Antisense oligonucleotide derivatives as gene-targeted drugs.
The strategies and problems involved in designing oligonucleotide derivatives as gene-targeted drugs are discussed. Experiments with isolated and cellular nucleic acids, studies with infected cell cultures, and preliminary animal tests all demonstrate that various derivatives of complementary oligonucleotides (antisense oligonucleotide derivatives) can act as extremely specific and potent inhibitors of gene expression. The design and synthesis of more stable oligonucleotide analogues that can enter mammalian cells and efficiently affect preselected nucleic acids will result in the development of a new generation of drugs, including those with antiviral and anticancer properties.
Knorre
D G
DG
Institute of Bioorganic Chemistry, Siberian Division of the Academy of Sciences of the USSR, Novosibirsk.
Vlassov
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VV
D000818:Animals; D000890:Anti-Infective Agents / Q000494:pharmacology; D000970:Antineoplastic Agents / Q000008:administration & dosage; D001483:Base Sequence; D004247:DNA / Q000187:drug effects; D015195:Drug Design; D004353:Drug Evaluation, Preclinical; D005057:Eukaryotic Cells / Q000187:drug effects / Q000378:metabolism; D005786:Gene Expression Regulation / Q000187:drug effects; D006801:Humans; D051379:Mice; D008969:Molecular Sequence Data; D016376:Oligonucleotides, Antisense / Q000138:chemical synthesis / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use; D014176:Protein Biosynthesis / Q000187:drug effects; D012313:RNA / Q000187:drug effects; D014158:Transcription, Genetic / Q000187:drug effects
D000890:Anti-Infective Agents; D000970:Antineoplastic Agents; D016376:Oligonucleotides, Antisense; D012313:RNA; D004247:DNA