Synthetic virology: engineering viruses for gene delivery.

Synthetic virology: engineering viruses for gene delivery.
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合成病毒学:基因输送的工程病毒。

DOI:
10.1002/wnan.1287
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发表时间:
2014-11
影响因子:
8.6
通讯作者:
Suh, Junghae
Suh, Junghae
中科院分区:
医学2区
文献类型:
--
作者:
Guenther, Caitlin M.;Kuypers, Brianna E.;Lam, Michael T.;Robinson, Tawana M.;Zhao, Julia;Suh, Junghae

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基因治疗的成功在很大程度上依赖于能够有效地将转基因输送到所需细胞群体的载体的性能。随着病毒的进化,将遗传物质输送到细胞中,在过去的几十年里,出现了一个多产的研究领域,以利用病毒的固有属性以及为它们设计新的特征。具体地说,合成病毒学领域的目标是利用基础病毒学研究积累的知识来设计功能增强的基因递送载体。增强型病毒载体或“仿生”病毒以天然的(病毒固有的或来自其他生物体的)和人工合成的(如人造聚合物或无机纳米颗粒)的工程成分或“部分”为特征。各种设计策略--有理、组合和伪有理--被用来创造混合病毒。未来的基因传递载体可能会纵横交错地跨越自然区域和合成区域之间的边界,以利用可以嫁接到病毒衣壳上的各种功能部分所提供的独特优势。这些研究工作将进一步扩大和加强对这些用于生物医学的纳米设备的功能能力的控制。
The success of gene therapy relies heavily on the performance of vectors that can effectively deliver transgenes to desired cell populations. As viruses have evolved to deliver genetic material into cells, a prolific area of research has emerged over the last several decades to leverage the innate properties of viruses as well as to engineer new features into them. Specifically, the field of synthetic virology aims to capitalize on knowledge accrued from fundamental virology research in order to design functionally enhanced gene delivery vectors. The enhanced viral vectors, or “bionic” viruses, feature engineered components, or “parts”, that are natural (intrinsic to viruses or from other organisms) and synthetic (such as man-made polymers or inorganic nanoparticles). Various design strategies – rational, combinatorial, and pseudo-rational – have been pursued to create the hybrid viruses. The gene delivery vectors of the future will likely criss-cross the boundaries between natural and synthetic domains to harness the unique strengths afforded by the various functional parts that can be grafted onto virus capsids. Such research endeavours will further expand and enable enhanced control over the functional capacity of these nanoscale devices for biomedicine.
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