da Silva Nuno R, Jorge Paula, Martins José A, Teixeira José A, Marcos João C
Centre of Biological Engineering, University of Minho, Campus de Gualtar, 4710-057 Braga, Portugal.
Centre of Chemistry, University of Minho, Campus de Gualtar, 4710-057 Braga, Portugal.
Life (Basel). 2021 Oct 26;11(11):1138. doi: 10.3390/life11111138.
Gene therapy and DNA vaccination are among the most expected biotechnological and medical advances for the coming years. However, the lack of cost-effective large-scale production and purification of pharmaceutical-grade plasmid DNA (pDNA) still hampers their wide application. Downstream processing, which is mainly chromatography-based, of pDNA remains the key manufacturing step. Despite its high resolution, the scaling-up of chromatography is usually difficult and presents low capacity, resulting in low yields. Alternative methods that are based on aqueous two-phase systems (ATPSs) have been studied. Although higher yields may be obtained, its selectivity is often low. In this work, modified polymers based on poly(ethylene glycol) (PEG) derivatisation with amino groups (PEG-amine) or conjugation with positively charged amino acids (PEG-lysine, PEG-arginine, and PEG-histidine) were studied to increase the selectivity of PEG-dextran systems towards the partition of a model plasmid. A two-step strategy was employed to obtain suitable pure formulations of pDNA. In the first step, a PEG-dextran system with the addition of the affinity ligand was used with the recovery of the pDNA in the PEG-rich phase. Then, the pDNA was re-extracted to an ammonium-sulphate-rich phase in the second step. After removing the salt, this method yielded a purified preparation of pDNA without RNA and protein contamination.
基因治疗和DNA疫苗接种是未来几年最值得期待的生物技术和医学进展之一。然而,缺乏具有成本效益的大规模生产和纯化药用级质粒DNA(pDNA)仍然阻碍了它们的广泛应用。pDNA的下游加工主要基于色谱法,仍然是关键的制造步骤。尽管色谱法具有高分辨率,但扩大规模通常很困难且容量较低,导致产量不高。基于双水相系统(ATPS)的替代方法已得到研究。尽管可能获得更高的产量,但其选择性往往较低。在这项工作中,研究了基于聚乙二醇(PEG)用氨基衍生化(PEG-胺)或与带正电荷的氨基酸(PEG-赖氨酸、PEG-精氨酸和PEG-组氨酸)偶联的改性聚合物,以提高PEG-葡聚糖系统对模型质粒分配的选择性。采用两步策略来获得合适的pDNA纯制剂。第一步,使用添加了亲和配体的PEG-葡聚糖系统,将pDNA在富含PEG的相中回收。然后,在第二步中将pDNA重新提取到富含硫酸铵的相中。去除盐分后,该方法得到了不含RNA和蛋白质污染的pDNA纯化制剂。