Kara Goknur, Arun Banu, Calin George A, Ozpolat Bulent
Department of Nanomedicine, Houston Methodist Research Institute, Houston, TX 77030, USA.
Department of Chemistry, Biochemistry Division, Ordu University, Ordu 52200, Turkey.
Cancers (Basel). 2022 Aug 6;14(15):3818. doi: 10.3390/cancers14153818.
MicroRNAs (miRNAs) are non-protein-coding RNA molecules 20-25 nucleotides in length that can suppress the expression of genes involved in numerous physiological processes in cells. Accumulating evidence has shown that dysregulation of miRNA expression is related to the pathogenesis of various human diseases and cancers. Thus, stragegies involving either restoring the expression of tumor suppressor miRNAs or inhibiting overexpressed oncogenic miRNAs hold potential for targeted cancer therapies. However, delivery of miRNAs to tumor tissues is a challenging task. Recent advances in nanotechnology have enabled successful tumor-targeted delivery of miRNA therapeutics through newly designed nanoparticle-based carrier systems. As a result, miRNA therapeutics have entered human clinical trials with promising results, and they are expected to accelerate the transition of miRNAs from the bench to the bedside in the next decade. Here, we present recent perspectives and the newest developments, describing several engineered natural and synthetic novel miRNA nanocarrier formulations and their key in vivo applications and clinical trials.
微小RNA(miRNA)是长度为20 - 25个核苷酸的非蛋白质编码RNA分子,可抑制参与细胞众多生理过程的基因表达。越来越多的证据表明,miRNA表达失调与各种人类疾病和癌症的发病机制有关。因此,恢复肿瘤抑制性miRNA的表达或抑制过度表达的致癌性miRNA的策略具有靶向癌症治疗的潜力。然而,将miRNA递送至肿瘤组织是一项具有挑战性的任务。纳米技术的最新进展使得通过新设计的基于纳米颗粒的载体系统成功实现miRNA治疗剂的肿瘤靶向递送成为可能。结果,miRNA治疗剂已进入人体临床试验并取得了有前景的结果,预计它们将在未来十年加速miRNA从实验室到临床应用的转变。在此,我们展示了近期的观点和最新进展,描述了几种工程化的天然和合成新型miRNA纳米载体配方及其关键的体内应用和临床试验。