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Utility of AAV vectors derived from novel serotypes

机译：源自新型血清型的AAV载体的效用

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摘要

AAV vector is derived from nonpathogenic virus and has a number of attractive features as a vector for human gene transfer including safety, broad tissue specificity, and low immunogenicity following gene transfer. Moreover, persistent transgene expression (for years) was demonstrated in multiple animal experiments. For these reasons, applications to a wide spectrum of diseases are expected, and several clinical trials have been conducted. Although it is too early to conclude the outcome, the efficacy of treatment was not sufficiently substantiated in most of the trials despite confirming the safety of the vector. These results are primarily due to low levels of transgene expression. One of the approaches to improve this situation is the use of alternative serotypes of AAV. Traditionally, serotype 2 was considered to be a prototype of AAV, and the majority of studies including human clinical trials have been conducted using this serotype. On the other hand, there are five "classical" serotypes, and several have been additionally discovered from tissues of primates including humans. These serotypes are considered to be valuable resources for vector development to overcome the shortcomings of serotype 2. This review focuses on the difference in expression levels and tissue specificity of various serotype-derived vectors and summarizes current status in the treatment of candidate diseases.
机译：AAV载体源自非致病性病毒，作为人类基因转移的载体具有许多吸引人的特征，包括安全性，广泛的组织特异性和基因转移后的低免疫原性。此外，在多个动物实验中证明了持续的转基因表达（多年）。由于这些原因，期望将其应用于多种疾病，并且已经进行了一些临床试验。尽管结论尚为时过早，但尽管证实了载体的安全性，但在大多数试验中仍未充分证实治疗的有效性。这些结果主要是由于低水平的转基因表达。改善这种情况的方法之一是使用AAV的其他血清型。传统上，血清型2被认为是AAV的原型，大多数研究包括人类临床试验都是使用这种血清型进行的。另一方面，存在五种“经典”血清型，并且还从包括人在内的灵长类动物组织中发现了几种。这些血清型被认为是用于克服血清型2缺点的载体开发的宝贵资源。本综述着重于各种血清型衍生载体在表达水平和组织特异性方面的差异，并总结了候选疾病的治疗现状。

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