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Downloaded from http://journals.lww.com/apjoo by BhDMf5ePHKbH4TTImqenVA5KvPVPZ0P5BEgU+IUTEfzO/GUWifn2IfwcEVVH9SSn on 06/03/2020 REVIEW ARTICLE The Pathway From Genes to Gene Therapy in Glaucoma: A Review of Possibilities for Using Genes as Glaucoma Drugs Teresa Borrás, PhD negligible distribution to organs other than the targeted eye. Abstract: Treatment of diseases with gene therapy is advancing rapidly. Human clinical trials on congenital retinal diseases such as The use of gene therapy has expanded from the original concept of re- Leber congenital amaurosis have broken the barrier to the placing the mutated gene causing the disease to the use of genes to con- reality of a direct gene medical application and have opened trol nonphysiological levels of expression or to modify pathways known the door to the use of gene therapy in all other tissues of the to affect the disease. Genes offer numerous advantages over conventional eye. Because of the particular physiology of the eye’s aqueous drugs. They have longer duration of action and are more specific. Genes humor, agents delivered to the anterior chamber and even to can be delivered to the target site by naked DNA, cells, nonviral, and the vitreous make their way to the trabecular meshwork (TM), viral vectors. The enormous progress
The Asia-Pacific Journal of Ophthalmology – Wolters Kluwer Health
Published: Jan 1, 2017
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