Elfakess Rofa, Peer Dan
Center for Nanoscience and Nanotechnology, George S Wise Faculty of Life Sciences, Department of Cell Research and Immunology, Laboratory of Nanomedicine, Tel Aviv University, Tel Aviv, Israel.
IDrugs. 2010 Sep;13(9):626-31.
RNAi-based approaches have contributed significantly to the improved understanding of gene expression and function in vitro. The ability to apply these strategies in vivo to validate the role of specific genes in normal or pathological conditions, and to induce gene silencing, has led to new possibilities for using RNAi as a novel therapeutic modality. However, the translation of RNAi from an effective genomic tool into clinical applications has been hindered by the challenge of delivering RNAi molecules to their target tissues by systemic administration, particularly to hematopoietic cells. This feature review describes the current systemic RNAi delivery platforms targeted to leukocytes, with a focus on the integrin-targeted stabilized nanoparticles strategy, which uses leukocyte integrins for the delivery of siRNAs exclusively to cells of the immune system.
基于RNA干扰的方法为体外更好地理解基因表达和功能做出了重大贡献。将这些策略应用于体内以验证特定基因在正常或病理条件下的作用并诱导基因沉默的能力，为将RNA干扰用作一种新型治疗方式带来了新的可能性。然而，将RNA干扰从一种有效的基因组工具转化为临床应用受到了通过全身给药将RNA干扰分子递送至其靶组织（尤其是造血细胞）这一挑战的阻碍。这篇专题综述描述了当前针对白细胞的全身RNA干扰递送平台，重点介绍了整合素靶向稳定纳米颗粒策略，该策略利用白细胞整合素将小干扰RNA专门递送至免疫系统细胞。
