Research Department, Oxford BioMedica Ltd., Windrush Court, Transport Way, Oxford OX4 6LT, UK.
Nat Commun. 2017 Mar 27;8:14834. doi: 10.1038/ncomms14834.
A key challenge in the field of therapeutic viral vector/vaccine manufacturing is maximizing production. For most vector platforms, the 'benchmark' vector titres are achieved with inert reporter genes. However, expression of therapeutic transgenes can often adversely affect vector titres due to biological effects on cell metabolism and/or on the vector virion itself. Here, we exemplify the novel 'Transgene Repression In vector Production' (TRiP) system for the production of both RNA- and DNA-based viral vectors. The TRiP system utilizes a translational block of one or more transgenes by employing the bacterial tryptophan RNA-binding attenuation protein (TRAP), which binds its target RNA sequence close to the transgene initiation codon. We report enhancement of titres of lentiviral vectors expressing Cyclo-oxygenase-2 by 600-fold, and adenoviral vectors expressing the pro-apoptotic gene Bax by >150,000-fold. The TRiP system is transgene-independent and will be a particularly useful platform in the clinical development of viral vectors expressing problematic transgenes.
治疗性病毒载体/疫苗生产领域的一个关键挑战是最大限度地提高产量。对于大多数载体平台而言,使用惰性报告基因可以达到“基准”载体滴度。然而,由于对细胞代谢和/或载体病毒粒子本身的生物学影响,治疗性转基因的表达常常会降低载体滴度。在这里,我们举例说明了用于生产基于 RNA 和 DNA 的病毒载体的新型“转基因在载体生产中抑制”(TRiP)系统。TRiP 系统利用一个或多个转基因的翻译阻断来实现,方法是使用细菌色氨酸 RNA 结合衰减蛋白(TRAP),该蛋白结合其靶 RNA 序列靠近转基因起始密码子。我们报告说,表达环氧化酶-2 的慢病毒载体的滴度提高了 600 倍,表达促凋亡基因 Bax 的腺病毒载体的滴度提高了 150000 倍以上。TRiP 系统是转基因独立的,在表达有问题的转基因的病毒载体的临床开发中将是一个特别有用的平台。