Ribozymes in Biotechnology


Ribozymes are RNA molecules that catalyse the cleavage and/or ligation of other RNA molecules. This capacity of ribozymes has therapeutic application for the inactivation of deleterious genes and the repair of mutated genes involved in many disease states.

Keywords: ribozyme; catalytic RNA; antisense; gene therapy

Figure 1.

The hammerhead (a) and M1 ribozymes (b), and the group I self‐splicing intron (c). For the hammerhead N equals A, C, G or U, and H equals A, C or U. Arrows indicate the cleavage sites on the hammerhead and the group I intron. Due to the large size of the M1 ribozyme and the group I intron, only schematic illustrations of their secondary structures are shown. IGS is the internal guide sequence of the group I intron, and thick lines indicate the exons. The invariant G is indicated in (c).

Figure 2.

The group II self‐splicing intron (a), and the hepatitis δ (b), hairpin (c) and Neurospora VS ribozymes (d). The circled ‘A’ in the group II intron is the internal adenosine that serves as the initiating nucleophile. Exons are indicated by thick lines. Arrows indicate the locations of cleavage sites. N equals A, C, G or U; P equals A or G; and Y equals C or U.

Figure 3.

Therapeutic applications of ribozymes. (a) Cleavage of a complementary messenger RNA (mRNA) by the hammerhead ribozyme. (b) Normal reaction catalysed by ribonuclease P or the M1 ribozyme. (c) An external guide sequence (EGS) can be used to elicit in vivo cleavage of a complementary RNA target by ribonuclease P. (d) The use of the group I ribozyme to trans‐splice and repair a defective mRNA.


Further Reading

Amarzguioui M and Prydz H (1998) Hammerhead ribozyme design and application. Cellular and Molecular Life Sciences 54: 1175–1202.

Birikh KR, Heaton PA and Eckstein F (1997) The structure, function and application of the hammerhead ribozyme. European Journal of Biochemistry 245: 1–16.

Burke JM (1996) Hairpin ribozyme: current status and future prospects. Biochemical Society Transactions 24: 608–615.

Earnshaw DJ and Gait MJ (1997) Progress toward the structure and therapeutic use of the hairpin ribozyme. Antisense Nucleic Acid Drug Development 7: 403–411.

Friedmann T (ed.) (1999) The Development of Human Gene Therapy. Cold Spring Harbor, New York: Cold Spring Harbor Laboratory Press.

Gibson SA and Shillitoe EJ (1997) Ribozymes. Their functions and strategies for their use. Molecular Biotechnology 7: 125–137.

Kiehntopf M, Esquivel EL, Brach MA and Herrmann F (1995) Clinical applications of ribozymes. Lancet 345: 1027–1031.

Kiehntopf M, Esquivel EL, Brach MA and Herrmann F (1995) Ribozymes: biology, biochemistry, and implications for clinical medicine. Journal of Molecular Medicine 73: 65–71.

Phylactou LA, Kilpatrick MW and Wood MJ (1998) Ribozymes as therapeutic tools for genetic disease. Human Molecular Genetics 7: 1649–1653.

Scanlon KJ (ed.) (1998) Therapeutic Applications of Ribozymes. Totowa, New Jersey: Humana Press.

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How to Cite close
Ruffner, Duane E(Sep 2001) Ribozymes in Biotechnology. In: eLS. John Wiley & Sons Ltd, Chichester. http://www.els.net [doi: 10.1038/npg.els.0000985]