Surgical Technique for Subretinal Gene Therapy in Humans with Inherited Retinal Degeneration

Abstract excerpt

The technique draws on personal experience with gene augmentation therapy by subretinal injection in patients with choroideremia, RPE65 mutation-associated retinal dystrophy, and X-linked retinitis pigmentosa. Only 2 surgeons initially performed RPE65 gene therapy in the Phase 3 clinical trial leading to drug approval in the United States and the European Union.1 As new surgeons begin to use the commercially...

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