申请人:Deiters Alexander
公开号:US20130005759A1
公开(公告)日:2013-01-03
MicroRNAs are a class of endogenous regulators of gene function. Aberrant regulation of microRNAs has been linked to various human diseases, most importantly cancer. Small molecule intervention of microRNA misregulation has the potential to provide new therapeutic approaches to such diseases. microRNA miR-122 is the most abundant microRNA in the liver and is involved in hepatocellular carcinoma development and hepatitis C virus (HCV) infection. Small molecule inhibitors and activators of the microRNA miR-122 are described, and methods for their identification are reported. These small molecule inhibitors reduce viral replication in liver cells and thus represent a new approach to the treatment of HCV infections. Moreover, small molecule activation of miR-122 in liver cancer cells selectively induced apoptosis through caspase activation, and thus has implications in cancer chemotherapy.
MicroRNAs是一类内源性基因调控因子。微小RNA的异常调控已与各种人类疾病,尤其是癌症相关联。小分子干预微小RNA的错调可能为这些疾病提供新的治疗方法。微小RNA miR-122是肝脏中最丰富的微小RNA,参与肝细胞癌的发展和丙型肝炎病毒(HCV)感染。描述了微小RNA miR-122的小分子抑制剂和激活剂,并报道了其鉴定方法。这些小分子抑制剂能够减少肝细胞中的病毒复制,因此代表了治疗HCV感染的新方法。此外,小分子激活miR-122在肝癌细胞中选择性地通过caspase激活诱导凋亡,因此在癌症化疗中具有重要意义。