Simonson Bridget, Das Saumya
Cardiovascular Division, Beth Israel Deaconess Medical Center, 330 Brookline Avenue, CLS 907, Boston, MA 02215, USA.
Mini Rev Med Chem. 2015;15(6):467-74. doi: 10.2174/1389557515666150324123208.
MicroRNAs are short noncoding 18-25 nucleotide long RNA which bind and inhibit mRNA. Currently, there are over 1000 known human microRNAs, and microRNAs control over 50% of mammalian protein coding genes. MicroRNAs can be overexpressed or repressed in different diseases and inhibition or replacement of microRNAs is a promising area of study for therapeutics. Here we review the current knowledge of microRNA therapy, and discuss ways in which they can be utilized. We also discuss different methods of delivery of miRNA, and current clinical trials of microRNA-based therapies for disease. Finally we discuss the current limitations in the field, and how these limitations are being overcome.
微小RNA是长度为18 - 25个核苷酸的短链非编码RNA，它们能结合并抑制信使核糖核酸。目前，已知的人类微小RNA有1000多种，且微小RNA控制着超过50%的哺乳动物蛋白质编码基因。微小RNA在不同疾病中可能会过度表达或受到抑制，对微小RNA的抑制或替代是一个很有前景的治疗研究领域。在此，我们综述了微小RNA治疗的现有知识，并讨论了其可被利用的方式。我们还讨论了微小RNA的不同递送方法，以及基于微小RNA的疾病治疗的当前临床试验。最后，我们讨论了该领域目前的局限性，以及这些局限性是如何被克服的。
