Three patients with RPE65-related retinopathy showed improved function in both eyes following gene augmentation therapy treatment with voretigene neparvovec. Immunosuppressants used in adeno ...
That effort is led by OCU400, a gene augmentation therapy for patients with inherited retinal diseases (IRDs) caused by mutations in the NR2E3 gene. OCU400 recently received orphan drug ...
Recent advances in the treatment of choroideremia include in vivo gene augmentation therapy. The disease is well suited for gene therapy approaches due to, amongst other factors, the accessibility ...
New York, United States, Jan. 20, 2025 (GLOBE NEWSWIRE) -- Gene therapy prevents the progression of an illness by altering the expression of mutated genes or the biological characteristics of ...
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