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The safety and efficacy of gene therapy treatment for monogenic retinal and optic nerve diseases: A systematic review

Alexis Ceecee Britten‐Jones, Rui Jin, Sena A. Gocuk, Elise Cichello, Fleur O’Hare, Doron G. Hickey, Thomas L. Edwards, Lauren N. Ayton

2021Genetics in Medicine63 citationsDOI

Topics & Concepts

MedicineClinical trialChoroideremiaGenetic enhancementOptic nerveOphthalmologyAchromatopsiaAdverse effectBioinformaticsRetinalInternal medicineGeneGeneticsBiologyRetinal Development and DisordersOcular Disorders and TreatmentsVirus-based gene therapy research
The safety and efficacy of gene therapy treatment for monogenic retinal and optic nerve diseases: A systematic review | Litcius