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Gene therapy for visual loss: opportunities and concerns
journal contribution
posted on 2023-05-19, 21:04 authored by Lee, JH, Wang, J-H, Chen, J, Li, F, Edwards, TL, Alexander HewittAlexander Hewitt, Guei-Sheung LiuGuei-Sheung LiuMany clinical trials using gene therapy have shown significant therapeutic benefits and exceptional safety records. Increasing evidence is verifying the long sought-after promise that gene therapy will genetically 'cure' some severely disabling diseases. In particular, the first gene therapy bioproduct for RPE65-associated Leber's congenital amaurosis, which was approved by the US Food and Drug Administration in 2017, has provided tremendous encouragement to the field of gene therapy. Recent developments in genome editing technologies have significantly advanced our capability to precisely engineer genomes in eukaryotic cells. Programmable nucleases, particularly the CRISPR/Cas system, have been widely adopted in studies applying genome engineering therapy to ocular diseases with the hope of managing these diseases. In this review article, we summarize the current approaches that have been developed in the area of gene therapy for ocular disease. We also discuss the challenges and opportunities facing gene therapy for ocular diseases, as well as its prospects.
History
Publication title
Progress in Retinal and Eye ResearchVolume
68Pagination
31-53ISSN
1350-9462Department/School
Menzies Institute for Medical ResearchPublisher
Pergamon-Elsevier Science LtdPlace of publication
The Boulevard, Langford Lane, Kidlington, Oxford, England, Ox5 1GbRights statement
Copyright 2018 Elsevier Ltd.Repository Status
- Restricted