therapy for other single-gene defect diseases of the retina and optic nerve are also currently underway. However, for optic nerve diseases not caused by single-gene defects, gene therapy strategies are likely to focus on slowing or preventing neuronal death through the expression of neuroprotective agents. In addition to these strategies, there has also been recent interest in the potential use of precise genome editing techniques to treat ocular disease. This review focuses on recent developments in gene therapy techniques for the treatment of glaucoma and Leber\u2019s hereditary optic neuropathy (LHON). We discuss recent successes in clinical trials for the treatment of LHON using gene supplementation therapy, promising neuroprotective strategies that have been employed in animal models of glaucoma and the potential use of genome editing techniques in treating optic nerve disease.<\/jats:p>","DOI":"10.1155\/2018\/7108948","type":"journal-article","created":{"date-parts":[[2018,5,2]],"date-time":"2018-05-02T19:40:26Z","timestamp":1525290026000},"page":"1-11","source":"Crossref","is-referenced-by-count":40,"title":["Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber\u2019s Hereditary Optic Neuropathy"],"prefix":"10.1155","volume":"2018","author":[{"given":"Sara E.","family":"Ratican","sequence":"first","affiliation":[{"name":"John van Geest Centre for Brain Repair, Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK"},{"name":"Geisel School of Medicine, Dartmouth College, Hanover, NH, 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