THE THERAPEUTIC POTENTIAL OF RIBOZYMES

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