ENA oligonucleotides as therapeutics.
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.
Koizumi
Makoto
M
Core Technology Research Laboratories, Sankyo Co Ltd, 1-2-58 Hiromachi, Shinagawa-ku, Tokyo 140-8710, Japan. koizum@sankyo.co.jp
D000818:Animals; D001952:Bridged-Ring Compounds / Q000138:chemical synthesis / Q000737:chemistry; D005030:Ethylenes / Q000138:chemical synthesis / Q000737:chemistry*; D005091:Exons; D006801:Humans; D008968:Molecular Conformation; D009696:Nucleic Acids / Q000138:chemical synthesis / Q000737:chemistry*; D016376:Oligonucleotides, Antisense / Q000138:chemical synthesis / Q000737:chemistry* / Q000235:genetics / Q000494:pharmacology* / Q000627:therapeutic use*; D034741:RNA, Small Interfering / Q000378:metabolism
D001952:Bridged-Ring Compounds; D005030:Ethylenes; D009696:Nucleic Acids; D016376:Oligonucleotides, Antisense; D034741:RNA, Small Interfering