《生命科学》 2010, 22(3): 216-223

RNA在生物学的重要地位及其作为常规治疗药物的研究进展

Sidney Altman

(美国耶鲁大学分子细胞和发育生物学系)

摘　要：

摘　要：20多年来的研究发现，RNA除了具有如tRNA、rRNA参与蛋白质生物合成的基本功能外，细胞内还存在许多种类的RNA，它们执行着不同的功能，在细胞内生物化学反应及机体发育调控过程中发挥着重要作用。正因为RNA功能多样性，在体内、体外开展的众多实验表明，RNA或其修饰形式可以抑制基因的表达。该文将探讨RNA在常规基因治疗中的研究。
关键词：核糖核酸；功能多样性；核糖核酸酶P；基因治疗

The central role of RNA in biology and as a general therapeutic agent

Sidney Altman

(Department of Molecular， Cellular and Developmental Biology， Yale University， USA)

Abstract:

Abstract: The diverse roles of different RNA molecules discovered over the past 20 years show the importance in intracellular biochemistry and in the regulation of developmental events in vivo. These novel functions of RNA are in addition to the basic roles of tRNA and rRNA in protein synthesis. RNA， or modified versions thereof， can be shown to inhibit the expression of genes in vitro or in vivo. A new function of RNA in general gene therapy will be discussed.
Key words: RNA; diverse functions; RNase P; gene therapy