Lentiviral vectors in gene therapy: their current status and future potential.
Lentiviral vectors in gene therapy: their current status and future potential.
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DOI：
10.1007/s00005-010-0063-4
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发表时间：
2010-04
影响因子：
3.2
通讯作者：
Breckpot, Karine
中科院分区：
文献类型：
--
作者：
Escors, David;Breckpot, Karine
基因治疗的概念起源于世纪中期，被认为是一种革命性的技术，有望治愈几乎任何疾病的分子基础是了解。从那时起，几种基因载体被开发出来，并且在许多人类疾病的动物模型中显示了基因治疗的可行性。然而，直到新世纪初，在一项治疗儿童致命免疫缺陷疾病的小规模临床试验中，临床疗效才得以证实。这是在逆转录病毒治疗后取得的第一次成功，但后来在相当数量的接受治疗的儿童中发生了与载体有关的白血病，这表明基因治疗的未来成功取决于我们对载体生物学的理解。这导致了具有改进的效率、特异性和安全性的新一代载体的开发。其中包括基于HIV-1慢病毒的载体（lentivectors），它们越来越多地用于基础和应用研究。人类基因治疗的临床试验目前正在使用慢病毒载体在广泛的人类疾病。这篇综述的目的是描述导致HIV-1慢病毒载体工程的主要科学步骤，并将其置于当前人类基因治疗的背景下。
The concept of gene therapy originated in the mid 20th century and was perceived as a revolutionary technology with the promise to cure almost any disease of which the molecular basis was understood. Since then, several gene vectors have been developed, and the feasibility of gene therapy shown in many animal models of human disease. However, clinical efficacy could not be demonstrated until the beginning of the new century in a small-scale clinical trial curing an otherwise fatal immunodeficiency disorder in children. This first success – achieved after retroviral therapy - was later on overshadowed by the occurrence of vector-related leukaemia in a significant number of the treated children, demonstrating that the future success of gene therapy depends on our understanding of vector biology. This has led to the development of later generation vectors with improved efficiency, specificity and safety. Amongst these are HIV-1 lentivirus-based vectors (lentivectors), which are being increasingly used in basic and applied research. Human gene therapy clinical trials are currently under way using lentivectors in a wide range of human diseases. The intention of this review is to describe the main scientific steps leading to the engineering of HIV-1 lentiviral vectors, and place them in the context of current human gene therapy.
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