Gradalis, Inc., Dallas, Texas, USA.
Pharm Res. 2011 Dec;28(12):2983-95. doi: 10.1007/s11095-011-0604-5. Epub 2011 Oct 19.
Since its discovery in 1998, RNA interference (RNAi) has revolutionized basic and clinical research. Small RNAs, including small interfering RNA (siRNA), short hairpin RNA (shRNA) and microRNA (miRNA), mediate RNAi effects through either cleavage-dependent or cleavage-independent RNA inducible silencing complex (RISC) effector processes. As a result of its efficacy and potential, RNAi has been elevated to the status of "blockbuster therapeutic" alongside recombinant protein and monoclonal antibody. RNAi has already contributed to our understanding of neoplasia and has great promise for anti-cancer therapeutics, particularly so for personalized cancer therapy. Despite this potential, several hurdles have to be overcome for successful development of RNAi-based pharmaceuticals. This review will discuss the potential for, challenges to, and the current status of RNAi-based cancer therapeutics.
自 1998 年发现以来,RNA 干扰(RNAi)彻底改变了基础和临床研究。小 RNA,包括小干扰 RNA(siRNA)、短发夹 RNA(shRNA)和 microRNA(miRNA),通过依赖切割或不依赖切割的 RNA 诱导沉默复合物(RISC)效应物过程介导 RNAi 效应。由于其功效和潜力,RNAi 已被提升到与重组蛋白和单克隆抗体相当的“重磅治疗药物”地位。RNAi 已经有助于我们对肿瘤发生的理解,并为癌症治疗带来了巨大的希望,特别是对于个性化癌症治疗。尽管有这种潜力,但要成功开发基于 RNAi 的药物,还需要克服几个障碍。这篇综述将讨论基于 RNAi 的癌症治疗的潜力、挑战和现状。