[Application of RNAi in gene-deficient models].
RNA interference (RNAi), a process of sequence-specific gene silence,can effectively and specifically suppress the activity of corresponding mRNAs in a gene-dependent manner induced by double-stranded RNA (dsRNA). This powerful technology has been widely employed to manipulate gene expression in mammalian and human cells, elucidate signal transduction pathways and identify gene functions in a whole-genome scale. Simultaneously, it also displays a bright and fascinating future in the research and development of RNAi-based drugs for various diseases such as viral infections, cancers, metabolic disorders and genetic diseases. In present review, we attempt to recapitulate the application of this breakthrough technology in establishing gene-deficient models and show the alluring foreground of RNAi-based gene therapy in these diseases.
Tan
Yu-Liang
YL
Institute of Biophysics, Chinese Academy of Sciences, Beijing 100101 , China.
Yin
Qin-Wei
QW
D000818:Animals; D005600:Fragile X Syndrome / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000235:genetics*; D016159:Tumor Suppressor Protein p53 / Q000235:genetics
D034741:RNA, Small Interfering; D016159:Tumor Suppressor Protein p53