rnai治疗的大时代（The great age of RNAi treatment）.docVIP

rnai治疗的大时代（The great age of RNAi treatment） RNA interference (RNA, interference, RNAi) is a mechanism of gene silencing after transcription. This mechanism is present in nature. 16 years ago, scientists began to pay attention to the potential of this mechanism for treatment, especially for diseases that are not readily available by conventional drugs, such as cancer. But for more than 10 years, the RNAi approach to clinical practice has been a roller coaster ride. Andrew, Fire, and Craig Mello first revealed the RNAi phenomenon in 1998, and the two won the Nobel prize in physiology and medicine in 2006 with this important discovery. Since then, RNAi with its simple operation method and good experimental effect is fast becoming a major technology for basic research, researchers around the world are beginning to use this method for silencing specific genes in cell and animal models. At the same time, more and more scientists are becoming interested in RNAi therapy. The higher, and does not require special binding protein, can target directly on the monoclonal antibody can not reach directly, prevent some disease causing gene translation in protein source block disease, thereby avoiding the use of small molecule drugs or other biological agents in the molecular cascade downstream treatment of disease. Despite the advantages of RNAi treatment, there are still some scholars who are cautious about RNAi treatment. Pharmaceutical enterprises have begun to burn money in this field to seize the commanding heights of technology. The stage of rational prosperity. A number of large pharmaceutical companies in the technical barriers does not take into account the circumstances have invested billions of dollars, will start some R D to RNAi treatment, especially those genetic targets is conclusive, but no drug disease. However, the results of early clinical trials were very disappointing, and RNAi treatment did not achieve the desired results. Most notably, in 2009, RNAi PKO, a Mi