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:	Refereed Article
Keywords:	gene therapy, eye, genome editing
Research Division:	Biomedical and Clinical Sciences
Research Group:	Medical biotechnology
Research Field:	Gene and molecular therapy
Objective Division:	Health
Objective Group:	Clinical health
Objective Field:	Clinical health not elsewhere classified
UTAS Author:	Chen, J (Ms Jane Chen)
UTAS Author:	Li, F (Dr Fan Li)
UTAS Author:	Hewitt, AW (Professor Alex Hewitt)
UTAS Author:	Liu, G-S (Associate Professor Guei-Sheung Liu)
ID Code:	128202
Year Published:	2018
Web of Science® Times Cited:	43
Deposited By:	Menzies Institute for Medical Research
Deposited On:	2018-09-07
Last Modified:	2021-01-12
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