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

ABSTRACT: Inherited 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.

SUBMITTER: Chan HW

PROVIDER: S-EPMC9979805 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature

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

Chan Hwei Wuen HW Oh Jaslyn J Leroy Bart B

Singapore medical journal 20230101 1

Inherited 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<sup>®</sup>) 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 ...[more]

PMID: 36722513

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

Publications

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

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