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Utility of AAV vectors derived from novel serotypes

机译：AAV载体的效用来自新型血清型

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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载体来自非遗传病毒，并且具有许多有吸引力的特征，作为人类基因转移的载体，包括在基因转移后的安全性，宽组织特异性和低免疫原性。此外，在多种动物实验中证明了持续转基因表达（多年）。由于这些原因，预期了在广谱疾病的应用，并且已经进行了几种临床试验。尽管结束结果为时过早，尽管确认载体的安全性，但在大多数试验中，治疗的疗效并未充分证实。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的替代血清型。传统上，血清型2被认为是AAV的原型，并且已经使用该血清型进行了大多数包括人类临床试验的研究。另一方面，存在五个“古典”血清型，并且还从包括人类的灵长类动物组织中另外发现了几种。这些血清型被认为是向量发展的有价值的资源，以克服血清型2的缺点。本综述侧重于各种血清型衍生载体的表达水平和组织特异性的差异，并总结了治疗候选疾病的当前状态。

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