Abstract:
Simple Summary The discovery of microRNAs has revolutionized the world of science and opened up new opportunities in cancer treatment. miRNA dysregulation plays a crucial role in carcinogenesis processes, such as invasion, metastasis, and angiogenesis, in a broad range of cancers. Although the use of miRNA therapy in cancer treatment is promising, its effective and safe application remains one of the most important challenges hindering its clinical use. Novel nanoparticles continue to be developed and used to enable tumor-targeted miRNA delivery. The aim of the present review is to provide insights into the strategies for miRNA-based therapeutics in cancer, focusing on recent in vivo and clinical studies that have used nanoparticles for miRNA delivery. 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.