rAAV规模化包装系统的研究进展

doi:10.13560/j.cnki.biotech.bull.1985.2015.12.009

摘要/Abstract

摘要： 重组腺相关病毒(rAAV)作为唯一一种通过欧盟FDA认证的基因治疗载体，具有宿主范围广、转染效率高、非致病性、免疫原性低、能够长期表达外源基因的优点。随着以rAAV基因治疗临床试验的深入与扩大，传统rAAV包装系统产能不足的缺点逐渐凸显出来，急需可放大、易规模化的rAAV包装系统来解决现有的供需矛盾。鉴于此，在介绍传统rAAV包装系统的基础之上，着重阐述了杆状病毒昆虫细胞系法、酵母法以及痘苗病毒-腺病毒法，尤其对后者寄予厚望。旨在介绍几种较好的rAAV规模化包装方案，探讨其规模化制备的发展趋势。

关键词: 重组腺相关病毒, 基因治疗, 载体, 包装系统

Abstract: As the only gene therapy vector certificated by EU FDA, recombinant adeno-associated virus(rAAV)has advantages of wide host range, high transfection efficiency, non-pathogenic, low immunogenicity and long-term expression of transgenosis. With the expansion of applying rAAV for gene therapy clinical trials, the shortage of capacity of packing system in traditional rAAV has emerged, thus the rAAV packaging system that can be easily enlarged and scaled up to solve the existing problem of supply and demand is in urgent need. Therefore, on the basis of describing traditional rAAV packaging system, the focus of this review is to emphatically expound the baculovirus insect cell lines, yeast, and vaccinia virus-adenovirus method, especially with more expectation to the last one. The aim of this article is to introduce several preferable systems for scale packaging of rAAV, and discuss the trend of development.

Key words: rAAV, gene therapy, vector, packaging system

占申标,唐明青,甘娜,曹苑青,李招发. rAAV规模化包装系统的研究进展[J]. 生物技术通报, 2015, 31(12): 63-69.

Zhan Shenbiao, Tang Mingqing, Gan Na, Cao Yuanqing, Li Zhaofa. Research Progress Regarding to Large-scale Packaging System of rAAV[J]. Biotechnology Bulletin, 2015, 31(12): 63-69.

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