Strayer David S, Agrawal Lokesh, Cordelier Pierre, Liu Bianling, Louboutin Jean-Pierre, Marusich Elena, McKee Hayley J, Ren Carmen N NiGongyi, Strayer Marlene S
Department of Pathology and Cell Biology, Jefferson Medical College, Philadelphia, PA, USA.
Mol Biotechnol. 2006 Oct;34(2):257-70. doi: 10.1385/MB:34:2:257.
Among the goals of gene therapy is long-term expression of delivered transgenes. Recombinant Tagdeleted SV40 vectors (rSV40s) are especially well suited for this purpose. rSV40s deliver transgene expression that endures for extended periods of time in tissue culture and in vivo, in both dividing and nondividing cells. These vectors are particularly effective in transducing some cell types that have been almost unapproachable using other gene delivery systems, such as quiescent hematopoietic progenitor cells and their differentiated derivatives. Other cellular targets include neurons, brain microglia, hepatocytes, dendritic cells, vascular endothelium, and others. Because rSV40s do not elicit neutralizing antibodies they are useful for in vivo gene delivery in settings where more than one administration may be desirable. The key characteristics of these vectors include their high production titers and therefore suitability for large cell pools, effectiveness in delivering intracellular proteins, and untranslated RNAs, maintenance of transgene expression at constant levels for extended times, suitability for constitutive or conditional promoters and for combinatorial gene delivery and ability to integrate into genomes of both dividing and nondividing cells.
基因治疗的目标之一是实现导入的转基因的长期表达。重组缺失Tag的SV40载体(rSV40)特别适合这一目的。rSV40能在组织培养和体内的分裂细胞与非分裂细胞中实现长时间的转基因表达。这些载体在转导某些使用其他基因递送系统几乎无法实现的细胞类型时特别有效,例如静止的造血祖细胞及其分化衍生物。其他细胞靶点包括神经元、脑小胶质细胞、肝细胞、树突状细胞、血管内皮细胞等。由于rSV40不会引发中和抗体,因此在可能需要多次给药的体内基因递送环境中很有用。这些载体的关键特性包括高生产滴度,因此适用于大量细胞群体;在递送细胞内蛋白质和非翻译RNA方面有效;能长时间维持转基因表达水平恒定;适用于组成型或条件型启动子以及组合基因递送;并且能够整合到分裂细胞和非分裂细胞的基因组中。