McCaffrey Anton P, Meuse Leonard, Pham Thu-Thao T, Conklin Douglas S, Hannon Gregory J, Kay Mark A
Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305-5208, USA.
Nature. 2002 Jul 4;418(6893):38-9. doi: 10.1038/418038a.
RNA interference is an evolutionarily conserved surveillance mechanism that responds to double-stranded RNA by sequence-specific silencing of homologous genes. Here we show that transgene expression can be suppressed in adult mice by synthetic small interfering RNAs and by small-hairpin RNAs transcribed in vivo from DNA templates. We also show the therapeutic potential of this technique by demonstrating effective targeting of a sequence from hepatitis C virus by RNA interference in vivo.
RNA干扰是一种进化上保守的监测机制,它通过对同源基因进行序列特异性沉默来响应双链RNA。在此我们表明,合成的小干扰RNA以及从DNA模板在体内转录的小发夹RNA能够在成年小鼠中抑制转基因表达。我们还通过在体内利用RNA干扰有效靶向丙型肝炎病毒的一个序列,展示了该技术的治疗潜力。