Adeno-associated virus: fit to serve.

Adeno-associated virus: fit to serve.
复制标题

DOI:
10.1016/j.coviro.2014.07.008
复制
发表时间:
2014-10
影响因子:
5.9
通讯作者:
Vandenberghe, Luk H.
Vandenberghe, Luk H.
中科院分区:
医学2区
文献类型:
--
作者:
Zinn, Eric;Vandenberghe, Luk H.

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腺相关病毒(Adeno-associated virus,AAV)是一种辅助病毒依赖的细小病毒,与人类疾病无关。这一方面,结合其广泛的细胞和组织嗜性,以及有限的病毒宿主应答,使其成为一种有吸引力的基因治疗载体系统。病毒蛋白衣壳是与宿主的主要界面,是这些表型的主要决定因素,是高度可变的,并且在复制过程中最容易受到压力。在这里,我们探讨了AAV和其他细小病毒在这些表型方面的进化路径,以及定向进化和工程策略,这些策略利用了从自然选择中吸取的教训,以解决AAV作为治疗性基因转移平台的剩余限制。
Adeno-associated virus (AAV) is a helper-dependent parvovirus which has not been linked with human disease. This aspect, in combination with its broad cell and tissue tropism, and limited viral host response has made it an attractive vector system for gene therapy. The viral protein capsid, the primary interface with the host, is the main determinant for these phenotypes, is highly variable, and is most subject to pressures during replication. Here, we explore the evolutionary path of AAV and other parvoviruses in respect to these phenotypes, as well as directed evolution and engineering strategies that have exploited the lessons learned from natural selection in order to address remaining limitations of AAV as a therapeutic gene transfer platform.
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