siRNA在治疗学中的应用

The Application of siRNA in Therapeutics

小干扰RNA(small interfering RNA,siRNA)是外源性双链RNA(double strand RNA,dsRNA)的加工产物,在细胞内能介导RNA干扰(RNA interference,RNA i)效应,识别特异性mRNA,沉默同源基因表达。其特异性和高效性显示出很高的实用价值,siRNA已成为许多疾病潜在的治疗手段。对于siRNA的应用,尽管还需要在减少非特异反应,发掘高效递药载体,应对新的基因变异等方面进行深入研究,但其可望在抗病毒、神经系统疾病和肿瘤治疗等许多领域发挥治疗作用。

Small interfering RNA （siRNA）is the processing product of exogenous double strand RNA （dsRNA）. siRNA mediate the RNA interference （RNAi）, induce the degradation of endogenous mRNA with homology showing high specificity and thus generate excited potential of therapeutic application. Although the obstacles including reduce non-specific effect, establish high-efficient delivery system and facing the new mutation are needed to harnessed, recent preclinical studies suggest that siRNA hold great promise for the treatment of various diseases.