Department of Pharmacology and Neuroscience, North Texas Eye Research Institute, University of North Texas Health Science Center, Fort Worth, TX, USA.
FEBS J. 2023 Nov;290(22):5248-5269. doi: 10.1111/febs.16771. Epub 2023 Mar 27.
Ocular diseases are a highly heterogeneous group of phenotypes, caused by a spectrum of genetic variants and environmental factors that exhibit diverse clinical symptoms. As a result of its anatomical location, structure and immune privilege, the eye is an ideal system to assess and validate novel genetic therapies. Advances in genome editing have revolutionized the field of biomedical science, enabling researchers to understand the biology behind disease mechanisms and allow the treatment of several health conditions, including ocular pathologies. The advent of clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing facilitates efficient and specific genetic modifications in the nucleic acid sequence, resulting in permanent changes at the genomic level. This approach has advantages over other treatment strategies and is promising for the treatment of various genetic and non-genetic ocular conditions. This review provides an overview of the CRISPR/CRISPR-associated protein 9 (Cas9) system and summarizes recent advances in the therapeutic application of CRISPR/Cas9 for the treatment of various ocular pathologies, as well as future challenges.
眼部疾病是一组高度异质的表型，由一系列遗传变异和环境因素引起，表现出不同的临床症状。由于其解剖位置、结构和免疫特权，眼睛是评估和验证新型基因治疗的理想系统。基因组编辑技术的进步彻底改变了生物医学科学领域，使研究人员能够了解疾病机制背后的生物学，并允许治疗多种健康状况，包括眼部疾病。基于成簇规律间隔短回文重复序列（CRISPR）的基因编辑的出现促进了核酸序列中高效和特异性的遗传修饰，导致基因组水平上的永久性变化。这种方法优于其他治疗策略，有望治疗各种遗传和非遗传眼部疾病。本文综述了 CRISPR/Cas9 系统，并总结了 CRISPR/Cas9 用于治疗各种眼部疾病的治疗应用的最新进展以及未来的挑战。
