中文摘要：

核酶是具有催化功能的结构性RNA分子,且大多数核酶具有剪切RNAs的功能,可以利用它们剪切信使RNA（mRNAs）调节基因表达,从而作为基因治疗的新手段.阐述核酶的发现历程,以及其在艾滋病、肝炎、肿瘤和生殖道系统感染等基因治疗的研究进展.最后,分析核酶在基因治疗中的技术优势及存在问题,并对核酶领域包括更多核酶晶体结构的确立、酶切机理的理解、核酶新的生物学功能的发现等研究进行展望.

英文摘要：

Ribozymes are structured RNA molecules with catalytic activities.Since most ribozymes have the ability to cleave RNAs,they could be used to cleave mRNAs and regulate gene expression,and therefore becoming new tools for gene therapy.This paper describes the discovery history of ribozymes and their applications in the therapy researches of acquired immune deficiency syndrome,hepatitis,tumor,reproductive tract infection and other diseases.Lastly,the paper analyzes the technological advantages and the existing problems of the ribozyme′s application in gene therapy,and looks into the future of researches on the establishment of more crystal structures of new ribozymes,understanding of ribozyme cleavage mechanisms,and the discovery of new biological functions of ribozymes.