核酶与反基因疗法：催化性RNA如何用于抑制基因功能。

Ribozymes and the anti-gene therapy: how a catalytic RNA can be used to inhibit gene function.

作者信息

Muotri A R, da Veiga Pereira L, dos Reis Vasques L, Menck C F

机构信息

Departamento de Microbiologia, Instituto de Ciências Biomédicas, Universidade de São Paulo, São Paulo, Brazil.

出版信息

Gene. 1999 Sep 17;237(2):303-10. doi: 10.1016/s0378-1119(99)00334-0.

DOI:10.1016/s0378-1119(99)00334-0

PMID:10521654

Abstract

Ribozymes are RNA molecules that possess the dual properties of RNA sequence-specific recognition and site-specific cleavage of other RNA molecules. These properties provide powerful tools for studies requiring gene inhibition, when the DNA sequence is known. The use of these molecules goes beyond basic research, with a potential impact in therapeutical practice in medicine in the near future. In this review, we briefly describe the progress towards developing this class of molecules and its applications for the control of gene expression.

摘要

核酶是一类RNA分子，具有对其他RNA分子进行序列特异性识别和位点特异性切割的双重特性。当DNA序列已知时，这些特性为需要基因抑制的研究提供了强大的工具。这类分子的应用不仅限于基础研究，在不久的将来还可能对医学治疗实践产生影响。在这篇综述中，我们简要描述了这类分子的开发进展及其在基因表达控制方面的应用。