Therapeutic potential and mechanism of action of oligonucleotides and ribozymes.
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.
Lavrovsky
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Cellular and Structural Biology, The University of Texas Health Science Center at San Antonio, 7703 Floyd Curl Drive, San Antonio, Texas 78284-7762, USA.
Chen
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Roy
A K
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D015870:Gene Expression / Q000187:drug effects; D006801:Humans; D009841:Oligonucleotides / Q000494:pharmacology / Q000627:therapeutic use*; D016376:Oligonucleotides, Antisense / Q000494:pharmacology / Q000627:therapeutic use; D016337:RNA, Catalytic / Q000494:pharmacology / Q000627:therapeutic use*; D012333:RNA, Messenger / Q000378:metabolism
D009841:Oligonucleotides; D016376:Oligonucleotides, Antisense; D016337:RNA, Catalytic; D012333:RNA, Messenger