Therapeutic potential and mechanism of action of oligonucleotides and ribozymes

Yan Lavrovsky, Shuo Chen, Arun K. Roy

Research output: Contribution to journalReview articlepeer-review

18 Scopus citations


Specific inactivation of gene expression is an attractive approach for rational drug design to combat degenerative diseases and infectious agents. Oligonucleotide-directed triple-helix formation at cis-acting elements of gene promoters, short oligonucleotides containing base sequences that are complementary to the messenger RNA (antisense oligos), and RNA enzymes (ribozymes) that specifically cleave messenger RNA molecules are currently being used both as experimental tools and as therapeutic agents. Mechanisms of action of various oligonucleotide-based drugs, recent developments in the drug-delivery approaches, and future potentials are discussed in this review.

Original languageEnglish (US)
Pages (from-to)11-22
Number of pages12
JournalBiochemical and Molecular Medicine
Issue number1
StatePublished - Oct 1997

ASJC Scopus subject areas

  • Biochemistry


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