[Potential role of microRNAs in human diseases and the exploration on design of small molecule agents].

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.