用于人类基因治疗的腺相关病毒载体的质量控制测试、特性描述和关键质量属性。

Quality Control Testing, Characterization and Critical Quality Attributes of Adeno-Associated Virus Vectors Used for Human Gene Therapy.

机构信息

Center for Definitive and Curative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, 94305, USA.

出版信息

Biotechnol J. 2021 Jan;16(1):e2000022. doi: 10.1002/biot.202000022. Epub 2020 Nov 27.

Abstract

For adeno-associated virus (AAV)-based human gene therapy, challenges for the translation of promising research results to successful clinical development include optimization of vector design and manufacturing processes to ensure that vectors prepared for administration to human subjects have attributes consistent with safe and durable expression. This article briefly reviews quality control methods for routine testing and supplemental characterization of AAV vectors for investigational product development. The relationship of vector and manufacturing process design with product critical quality attributes is discussed.

摘要

对于腺相关病毒(AAV)为基础的人类基因治疗,将有前途的研究成果转化为成功的临床开发的挑战包括优化载体设计和制造工艺,以确保准备用于人体的载体具有与安全和持久表达一致的属性。本文简要回顾了用于研究产品开发的 AAV 载体常规检测和补充鉴定的质量控制方法。讨论了载体和制造工艺设计与产品关键质量属性的关系。

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