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eRAM

encyclopedia of Rare Disease Annotation for Precision Medicine



  Leber's congenital amaurosis and the role of gene therapy in congenital retinal disorders

Leber's congenital amaurosis,this disease affects retinal photoreceptor cells.Patients with naturally see only big and bright objects, these patients were diagnosed as babies, their low vision to the blind, no difference. In 2017 Mar 18, Walid Sharif and Zuhair Sharif publish an article in << International Journal of Ophthalmology >> which title is “Leber's congenital amaurosis and the role of gene therapy in congenital retinal disorders”, reviewed the document of congenital retinal disease. A phase III trial is presently ongoing and if successful, it will lead the way to additional gene therapy attempts to cure monogenic, inherited retinopathies.

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