The therapeutic potential of ribozymes

H A James, Ian Gibson

Research output: Contribution to journalArticlepeer-review

114 Citations (Scopus)

Abstract

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, ras 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.
Original languageEnglish
Pages (from-to)371-82
Number of pages12
JournalBlood
Volume91
Issue number2
Publication statusPublished - 15 Jan 1998

Keywords

  • Gene Expression Regulation
  • Genetic Therapy
  • Humans
  • RNA, Catalytic

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