Vornlocher Hans-Peter
Alnylam Europe AG, Fritz-Hornschuch-Strasse 9, 95326 Kulmbach, Germany.
Trends Mol Med. 2006 Jan;12(1):1-3. doi: 10.1016/j.molmed.2005.10.009. Epub 2005 Nov 14.
Over the past four years, chemically synthesized short interfering RNA (siRNA) has become the standard tool for specific silencing of gene expression in vitro. The most difficult task in transferring this technology to an in vivo setting is to develop appropriate delivery strategies. With this aim, Song et al. recently reported the development of antibody-protamine fusion proteins as vehicles for receptor-directed delivery of siRNA. When a mixture of siRNA targeting tumor-related genes was administered in this way, tumor growth was inhibited in an engineered melanoma model, demonstrating the therapeutic potential of this technology. However, several challenges remain to be overcome before targeted gene silencing can become a reality for patients.
在过去四年中，化学合成的短干扰RNA（siRNA）已成为体外特异性沉默基因表达的标准工具。将该技术应用于体内环境时，最困难的任务是开发合适的递送策略。出于这一目的，宋等人最近报道了抗体-鱼精蛋白融合蛋白作为受体导向递送siRNA载体的开发。当以这种方式给予靶向肿瘤相关基因的siRNA混合物时，在工程化黑色素瘤模型中肿瘤生长受到抑制，证明了该技术的治疗潜力。然而，在靶向基因沉默成为患者的现实之前，仍有几个挑战有待克服。
