Grünweller A, Hartmann R K
Pharmazie. 2016 Jan;71(1):8-16.
RNA-based effector molecules (nucleic acid effectors) are important tools in molecular medicine because they offer a strategy to address therapeutically interesting targets that are not "druggable" with classic small molecule inhibitors. However, for in vivo applications, RNA-based effectors require specific chemical modifications to improve their stability and pharmacokinetic properties, as well as to minimize toxic and unspecific off-target effects.
基于RNA的效应分子(核酸效应物)是分子医学中的重要工具,因为它们提供了一种策略,可用于靶向那些无法被传统小分子抑制剂“靶向”的具有治疗意义的靶点。然而,对于体内应用而言,基于RNA的效应分子需要进行特定的化学修饰,以提高其稳定性和药代动力学性质,并将毒性和非特异性脱靶效应降至最低。