腺相关病毒
衣壳
基因传递
载体(分子生物学)
生物
计算生物学
基因组
病毒
病毒学
基因
遗传增强
重组DNA
遗传学
作者
László Hajba,András Guttman
出处
期刊:Current Molecular Medicine
[Bentham Science]
日期:2021-02-03
卷期号:20 (10): 806-813
被引量:14
标识
DOI:10.2174/1566524020999200730181042
摘要
Adeno-associated virus (AAV) is one of the most promising viral gene delivery vectors with long-term gene expression and disease correction, featuring high efficiency and excellent safety in human clinical trials. During the production of AAV vectors, there are several quality control (QC) parameters that should be rigorously monitored to comply with clinical safety and efficacy. This review gives a short summary of the most frequently used AVV production and purification methods, focusing on the analytical techniques applied to determine the full/empty capsid ratio and the integrity of the encapsidated therapeutic DNA of the products.
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