The therapeutic potential of ribozymes

被引：105

作者：

James, HA ^{[1
]}

Gibson, I ^{[1
]}

机构：

[1] Univ E Anglia, Sch Biol Sci, Norwich NR4 7TJ, Norfolk, England

来源：

BLOOD | 1998年 / 91卷 / 02期

关键词：

D O I：

10.1182/blood.V91.2.371.371_371_382

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Ribozymes are catalytic RNA molecules that recognize their target RNA in a highly sequence-specific manner. They can therefore be used to inhibit deleterious gene expression (by cleavage of the target mRNA) or even repair mutant cellular RNAs. Targets such as the mRNAs of oncogenes (resulting from base mutations or chromosome translocations, eg, res or bcr-abl) and viral genomes and transcripts (human immunodeficiency virus-type 1 [HIV-1]) are ideal targets for such sequence-specific agents. The aim of this review is therefore to introduce the different classes of ribozymes, highlighting some of the chemistry of the reactions they catalyze, to address the specific inhibition of genes by ribozymes, the problems yet to be resolved, and how new developments in the field give hope to the future for ribozymes in the therapeutic field. (C) 1998 by The American Society of Hematology.

引用

页码：371 / 382

页数：12

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