Bandeira Vanessa, Peixoto Cristina, Rodrigues Ana F, Cruz Pedro E, Alves Paula M, Coroadinha Ana S, Carrondo Manuel J T
Instituto de Biologia Experimental e Tecnológica (IBET), 2781-901 Oeiras, Portugal.
Hum Gene Ther Methods. 2012 Aug;23(4):255-63. doi: 10.1089/hgtb.2012.059. Epub 2012 Aug 30.
Lentiviral vectors (LVs) hold great potential as gene delivery vehicles. However, the manufacturing and purification of these vectors still present major challenges, mainly because of the low stability of the virus, essentially due to the fragility of the membrane envelope. The main goal of this work was the establishment of a fast, scalable, and robust downstream protocol for LVs, combining microfiltration, anion-exchange, and ultrafiltration membrane technologies toward maximization of infectious LVs recovery. CIM(®) (Convective Interaction Media) monolithic columns with diethylaminoethanol (DEAE) anion exchangers were used for the purification of clarified LV supernatants, allowing infectious vector recoveries of 80%, which is 10% higher than the values currently reported in the literature. These recoveries, combined with the results obtained after optimization of the remaining downstream purification steps, resulted in overall infectious LV yields of 36%. Moreover, the inclusion of a Benzonase step allowed a removal of approximately 99% of DNA impurities. The entire downstream processing strategy herein described was conceived based on disposable and easily scalable technologies. Overall, CIM DEAE columns have shown to be a good alternative for the purification of LVs, since they allow faster processing of the viral bulks and enhanced preservation of virus biological activity, consequently, increasing infectious vector recoveries.
慢病毒载体(LVs)作为基因传递载体具有巨大潜力。然而,这些载体的生产和纯化仍然面临重大挑战,主要是因为病毒稳定性低,这主要归因于膜包膜的脆弱性。这项工作的主要目标是建立一种快速、可扩展且稳健的LVs下游处理方案,结合微滤、阴离子交换和超滤膜技术,以最大限度地提高有感染性的LVs回收率。使用带有二乙氨基乙醇(DEAE)阴离子交换剂的CIM(®)(对流相互作用介质)整体柱对澄清的LV上清液进行纯化,可实现80%的有感染性载体回收率,比目前文献报道的值高10%。这些回收率与优化其余下游纯化步骤后获得的结果相结合,使有感染性的LVs总产率达到36%。此外,加入核酸酶步骤可去除约99%的DNA杂质。本文所述的整个下游处理策略是基于一次性且易于扩展的技术构思的。总体而言,CIM DEAE柱已被证明是纯化LVs的良好选择,因为它们能够更快地处理病毒原液并增强病毒生物活性的保存,从而提高有感染性载体的回收率。