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Gene therapy for visual loss: Opportunities and concerns

Lee, JH, Wang, J-H, Chen, J, Li, F, Edwards, TL, Hewitt, AW ORCID: 0000-0002-5123-5999 and Liu, G ORCID: 0000-0003-3379-724X 2018 , 'Gene therapy for visual loss: Opportunities and concerns' , Progress in Retinal and Eye Research , pp. 1-23 , doi: 10.1016/j.preteyeres.2018.08.003.

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Abstract

Many 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.

Item Type: Article
Authors/Creators:Lee, JH and Wang, J-H and Chen, J and Li, F and Edwards, TL and Hewitt, AW and Liu, G
Keywords: Gene therapy, eye
Journal or Publication Title: Progress in Retinal and Eye Research
Publisher: Pergamon-Elsevier Science Ltd
ISSN: 1350-9462
DOI / ID Number: 10.1016/j.preteyeres.2018.08.003
Copyright Information:

Copyright 2018 Elsevier Ltd.

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