Shaw Aaron, Bischof Daniela, Jasti Aparna, Ernstberger Aaron, Hawkins Troy, Cornetta Kenneth
Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Hum Gene Ther Methods. 2012 Feb;23(1):65-71. doi: 10.1089/hgtb.2011.204.
In the production of lentiviral vector for clinical studies the purity of the final product is of vital importance. To remove plasmid and producer cell line DNA, investigators have incubated the vector product with Benzonase, a bacterially derived DNase. As an alternative we investigated the use of Pulmozyme, a U.S. Food and Drug Administration-approved human DNase for the treatment of cystic fibrosis, by comparing the efficiency of DNA removal from lentiviral vector preparations. A green fluorescent protein-expressing lentiviral vector was prepared by transient calcium phosphate transfection of HEK 293T cells and DNA removal was compared when treating vector after harvest or immediately after transfection. The effectiveness of DNase treatment was measured by quantitative PCR using primers for vesicular stomatitis virus glycoprotein G viral envelope plasmid. When treating the final product, 1-hr incubations (37°C) with Pulmozyme at 20 U/ml reduced plasmid DNA to undetectable levels. Longer incubations (up to 4 hr) did not improve DNA removal at lower concentrations and the effectiveness was equivalent to or better than Benzonase at 50 U/ml. Attempting to use Pulmozyme immediately after transfection, but before final medium change, as a means to decrease Pulmozyme concentration in the final product provided a 2-log reduction in DNA but was inferior to treatment at the end of production. Pulmozyme, at concentrations up to 100 U/ml, had no measurable effect on infectious titer of the final vector product. The use of Pulmozyme is likely to increase the cost of DNase treatment when preparing vector product and should be considered when generating clinical-grade vector products.
在用于临床研究的慢病毒载体生产中,最终产品的纯度至关重要。为去除质粒和生产细胞系DNA,研究人员用源自细菌的核酸酶Benzonase处理载体产品。作为替代方法,我们通过比较从慢病毒载体制剂中去除DNA的效率,研究了使用美国食品药品监督管理局批准用于治疗囊性纤维化的人核酸酶Pulmozyme。通过HEK 293T细胞的瞬时磷酸钙转染制备表达绿色荧光蛋白的慢病毒载体,并比较收获后或转染后立即处理载体时的DNA去除情况。使用水泡性口炎病毒糖蛋白G病毒包膜质粒的引物通过定量PCR测量核酸酶处理的有效性。处理最终产品时,在37°C下用20 U/ml的Pulmozyme孵育1小时可将质粒DNA降至检测不到的水平。在较低浓度下,更长时间的孵育(长达4小时)并不能改善DNA去除效果,其有效性与50 U/ml的Benzonase相当或更好。在转染后但在最终换液前立即使用Pulmozyme作为降低最终产品中Pulmozyme浓度的方法,可使DNA降低2个对数,但不如生产结束时处理有效。浓度高达100 U/ml的Pulmozyme对最终载体产品的感染滴度没有可测量的影响。制备载体产品时使用Pulmozyme可能会增加核酸酶处理的成本,在生产临床级载体产品时应予以考虑。