Mishra Prasun J, Merlino Glenn
Laboratory of Cancer Biology and Genetics, National Cancer Institute, NIH, Bethesda, Maryland 20892-4264, USA.
J Clin Invest. 2009 Aug;119(8):2119-23. doi: 10.1172/JCI40107. Epub 2009 Jul 20.
Since their discovery in the early 2000s, microRNAs (miRNAs) and their penchant for RNA interference have taken the scientific community by storm, working their way into virtually every corner of biological inquiry. The very nature of their action, the ability to simultaneously extinguish the expression of a multitude of genes and negate their functions, immediately suggested therapeutic promise. In this issue of the JCI, a step toward the realization of this promise is described. Taulli et al. demonstrate that the miRNAs miR-1/miR-206, which are routinely lost in advanced, poorly differentiated rhabdomyosarcoma (RMS) but characteristically expressed in the mature skeletal muscle from which these tumors arise, restore the myogenic differentiation program and block the tumorigenic phenotype (see the related article beginning on page 2366). Their data support the notion that these small RNAs, effectively functioning as "micro-sheriffs" by restoring myogenic law and order, hold substantial clinical potential as differentiation therapy for RMS and perhaps other solid tumors. miRNA reexpression therapy constitutes a novel approach to handcuff oncogenes and arrest tumor development.
自21世纪初被发现以来，微小RNA（miRNA）及其对RNA干扰的偏好席卷了科学界，几乎渗透到生物学研究的每一个角落。它们的作用本质，即能够同时抑制众多基因的表达并消除其功能，立刻显示出了治疗前景。在本期《临床研究杂志》中，描述了朝着实现这一前景迈出的一步。陶利等人证明，miR-1/miR-206这两种miRNA在晚期低分化横纹肌肉瘤（RMS）中通常缺失，但在这些肿瘤起源的成熟骨骼肌中特异性表达，它们能恢复肌源性分化程序并阻断致瘤表型（见第2366页开始的相关文章）。他们的数据支持了这样一种观点，即这些小RNA通过恢复肌源性的正常秩序，有效地充当“微型治安官”，作为RMS以及可能其他实体瘤的分化疗法具有巨大的临床潜力。miRNA重新表达疗法是一种抑制癌基因并阻止肿瘤发展的新方法。
