RNA-targeting strategies as a platform for ocular gene therapy

IF 18.6 1区 医学 Q1 OPHTHALMOLOGY Progress in Retinal and Eye Research Pub Date : 2023-01-01 DOI:10.1016/j.preteyeres.2022.101110
Satheesh Kumar , Lewis E. Fry , Jiang-Hui Wang , Keith R. Martin , Alex W. Hewitt , Fred K. Chen , Guei-Sheung Liu
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引用次数: 6

Abstract

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 RNA-targeting therapeutics in the development of future drugs to treat eye disease.

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rna靶向策略作为眼部基因治疗的平台
随着许多以前无法治愈的疾病的新疗法的出现,基因医学给人们带来了希望。2017年,美国食品和药物管理局(FDA)批准Luxturna®用于治疗遗传性失明的一种莱伯先天性黑朦(LCA),这就是一个例证。Luxturna®也是第一个获得美国FDA批准的体内人类基因疗法。许多其他眼病的基因治疗临床试验正在进行中,新的给药系统、新药物靶点的发现和新兴技术正在推动这一领域的发展。特别是靶向RNA是一种有吸引力的遗传病治疗策略,由于避免了基因组的永久性变化,与其他方法相比,它可能具有安全优势。在这方面,反义寡核苷酸(ASO)和RNA干扰(RNAi)是目前开发RNA靶向治疗的流行策略。聚集规律穿插短回文重复序列(CRISPR)-CRISPR相关(Cas)系统的出现进一步激发了人们的热情,这些系统允许对核酸进行靶向操作。rna靶向CRISPR-Cas系统现在提供了一种开发rna靶向治疗的新方法,并且可能比现有技术提供更高的效率和特异性。此外,使用CRISPR-Cas和其他方式的RNA碱基编辑技术也可以精确地改变单个核苷酸。在这篇综述中,我们展示了rna靶向治疗眼病的进展,讨论了ASO和RNAi技术的应用,重点介绍了新兴的CRISPR-Cas系统,并考虑了rna靶向治疗在未来治疗眼病药物开发中的意义。
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来源期刊
CiteScore
34.10
自引率
5.10%
发文量
78
期刊介绍: Progress in Retinal and Eye Research is a Reviews-only journal. By invitation, leading experts write on basic and clinical aspects of the eye in a style appealing to molecular biologists, neuroscientists and physiologists, as well as to vision researchers and ophthalmologists. The journal covers all aspects of eye research, including topics pertaining to the retina and pigment epithelial layer, cornea, tears, lacrimal glands, aqueous humour, iris, ciliary body, trabeculum, lens, vitreous humour and diseases such as dry-eye, inflammation, keratoconus, corneal dystrophy, glaucoma and cataract.
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