Leber congenital amaurosis is an IRD caused by a defect in the CEP20 gene, resulting in progressive vision loss in infants. According to ClinicalTrials.gov, two new agents that target the CEP20 gene are currently in clinical trials:
The BRILLIANCE phase 1/2 clinical trial is investigating gene-editing techniques using CRISPR-Cas9 to delete the mutation in the CEP290 gene that is responsible for destroying light-sensing cells in the retina.
Other novel agents for Leber congenital amaurosis are in clinical trials.
Learn more about inherited retinal diseases.
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Cite this: Donny W. Suh, Raj K. Maturi. Fast Five Quiz: Management of Inherited Retinal Diseases - Medscape - Mar 31, 2023.