Tumor Genomics Unit, Department of Research; Fondazione IRCCS Istituto Nazionale dei Tumori, Via Venezian 1, 20133 Milan, Italy.
Pediatric Oncology Unit; Fondazione IRCCS Istituto Nazionale dei Tumori, Via Venezian 1, 20133 Milan, Italy.
Int J Mol Sci. 2019 Nov 19;20(22):5818. doi: 10.3390/ijms20225818.
Rhabdomyosarcoma (RMS), the most common soft tissue sarcoma of childhood and adolescence, is a rare but aggressive malignancy that originates from immature mesenchymal cells committed to skeletal muscle differentiation. Although RMS is, generally, responsive to the modern multimodal therapeutic approaches, the prognosis of RMS depends on multiple variables and for some patients the outcome remains dismal. Further comprehension of the molecular and cellular biology of RMS would lead to identification of novel therapeutic targets. MicroRNAs (miRNAs) are small non-coding RNAs proved to function as key regulators of skeletal muscle cell fate determination and to play important roles in RMS pathogenesis. The purpose of this review is to better delineate the role of miRNAs as a biomarkers or functional leaders in RMS development, so to possibly elucidate some of RMS molecular mechanisms and potentially therapeutically target them to improve clinical management of pediatric RMS.
横纹肌肉瘤(RMS)是儿童和青少年中最常见的软组织肉瘤,是一种罕见但具有侵袭性的恶性肿瘤,起源于向骨骼肌分化的未成熟间充质细胞。尽管 RMS 通常对现代多模式治疗方法有反应,但 RMS 的预后取决于多个变量,对于一些患者,其结果仍然不佳。进一步了解 RMS 的分子和细胞生物学将有助于确定新的治疗靶点。microRNAs(miRNAs)是小的非编码 RNA,已被证明在调节骨骼肌细胞命运决定方面发挥关键作用,并在 RMS 发病机制中发挥重要作用。本综述的目的是更好地阐明 miRNAs 作为 RMS 发展中的生物标志物或功能领导者的作用,以便可能阐明 RMS 的一些分子机制,并可能针对它们进行治疗,以改善儿科 RMS 的临床管理。