Arsenijevic Yvan, Berger Adeline, Udry Florian, Kostic Corinne
Unit Retinal Degeneration and Regeneration, Department of Ophthalmology, University of Lausanne, Jules-Gonin Eye Hospital, Fondation Asile des Aveugles, 1004 Lausanne, Switzerland.
Group Epigenetics of ocular diseases, Department of Ophthalmology, University of Lausanne, Jules-Gonin Eye Hospital, Fondation Asile des Aveugles, 1004 Lausanne, Switzerland.
Pharmaceutics. 2022 Jul 31;14(8):1605. doi: 10.3390/pharmaceutics14081605.
This review offers the basics of lentiviral vector technologies, their advantages and pitfalls, and an overview of their use in the field of ophthalmology. First, the description of the global challenges encountered to develop safe and efficient lentiviral recombinant vectors for clinical application is provided. The risks and the measures taken to minimize secondary effects as well as new strategies using these vectors are also discussed. This review then focuses on lentiviral vectors specifically designed for ocular therapy and goes over preclinical and clinical studies describing their safety and efficacy. A therapeutic approach using lentiviral vector-mediated gene therapy is currently being developed for many ocular diseases, e.g., aged-related macular degeneration, retinopathy of prematurity, inherited retinal dystrophies (Leber congenital amaurosis type 2, Stargardt disease, Usher syndrome), glaucoma, and corneal fibrosis or engraftment rejection. In summary, this review shows how lentiviral vectors offer an interesting alternative for gene therapy in all ocular compartments.
本综述介绍了慢病毒载体技术的基础知识、其优点和缺陷，并概述了其在眼科领域的应用。首先，阐述了在开发用于临床应用的安全高效慢病毒重组载体过程中遇到的全球挑战。还讨论了风险以及为尽量减少副作用所采取的措施，以及使用这些载体的新策略。然后，本综述重点关注专门为眼部治疗设计的慢病毒载体，并回顾了描述其安全性和有效性的临床前和临床研究。目前正在针对多种眼部疾病开发使用慢病毒载体介导的基因治疗的治疗方法，例如年龄相关性黄斑变性、早产儿视网膜病变、遗传性视网膜营养不良（2型莱伯先天性黑蒙、斯塔加特病、Usher综合征）、青光眼以及角膜纤维化或移植排斥反应。总之，本综述展示了慢病毒载体如何为所有眼内区域的基因治疗提供了一个有趣的选择。
