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Therapeutic Applications of Ribozymes

  • Chapter
Nucleic Acid Therapeutics in Cancer

Part of the book series: Cancer Drug Discovery and Development ((CDD&D))

Abstract

Ribozymes are RNA molecules capable of acting as enzymes even in the complete absence of proteins. They have the catalytic activity of breaking and/ or forming covalent bonds with extraordinary specificity, accelerating the rate of these reactions. The ability of RNA to serve as a catalyst was first shown for the self-splicing Group I intron of Tetrahymena and the RNA moiety of RNase P (1–3). Subsequent to the discovery of these two RNA enzymes, RNA-mediated catalysis has been found associated with the self-splicing group II introns of yeast, fungal and plant mitochondria (as well as chloroplasts) (4), single-stranded plant viroid and virusoid RNAs (5–7), hepatitis delta virus (8), and a satellite RNA from Neurospora mitochondria (9). It is rather clear that the RNA component of the larger ribosomal subunit is functioning as a peptidyltransferase as well (10–12). The potential functioning of spliceosomal snRNAs as a ribozyme in complex with the pre-mRNA to catalyze pre-messenger-RNA splicing has also been proposed (13). It is highly likely that additional RNA catalytic motifs and new roles for RNA-mediated catalysis will also be found as we learn more about the genomes of a variety of organisms.

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  1. John J. Rossi PhD
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  1. Department of Internal Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA, USA

    Alan M. Gewirtz MD

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© 2004 Springer Science+Business Media New York

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Rossi, J.J. (2004). Therapeutic Applications of Ribozymes. In: Gewirtz, A.M. (eds) Nucleic Acid Therapeutics in Cancer. Cancer Drug Discovery and Development. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-59259-777-2_4

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  • DOI: https://doi.org/10.1007/978-1-59259-777-2_4

  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-4684-9858-5

  • Online ISBN: 978-1-59259-777-2

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