• Singap Med J · Jan 2023

    Review

    Therapeutic landscape for inherited ocular diseases: Current and emerging therapies.

    • Hwei Wuen Chan, Jaslyn Oh, and Bart Leroy.
    • Department of Ophthalmology, National University Hospital; Department of Ophthalmology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore.
    • Singap Med J. 2023 Jan 1; 64 (1): 172617-26.

    AbstractInherited ocular diseases comprise a heterogeneous group of rare and complex diseases, including inherited retinal diseases (IRDs) and inherited optic neuropathies. Recent success in adeno-associated virus-based gene therapy, voretigene neparvovec (Luxturna®) for RPE65-related IRDs, has heralded rapid evolution in gene therapy platform technologies and strategies, from gene augmentation to RNA editing, as well as gene agnostic approaches such as optogenetics. This review discusses the fundamentals underlying the mode of inheritance, natural history studies and clinical trial outcomes, as well as current and emerging therapies covering gene therapy strategies, cell-based therapies and bionic vision.

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