Abstract
Oligonucleotides containing 2'-O,4'-C-ethylene-bridged nucleic acid (ENA) residues have two notable properties: (i) considerable affinity to complementary single-stranded RNA and double-stranded DNA; and (ii) dramatically high resistance against nucleases. On the basis of these properties, the in vitro and in vivo applications of ENA for gene silencing as antisense oligonucleotides, triplex-forming oligonucleotides and small interfering RNAs are discussed here. ENA oligonucleotides also have potential as therapeutic agents for Duchenne muscular dystrophy by a mechanism of exon skipping.
MeSH terms
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Animals
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Bridged-Ring Compounds / chemical synthesis
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Bridged-Ring Compounds / chemistry
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Ethylenes / chemical synthesis
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Ethylenes / chemistry*
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Exons
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Humans
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Molecular Conformation
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Nucleic Acids / chemical synthesis
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Nucleic Acids / chemistry*
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Oligonucleotides, Antisense / chemical synthesis
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Oligonucleotides, Antisense / chemistry*
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Oligonucleotides, Antisense / genetics
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Oligonucleotides, Antisense / pharmacology*
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Oligonucleotides, Antisense / therapeutic use*
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RNA, Small Interfering / metabolism
Substances
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Bridged-Ring Compounds
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Ethylenes
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Nucleic Acids
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Oligonucleotides, Antisense
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RNA, Small Interfering