全世界有数百万人患有遗传性疾病、外伤、传染病或眼癌，其中许多眼病会导致不可逆转的失明，这是一项重大的公共卫生负担。眼睛是一个相对较小且具有免疫特权的器官。使用基于核酸的药物来操纵功能失常的基因，针对眼部疾病的根源，被认为是一种很有前景的治疗方法。然而，由于某些不利的特性，在体内利用核酸疗法仍然存在一些挑战，例如不稳定性、生物载体依赖性细胞摄取、体内药代动力学特征（RNA）较短以及靶向和脱靶副作用（脱氧核糖核酸）。作为基因载体的脂质纳米粒子（LNP）的发展是革命性的进步，为核酸治疗的临床应用做出了贡献。 LNP 具有捕获和运输各种遗传物质的能力，例如小干扰 RNA、mRNA、DNA 和基因编辑复合物。这为通过抑制致病基因、表达治疗性蛋白质或纠正遗传缺陷来解决眼部疾病开辟了途径。在这里，我们深入研究用于眼部基因治疗的尖端 LNP 技术，包括配方设计、临床前开发和临床转化。© 2024 作者。

Millions of people worldwide have hereditary genetic disorders, trauma, infectious diseases, or cancer of the eyes, and many of these eye diseases lead to irreversible blindness, which is a major public health burden. The eye is a relatively small and immune-privileged organ. The use of nucleic acid-based drugs to manipulate malfunctioning genes that target the root of ocular diseases is regarded as a therapeutic approach with great promise. However, there are still some challenges for utilizing nucleic acid therapeutics in vivo because of certain unfavorable characteristics, such as instability, biological carrier-dependent cellular uptake, short pharmacokinetic profiles in vivo (RNA), and on-target and off-target side effects (DNA). The development of lipid nanoparticles (LNPs) as gene vehicles is revolutionary progress that has contributed the clinical application of nucleic acid therapeutics. LNPs have the capability to entrap and transport various genetic materials such as small interfering RNA, mRNA, DNA, and gene editing complexes. This opens up avenues for addressing ocular diseases through the suppression of pathogenic genes, the expression of therapeutic proteins, or the correction of genetic defects. Here, we delve into the cutting-edge LNP technology for ocular gene therapy, encompassing formulation designs, preclinical development, and clinical translation.© 2024 The Author(s).