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Báo cáo y học: " Selective gene silencing by viral delivery of short hairpin RNA"

Tuyển tập báo cáo các nghiên cứu khoa học quốc tế ngành y học dành cho các bạn tham khảo đề tài: Selective gene silencing by viral delivery of short hairpin RNA | Sliva and Schnierle Virology Journal 2010 7 248 http www.virologyj.eom content 7 1 248 VIROLOGY JOURNAL REVIEW Open Access Selective gene silencing by viral delivery of short hairpin RNA Katja Sliva Barbara S Schnierle Abstract RNA interference RNAi technology has not only become a powerful tool for functional genomics but also allows rapid drug target discovery and in vitro validation of these targets in cell culture. Furthermore RNAi represents a promising novel therapeutic option for treating human diseases in particular cancer. Selective gene silencing by RNAi can be achieved essentially by two nucleic acid based methods i cytoplasmic delivery of short doublestranded ds interfering RNA oligonucleotides siRNA where the gene silencing effect is only transient in nature and possibly not suitable for all applications or ii nuclear delivery of gene expression cassettes that express short hairpin RNA shRNA which are processed like endogenous interfering RNA and lead to stable gene downregulation. Both processes involve the use of nucleic acid based drugs which are highly charged and do not cross cell membranes by free diffusion. Therefore in vivo delivery of RNAi therapeutics must use technology that enables the RNAi therapeutic to traverse biological membrane barriers in vivo. Viruses and the vectors derived from them carry out precisely this task and have become a major delivery system for shRNA. Here we summarize and compare different currently used viral delivery systems give examples of in vivo applications and indicate trends for new developments such as replicating viruses for shRNA delivery to cancer cells. Introduction The human genome project not only unraveled the human genetic code but spin-off technical improvements also inspired genome sequencing of a multitude of other organisms. However since sequence data alone are not sufficient to identify gene function gene knockout or knock-in strategies have to replenish the results in order to analyze the .