摘要
微小RNA(microRNA,miRNA)是一类含有约22个核苷酸的内源性非编码RNA,通过与靶mRNA的3′非翻译区(3′UTR)不完全性互补配对,介导转录后基因调控。人类疾病的发生和发展过程都有miRNA参与,其表达方式与疾病的诊断、分期、进展、预后及对治疗的反应程度密切相关。最近的研究表明miRNA可能成为大部分疾病的治疗靶点,随之产生基于miRNA的实验技术,结果表明这些技术不仅可以通过基因特异性方式干扰miRNA的作用,还可能为今后进一步探索其功能提供良好的研究工具。另外,新技术的应用还为以miRNA为靶点设计小分子药物应用于人类疾病的基因治疗迈出重要的一步。本篇综述首先总结miRNA与肿瘤、心律失常、心力衰竭和高血压等人类疾病发病的关系,并进而探讨其为靶点进行小分子药物设计的可行性。
MicroRNAs （miRNAs） are endogenous noncoding RNAs, about 22 nucleotides in length, that mediate post-transcriptional gene modulation by annealing to inexactly complementary sequences in the 3＇-untranslated regions of target mRNAs, miRNA alterations are involved in the initiation and progression of human diseases, miRNA-expression profiling of human diseases has identified signatures associated with diagnosis, staging, progression, prognosis and response to treatment. Recent evidence has suggested miRNAs as viable therapeutic targets for a wide range of human diseases. Several approaches were performed, the experimental examination of these techniques and the resultant findings not only indicate feasibility of interfering miRNA action in a gene-specific fashion but also may provide a new research tool for studying function of miRNAs. The new approaches also have the potential of becoming alternative gene therapy strategies.
出处
《药学学报》
CAS
CSCD
北大核心
2007年第11期1115-1121,共7页
Acta Pharmaceutica Sinica
基金
国家重点基础研究发展计划(973计划)资助项目(2007CB512000
2007CB512006)
国家自然科学基金重点资助项目(30430780)
国家自然科学基金资助项目(30672644).
