Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber's Hereditary Optic Neuropathy.

Neural Plasticity
Sara E RaticanKeith R Martin

Abstract

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's 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's 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 n...Continue Reading

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Citations

Sep 3, 2019·Current Eye Research·Tasneem Z Khatib, Keith R Martin
Sep 6, 2019·International Journal of Molecular Sciences·Marie ClaesLieve Moons
Jul 29, 2020·Human Gene Therapy·Leonardo Fernandes Moutinho RochaFabio Batista Mota
Jan 9, 2019·Frontiers in Neuroscience·Zhimin XuRuth B Caldwell
Aug 11, 2020·Journal of Ophthalmology·Barbara Cvenkel, Miriam Kolko
Jan 28, 2021·Genes·Michael James GilhooleyPatrick Yu Wai Man
Dec 31, 2020·Pharmaceutics·Ognjenka RahićEdina Vranić
Apr 29, 2021·Neural Regeneration Research·Mohamed TawfikBernhard A Sabel

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Methods Mentioned

BETA
gene knockout

Clinical Trials Mentioned

NCT02862938

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