Wright J Fraser, Zelenaia Olga
Clinical Vector Core, Center for Cellular and Molecular Therapeutics, The Childrens Hospital of Philadelphia and University of Pennsylvania School of Medicine, Philadelphia, PA, USA.
Methods Mol Biol. 2011;737:247-78. doi: 10.1007/978-1-61779-095-9_11.
Adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A major challenge for translation of promising research to clinical development is the establishment of appropriate quality control (QC) test methods to characterize clinical grade AAV vectors. This chapter focuses on QC testing, providing an overview of characterization methods appropriate for clinical vectors prepared for early phase clinical studies, and detailed descriptions for selected assays that are useful to assess AAV vector safety, potency, and purity.
表达治疗性基因产物的腺相关病毒（AAV）载体在人类基因治疗中显示出巨大潜力。将有前景的研究转化为临床开发面临的一个主要挑战是建立合适的质量控制（QC）测试方法来表征临床级AAV载体。本章重点介绍质量控制测试，概述适用于为早期临床研究准备的临床载体的表征方法，并详细描述用于评估AAV载体安全性、效力和纯度的选定检测方法。
