Engineered catalytic RNA and DNA

New biochemical tools for drug discovery and design

David Steele, Alexis Kertsburg, Garrett Soukup

Research output: Contribution to journalReview article

18 Citations (Scopus)

Abstract

Since the fundamental discovery that RNA catalyzes critical biological reactions, the conceptual and practical utility of nucleic acid catalysts as molecular therapeutic and diagnostic agents continually develops. RNA and DNA catalysts are particularly attractive tools for drug discovery and design due to their relative ease of synthesis and tractable rational design features. Such catalysts can intervene in cellular or viral gene expression by effectively destroying virtually any target RNA, repairing messenger RNAs derived from mutant genes, or directly disrupting target genes. Consequently, catalytic nucleic acids are apt tools for dissecting gene function and for effecting gene pharmacogenomic strategies. It is in this capacity that RNA and DNA catalysts have been most widely utilized to affect gene expression of medically relevant targets associated with various disease states, where a number of such catalysts are presently being evaluated in clinical trials. Additionally, biotechnological prospects for catalytic nucleic acids are seemingly unlimited. Controllable nucleic acid catalysts, termed allosteric ribozymes or deoxyribozymes, form the basis of effector or ligand-dependent molecular switches and sensors. Allosteric nucleic acid catalysts promise to be useful tools for detecting and scrutinizing the function of specified components of the metabolome, proteome, transcriptome, and genome. The remarkable versatility of nucleic acid catalysis is thus the fountainhead for wide-ranging applications of ribozymes and deoxyribozymes in biomedical and biotechnological research.

Original languageEnglish
Pages (from-to)131-144
Number of pages14
JournalAmerican Journal of PharmacoGenomics
Volume3
Issue number2
DOIs
StatePublished - 2003

Fingerprint

Catalytic DNA
Catalytic RNA
Drug Design
Drug Discovery
Nucleic Acids
RNA
Genes
Gene Expression
Metabolome
Viral Genes
Molecular Pathology
Pharmacogenetics
DNA
Proteome
Catalysis
Transcriptome
Biomedical Research
Clinical Trials
Genome
Ligands

All Science Journal Classification (ASJC) codes

  • Pharmacology
  • Molecular Medicine
  • Genetics

Cite this

Engineered catalytic RNA and DNA : New biochemical tools for drug discovery and design. / Steele, David; Kertsburg, Alexis; Soukup, Garrett.

In: American Journal of PharmacoGenomics, Vol. 3, No. 2, 2003, p. 131-144.

Research output: Contribution to journalReview article

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