Weiler J, Hunziker J, Hall J
Novartis Institutes for BioMedical Research, Genome and Proteome Sciences, Basel, Switzerland.
Gene Ther. 2006 Mar;13(6):496-502. doi: 10.1038/sj.gt.3302654.
MicroRNAs (miRNAs) are endogenous 19-25 nucleotide RNAs that have recently emerged as a novel class of important gene-regulatory molecules involved in many critical developmental and cellular functions. miRNAs have been implicated in the pathogenesis of several human diseases, such as neurodegenerative disorders, cancer, and more recently in viral and metabolic diseases. Unraveling the roles of miRNAs in cellular processes linked to human diseases will lead to novel opportunities for the regulation of protein function and will help to evaluate their potential for therapeutic intervention. Approaches to interfere with miRNA function in vitro and in vivo based on synthetic anti-miRNA oligonucleotides (AMOs) are discussed in this review.
微小RNA(miRNA)是一类内源性的19 - 25个核苷酸的RNA,最近作为一类新型的重要基因调控分子出现,参与许多关键的发育和细胞功能。miRNA与多种人类疾病的发病机制有关,如神经退行性疾病、癌症,以及最近发现的病毒和代谢疾病。阐明miRNA在与人类疾病相关的细胞过程中的作用,将为蛋白质功能调控带来新的机遇,并有助于评估它们在治疗干预方面的潜力。本综述讨论了基于合成抗miRNA寡核苷酸(AMO)在体外和体内干扰miRNA功能的方法。