Aberrant expression of certain genes and microRNAs (miRNAs) has been shown to drive cancer development and progression, thus the modification of aberrant gene and miRNA expression presents an opportunity for therapeutic targeting. Ectopic modulation of a single dysregulated miRNA has the potential to revert therapeutically unfavorable gene expression in cancer cells by targeting multiple genes simultaneously. Although the use of noncoding RNA-based cancer therapy is a promising approach, the lack of a feasible delivery platform for small noncoding RNAs has hindered the development of this therapeutic modality. Recently, however, there has been an evolution in RNA nanotechnology, in which small noncoding RNA is loaded onto nanoparticles derived from the pRNA-3WJ viral RNA motif of the bacteriophage phi29. Preclinical studies have shown the capacity of this technology to specifically target tumor cells by conjugating these nanoparticles with ligands specific for cancer cells and resulting in the endocytic delivery of siRNA and miRNA inhibitors directly into the cell. Here we provide a systematic review of the various strategies, which have been utilized for miRNA delivery with a specific focus on the preclinical evaluation of promising RNA nanoparticles for glioblastoma (GBM) targeted therapy.

某些基因和 microRNA (miRNA) 的异常表达已被证明会驱动癌症的发展和进展，因此异常基因和 miRNA 表达的修饰为治疗靶向提供了机会。单个失调的 miRNA 的异位调节有可能通过同时靶向多个基因来恢复癌细胞中治疗上不利的基因表达。尽管使用基于非编码 RNA 的癌症治疗是一种很有前景的方法，但缺乏可行的小非编码 RNA 递送平台阻碍了这种治疗方式的发展。然而，最近 RNA 纳米技术发生了发展，其中小的非编码 RNA 被加载到源自噬菌体 phi29 的 pRNA-3WJ 病毒 RNA 基序的纳米颗粒上。临床前研究表明，该技术具有特异性靶向肿瘤细胞的能力，方法是将这些纳米颗粒与癌细胞特异性配体结合，并导致 siRNA 和 miRNA 抑制剂直接内吞递送到细胞中。在这里，我们对已用于 miRNA 递送的各种策略进行系统评价，特别关注用于胶质母细胞瘤 (GBM) 靶向治疗的有前景的 RNA 纳米颗粒的临床前评估。