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Genetic medicine is offering hope as new therapies are emerging for many previously untreatable diseases. The eye is at the forefront of these advances, as exemplified by the approval of Luxturna® by the United States Food and Drug Administration (US FDA) in 2017 for the treatment of one form of Leber Congenital Amaurosis (LCA), an inherited blindness. Luxturna® was also the first in vivo human gene therapy to gain US FDA approval. Numerous gene therapy clinical trials are ongoing for other eye diseases, and novel delivery systems, discovery of new drug targets and emerging technologies are currently driving the field forward. Targeting RNA, in particular, is an attractive therapeutic strategy for genetic disease that may have safety advantages over alternative approaches by avoiding permanent changes in the genome. In this regard, antisense oligonucleotides (ASO) and RNA interference (RNAi) are the currently popular strategies for developing RNA-targeted therapeutics. Enthusiasm has been further fuelled by the emergence of clustered regularly interspersed short palindromic repeats (CRISPR)-CRISPR associated (Cas) systems that allow targeted manipulation of nucleic acids. RNA-targeting CRISPR-Cas systems now provide a novel way to develop RNA-targeted therapeutics and may provide superior efficiency and specificity to existing technologies. In addition, RNA base editing technologies using CRISPR-Cas and other modalities also enable precise alteration of single nucleotides. In this review, we showcase advances made by RNA-targeting systems for ocular disease, discuss applications of ASO and RNAi technologies, highlight emerging CRISPR-Cas systems and consider the implications of 1000 RNA-targeting therapeutics in the development of future drugs to treat eye disease.

Item Type:	Refereed Article
Keywords:	Antisense oligonucleotides; CRISPR-Cas13; Ocular gene therapy; RNA editing; RNA interference.
Research Division:	Biological Sciences
Research Group:	Genetics
Research Field:	Genetic immunology
Objective Division:	Health
Objective Group:	Clinical health
Objective Field:	Prevention of human diseases and conditions
UTAS Author:	Kumar, Satheesh (Mr Satheesh Kumar)
UTAS Author:	Hewitt, AW (Professor Alex Hewitt)
UTAS Author:	Liu, GL (Associate Professor Guei-Sheung Liu)
ID Code:	155595
Year Published:	2023
Deposited By:	Menzies Institute for Medical Research
Deposited On:	2023-03-01
Last Modified:	2023-03-01
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