Journal article

Gene therapy for inherited retinal diseases: progress and possibilities

Monica L Hu, Thomas L Edwards, Fleur O'Hare, Doron G Hickey, Jiang-Hui Wang, Zhengyang Liu, Lauren N Ayton

Clinical and Experimental Optometry | WILEY | Published : 2021


Inherited retinal diseases (IRDs) comprise a heterogeneous group of genetic disorders affecting the retina. Caused by mutations in over 300 genes, IRDs result in visual impairment due to dysfunction and degeneration of photoreceptors, retinal pigment epithelium, or the choroid. Important photoreceptor IRDs include retinitis pigmentosa and Leber congenital amaurosis. Macular dystrophies include Stargardt and Best disease. Currently, IRDs are largely incurable but the landscape of treatment options is rapidly changing for these diseases which, untreated, result in severe visual impairment and blindness.Advances in DNA delivery to the retina and improved genetic diagnosis of IRDs have led to a ..

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