【摘要】 可稳定表达治疗基因而无明显不良反应的重组腺相关病毒(rAAV)载体被认为是最有发展前景的基因治疗载体。但如何建立可以有效去除rAAV载体中具潜在致病危害的空病毒颗粒,使产品质量符合临床使用要求的纯化工艺是研究人员面临的巨大挑战。以下总结了空病毒颗粒形成的过程及有别于rAAV载体的特点,并对可以将其与rAAV载体有效分离的技术手段进行了评价。更多还原

【Abstract】 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 empty virus particle.In this review we summarize the assembly process of empty virus particle and their characteristics differed with rAAV vectors,and evaluate several current technologies to separate them from rAAV stocks.更多还原