Vorhies John S, Nemunaitis John
Mary Crowley Medical Research Center, 1717 Main St, Suite 6000, Dallas, TX 75201, USA.
Expert Rev Anticancer Ther. 2007 Mar;7(3):373-82. doi: 10.1586/14737140.7.3.373.
The use of DNA vector-based short hairpin (sh)RNA for RNA interference shows promise as a precise means for the disruption of gene expression to achieve a therapeutic effect. The in vivo usage of shRNA therapeutics in cancer is limited by obstacles related to effective delivery into the nuclei of target cancer cells. Nonviral delivery vehicles that are relevant for shRNA delivery into humans belong to a group of substances about which significant preclinical data has been amassed to show an acceptable safety profile, resistance to immune defenses and good transfection efficiency. Here, we review the most promising current nonviral gene delivery vehicles with a focus on their potential use in cancer shRNA therapeutics.
使用基于DNA载体的短发夹（sh）RNA进行RNA干扰，有望成为一种精确破坏基因表达以实现治疗效果的手段。shRNA疗法在癌症中的体内应用受到与有效递送至靶癌细胞核相关的障碍的限制。与将shRNA递送至人体相关的非病毒递送载体属于一类物质，关于这类物质已积累了大量临床前数据，以显示其可接受的安全性、对免疫防御的抗性和良好的转染效率。在此，我们综述了当前最有前景的非病毒基因递送载体，重点关注它们在癌症shRNA治疗中的潜在用途。
