Connelly Colleen M, Deiters Alexander
Department of Chemistry, North Carolina State University, Raleigh, NC, USA.
Methods Mol Biol. 2014;1095:147-56. doi: 10.1007/978-1-62703-703-7_12.
Aberrant expression of microRNAs (miRNAs) has been linked to many human diseases including cancer, immune disorders, heart disease, and viral infections. Thus, small molecule inhibitors of miRNAs have potential as new therapeutic agents, as probes for the elucidation of detailed mechanisms of miRNA function, and as tools for the discovery of new targets for the treatment of human diseases. In order to identify small molecule inhibitors of specific miRNAs, functional assays have been developed and applied to the screening of small molecule libraries. Here, we report the application of a luciferase-based reporter assay of miRNA miR-122 function to the discovery of small molecule miR-122 inhibitors.
微小RNA（miRNA）的异常表达与许多人类疾病相关，包括癌症、免疫紊乱、心脏病和病毒感染。因此，miRNA的小分子抑制剂有潜力成为新型治疗药物、阐明miRNA功能详细机制的探针以及发现人类疾病治疗新靶点的工具。为了鉴定特定miRNA的小分子抑制剂，已开发出功能测定法并应用于小分子文库的筛选。在此，我们报告将基于荧光素酶的miRNA miR-122功能报告基因测定法应用于小分子miR-122抑制剂的发现。
