New chemical approaches in modifying short interfering RNAs

本研究计划探讨利用天然RNA干扰（RNAi）途径降低特定基因表达水平的新方法。由于控制基因表达水平对于正常的生物学功能是必不可少的，因此沉默或控制基因表达的能力是非常可取的。RNAi途径是一种依靠短双链RNA靶向基因特异性信使RNA的自然生物学过程。利用这一自然途径的潜力将对包括加拿大在内的全世界的研究和医学产生巨大影响。然而，阻碍天然双链rna作为沉默剂成功的主要缺陷是：(1)寿命短；B)毒性和脱靶效应；C)细胞膜通透性差。

This research program investigates new approaches for lowering the expression level of a particular gene using the natural RNA interference (RNAi) pathway. Since controlled gene expression levels are essential for proper biological function, the ability to silence or control the expression of genes is highly desirable. The RNAi pathway is a natural biological process that relies on short double-stranded RNAs to target gene specific messenger RNA. The potential of harnessing this natural pathway would have enormous impact on research and medicine worldwide, including Canada. However, the main pitfalls prohibiting the success of natural double-stranded RNAs as silencing agents are a) short-lifetime; b) toxicity and off-target effects; c) and poor cell-membrane permeability.