Rev Bras Oftalmol.2025;84(Supl. 3):ex0002
Gene therapy for retinal diseases
DOI: 10.37039/1982.8551.2025S3ex0002
ABSTRACT
This update article aims to discuss gene therapies under study for retinal diseases, mainly among the different inherited retinal dystrophies. Inherited retinal dystrophies are considered rare diseases, some are ultra-rare. The use of Luxturna, which was approved by the FDA in 2017 and by Anvisa in 2020, changed the natural history of the retinopathy associated with biallelic variants in RPE65 gene and was a major milestone in ophthalmology. There are still many challenges for this new therapeutic modality, which encompasses different strategies being studied in different areas of medicine.
Keywords: Choroid diseases; Gene therapy; Rare diseases; Retinal dystrophies