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REVIEW ARTICLE
The Therapeutic Potential of Ribozymes
Helen A. James and
Ian Gibson
From the School of Biological Sciences, University of East Anglia,
Norwich, Norfolk, UK.
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.
Blood, Vol. 91 No. 2 (January 15), 1998:
pp. 371-382
© 1998 by The American Society of Hematology.
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