Diao Yong, Wang Qizhao, Xiao Weidong, Xu Rui'an
Engineering Research Center of Molecular Medicine, Ministry of Education, Institute of Molecular Medicine, Huaqiao University, Quanzhou 362021, China.
Sheng Wu Gong Cheng Xue Bao. 2011 May;27(5):717-23.
Recombinant adeno-associated virus (rAAV)-based vectors that can stably express therapeutic genes in vivo without detectable side-effect have shown great promise for human gene therapy. A major challenge for translation of promising research to clinical development is how to establish clinically compatible purification methods in separating rAAV from potentially pathogenic impurities, especially rAAV vector-related impurities, a class of impurities corresponding to AAV particles that closely resemble bona fide vectors and are difficult to remove. In this review we summarize the assembly process of rAAV vector-related impurities and their characteristics differed with rAAV vectors, and evaluate several current technologies to prevent their formation or separate them from rAAV stocks.
基于重组腺相关病毒（rAAV）的载体能够在体内稳定表达治疗性基因且无明显副作用，这为人类基因治疗展现出了巨大前景。将有前景的研究转化为临床应用面临的一个主要挑战是，如何建立临床适用的纯化方法，以从潜在的致病杂质中分离出rAAV，尤其是rAAV载体相关杂质，这类杂质是一类与真正的载体极为相似且难以去除的AAV颗粒。在这篇综述中，我们总结了rAAV载体相关杂质的组装过程及其与rAAV载体不同的特性，并评估了当前几种防止其形成或从rAAV储备中分离它们的技术。
