Vorhies John S, Nemunaitis John J
Mary Crowley Medical Research Center, Dallas, TX, USA.
Methods Mol Biol. 2009;480:11-29. doi: 10.1007/978-1-59745-429-2_2.
DNA vector-based short hairpin RNA (shRNA) as a means of effecting RNA interference (RNAi) is a promising mechanism for the precise disruption of gene expression to achieve a therapeutic effect. The clinical usage of shRNA therapeutics in cancer is limited by obstacles related to effective delivery into the nuclei of target cancer cells. Significant pre-clinical data have been amassed about biodegradable and non-biodegradable polymeric delivery vehicles that are relevant for shRNA delivery into humans. Here, we will review some leading candidates for clinical usage with a focus on studies relating to their potential for usage in cancer shRNA therapeutics and discuss some of the advantages and disadvantages of using biodegradable and non-biodegradable delivery vehicles.
基于DNA载体的短发夹RNA（shRNA）作为实现RNA干扰（RNAi）的一种手段，是一种很有前景的机制，可精确破坏基因表达以达到治疗效果。shRNA疗法在癌症临床应用中受到与有效递送至靶癌细胞核相关的障碍的限制。关于可生物降解和不可生物降解的聚合物递送载体，已有大量相关的临床前数据积累，这些载体与将shRNA递送至人体有关。在此，我们将回顾一些临床应用的主要候选物，重点关注与其在癌症shRNA疗法中的应用潜力相关的研究，并讨论使用可生物降解和不可生物降解递送载体的一些优缺点。
