Nature Technology Corporation, 4701 Innovation Drive, Lincoln, NE 68521, USA.
Mol Biotechnol. 2011 Jan;47(1):43-9. doi: 10.1007/s12033-010-9310-5.
Some DNA vaccine and gene therapy vector-encoded transgenes are toxic to the E. coli plasmid production host resulting in poor production yields. For plasmid products undergoing clinical evaluation, sequence modification to eliminate toxicity is undesirable because an altered vector is a new chemical entity. We hypothesized that: (1) insert-encoded toxicity is mediated by unintended expression of a toxic insert-encoded protein from spurious bacterial promoters; and (2) that toxicity could be eliminated with antisense RNA-mediated translation inhibition. We developed the pINT PR PL vector, a chromosomally integrable RNA expression vector, and utilized it to express insert-complementary (anti-insert) RNA from a single defined site in the bacterial chromosome. Anti-insert RNA eliminated leaky fluorescent protein expression from a target plasmid. A toxic retroviral gag pol helper plasmid produced in a gag pol anti-insert strain had fourfold improved plasmid fermentation yields. Plasmid fermentation yields were also fourfold improved when a DNA vaccine plasmid containing a toxic Influenza serotype H1 hemagglutinin transgene was grown in an H1 sense strand anti-insert production strain, suggesting that in this case toxicity was mediated by an antisense alternative reading frame-encoded peptide. This anti-insert chromosomal RNA expression technology is a general approach to improve production yields with plasmid-based vectors that encode toxic transgenes, or toxic alternative frame peptides.
有些 DNA 疫苗和基因治疗载体编码的转基因对大肠杆菌质粒生产宿主有毒,导致产量低下。对于正在进行临床评估的质粒产品,由于改变载体是一种新的化学实体,因此消除毒性的序列修饰是不可取的。我们假设:(1)插入编码的毒性是由细菌启动子意外表达有毒的插入编码蛋白介导的;(2)可以通过反义 RNA 介导的翻译抑制来消除毒性。我们开发了 pINT PR PL 载体,这是一种可整合到染色体上的 RNA 表达载体,并利用它从细菌染色体上的单一定义位置表达与插入物互补的(反插入物)RNA。反插入 RNA 消除了目标质粒中漏出的荧光蛋白表达。在 gag pol 反插入株中产生的毒性逆转录病毒 gag pol 辅助质粒,其质粒发酵产量提高了四倍。当含有毒性流感血清型 H1 血凝素转基因的 DNA 疫苗质粒在 H1 有义链反插入生产株中生长时,质粒发酵产量也提高了四倍,这表明在这种情况下,毒性是由反义可读框编码的肽介导的。这种反插入染色体 RNA 表达技术是一种提高基于质粒载体生产产量的通用方法,这些载体编码有毒转基因或有毒的框架肽。