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Optimizing the adeno-associated viral vector system: A brief summary

机译:优化腺相关病毒载体系统:简要概述

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摘要

rAAV vectors have emerged as one of the leading gene transfer systems in preclinical and clinical applications. Promising intrinsic features of this vector system are nonpathogenicity, high stability and low immunogenicity. Application of the vector system has been improved by simplifying the production procedure and by expanding the vector platform using alternative serotypes and vectors with artificial capsids (mosaic, hybrid or specifically engineered). The current limitationsof the vector system are addressed based on the increasing knowledge on the virus-host interaction by further optimizing the capsid as well as the vector genome.
机译:rAAV载体已成为临床前和临床应用中领先的基因转移系统之一。该载体系统的有前途的固有特征是非致病性,高稳定性和低免疫原性。通过简化生产程序并通过使用其他血清型和带有人工衣壳(马赛克,杂种或专门设计)的载体来扩展载体平台,可以改善载体系统的应用。通过对衣壳以及载体基因组的进一步优化,基于对病毒-宿主相互作用的日益了解,解决了载体系统的当前局限性。

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