The eye is at the forefront of the application of gene therapy techniques to medicine. In the United States, a gene therapy treatment\nfor Leber�s congenital amaurosis, a rare inherited retinal disease, recently became the first gene therapy to be approved by the FDA\nfor the treatment of disease caused by mutations in a specific gene. Phase III clinical trials of gene therapy for other single-gene\ndefect diseases of the retina and optic nerve are also currently underway. However, for optic nerve diseases not caused by\nsingle-gene defects, gene therapy strategies are likely to focus on slowing or preventing neuronal death through the expression\nof neuroprotective agents. In addition to these strategies, there has also been recent interest in the potential use of precise\ngenome editing techniques to treat ocular disease. This review focuses on recent developments in gene therapy techniques for\nthe treatment of glaucoma and Leber�s hereditary optic neuropathy (LHON). We discuss recent successes in clinical trials for\nthe treatment of LHON using gene supplementation therapy, promising neuroprotective strategies that have been employed in\nanimal models of glaucoma and the potential use of genome editing techniques in treating optic nerve disease.
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