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《China Biotechnology》 2015-01
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The Application and Perspectives of Multi-target RNAi in the Research and Development of Gene Therapy

XUE Yu-wen;LI Tie-jun;ZHOU Jia-ming;CHEN Li;Department of Pathological Anatomy,Nantong University;Small RNA Technology and Application Institute,Nantong University;  
The expression of target genes can be inhibited by RNA interference( RNAi) specificity via posttranscriptional gene silencing( PTGS) effects. RNAi silencing mechanism with efficiency, specificity and stability allows it to become an important tool in biomedical field. The focus is on the characteristics of RNAi technology and recent development in RNAi-based therapeutics,in particular structural modifications and designs for multi-target siRNAs were reviewed. It is illustrated that multi-target based siRNA therapeutics opens up a new therapeutic strategy and may help to improve the effectiveness of treatment. However,there are still some problems and challenges needing further researches for its widely use in clinical treatments.
【Fund】: 江苏省产学研前瞻性联合研究项目(BY2013042-06);; 江苏省教育厅项目(14KJB310016);; 江苏省优势学科(PAPD)资助项目
【CateGory Index】: R450
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【Citations】
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