Inherited retinal diseases (IRDs) are a group of rare disorders caused by genetic defects that lead to irreversible decline in vision and sometimes blindness.
Almost 300 genes known to cause IRDs have been mapped or identified, allowing for potential use of gene therapy, which did not exist a decade ago.
Dr Dominik Fischer, of the Eberhard Karls University of Tübingen, discusses groundbreaking research that has led to the first gene therapy, voretigene neparvovec, to be approved by the US Food and Drug Administration and the European Medicines Agency.
Fischer also reports on clinical trials that are further advancing the treatment of IRDs. The first investigates a therapy for X-linked retinitis pigmentosa, and the second examines gene therapy to treat X-linked choroideremia.
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Cite this: Diagnosis and Management of Inherited Retinal Dystrophy - Medscape - Jun 03, 2021.
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