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非灵长类慢病毒载体及其在眼部疾病基因治疗中的应用。

Non-Primate Lentiviral Vectors and Their Applications in Gene Therapy for Ocular Disorders.

机构信息

Department of Biological, Chemical and Pharmaceutical Sciences and Technologies (STEBICEF), University of Palermo, Viale delle Scienze Edificio 16, 90128 Palermo, Italy.

Advanced Technologies Network (ATeN) Center, University of Palermo, Viale delle Scienze Edificio 18, 90128 Palermo, Italy.

出版信息

Viruses. 2018 Jun 9;10(6):316. doi: 10.3390/v10060316.

Abstract

Lentiviruses have a number of molecular features in common, starting with the ability to integrate their genetic material into the genome of non-dividing infected cells. A peculiar property of non-primate lentiviruses consists in their incapability to infect and induce diseases in humans, thus providing the main rationale for deriving biologically safe lentiviral vectors for gene therapy applications. In this review, we first give an overview of non-primate lentiviruses, highlighting their common and distinctive molecular characteristics together with key concepts in the molecular biology of lentiviruses. We next examine the bioengineering strategies leading to the conversion of lentiviruses into recombinant lentiviral vectors, discussing their potential clinical applications in ophthalmological research. Finally, we highlight the invaluable role of animal organisms, including the emerging zebrafish model, in ocular gene therapy based on non-primate lentiviral vectors and in ophthalmology research and vision science in general.

摘要

慢病毒具有许多共同的分子特征,首先是能够将其遗传物质整合到非分裂感染细胞的基因组中。非灵长类慢病毒的一个特殊性质是它们不能感染和引起人类疾病,因此为基因治疗应用衍生出生物安全的慢病毒载体提供了主要的基本原理。在这篇综述中,我们首先概述了非灵长类慢病毒,强调了它们共同的和独特的分子特征,以及慢病毒分子生物学中的关键概念。接下来,我们研究了导致慢病毒转化为重组慢病毒载体的生物工程策略,讨论了它们在眼科研究中的潜在临床应用。最后,我们强调了动物生物体,包括新兴的斑马鱼模型,在基于非灵长类慢病毒载体的眼科基因治疗以及一般的眼科学研究和视觉科学中的宝贵作用。

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