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Volumn 35, Issue 3, 2017, Pages 238-248

The chemical evolution of oligonucleotide therapies of clinical utility

Author keywords

[No Author keywords available]

Indexed keywords

ARCHITECTURE; CHEMICAL MODIFICATION; GENES; PRODUCTIVITY; TISSUE;

EID: 85014846150     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt.3765     Document Type: Review
Times cited : (848)

References (138)
  • 1
    • 0026175039 scopus 로고
    • Informational drugs: A new concept in pharmacology
    • Cohen, J. S. Informational drugs: a new concept in pharmacology. Antisense Res. Dev. 1, 191-193 (1991).
    • (1991) Antisense Res. Dev. , vol.1 , pp. 191-193
    • Cohen, J.S.1
  • 2
    • 84856389509 scopus 로고    scopus 로고
    • RNA-based therapeutics: Current progress and future prospects
    • Burnett, J. C., Rossi, J. J. RNA-based therapeutics: current progress and future prospects. Chem. Biol. 19, 60-71 (2012).
    • (2012) Chem. Biol. , vol.19 , pp. 60-71
    • Burnett, J.C.1    Rossi, J.J.2
  • 4
    • 84869120708 scopus 로고    scopus 로고
    • Discovering the first microRNA-targeted drug
    • Lindow, M., Kauppinen, S. Discovering the first microRNA-targeted drug. J. Cell Biol. 199, 407-412 (2012).
    • (2012) J. Cell Biol. , vol.199 , pp. 407-412
    • Lindow, M.1    Kauppinen, S.2
  • 6
    • 84975045794 scopus 로고    scopus 로고
    • From the RNA world to the clinic
    • Sullenger, B. A., Nair, S. From the RNA world to the clinic. Science 352, 1417-1420 (2016).
    • (2016) Science , vol.352 , pp. 1417-1420
    • Sullenger, B.A.1    Nair, S.2
  • 8
    • 84865526525 scopus 로고    scopus 로고
    • Designing chemically modified oligonucleotides for targeted gene silencing
    • Deleavey, G. F., Damha, M. J. Designing chemically modified oligonucleotides for targeted gene silencing. Chem. Biol. 19, 937-954 (2012).
    • (2012) Chem. Biol. , vol.19 , pp. 937-954
    • Deleavey, G.F.1    Damha, M.J.2
  • 9
    • 84947608741 scopus 로고    scopus 로고
    • Oligonucleotide therapeutics: Chemistry, delivery and clinical progress
    • Sharma, V. K., Watts, J. K. Oligonucleotide therapeutics: chemistry, delivery and clinical progress. Future Med. Chem. 7, 2221-2242 (2015).
    • (2015) Future Med. Chem. , vol.7 , pp. 2221-2242
    • Sharma, V.K.1    Watts, J.K.2
  • 10
    • 84994817779 scopus 로고    scopus 로고
    • The medicinal chemistry of therapeutic oligonucleotides
    • Wan, W. B., Seth, P. P. The medicinal chemistry of therapeutic oligonucleotides. J. Med. Chem. 59, 9645-9667 (2016).
    • (2016) J. Med. Chem. , vol.59 , pp. 9645-9667
    • Wan, W.B.1    Seth, P.P.2
  • 11
    • 85050192295 scopus 로고    scopus 로고
    • eds. Chackalamannil, S., et al. Elsevier, in the press
    • Ito, K. R., Obika, S. in Comprehensive Medicinal Chemistry 3rd edn. (eds. Chackalamannil, S., et al. ) http://dx. doi. org/10. 1016/b978-0-12-409547-2. 12420-5 (Elsevier, in the press).
    • Comprehensive Medicinal Chemistry 3rd Edn.
    • Ito, K.R.1    Obika, S.2
  • 12
    • 0005772676 scopus 로고
    • Inhibition of Rous sarcoma viral RNA translation by a specific oligodeoxyribonucleotide
    • Stephenson, M. L., Zamecnik, P. C. Inhibition of Rous sarcoma viral RNA translation by a specific oligodeoxyribonucleotide. Proc. Natl. Acad. Sci. USA 75, 285-288 (1978).
    • (1978) Proc. Natl. Acad. Sci. USA , vol.75 , pp. 285-288
    • Stephenson, M.L.1    Zamecnik, P.C.2
  • 13
    • 0041582974 scopus 로고
    • Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide
    • Zamecnik, P. C., Stephenson, M. L. Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide. Proc. Natl. Acad. Sci. USA 75, 280-284 (1978).
    • (1978) Proc. Natl. Acad. Sci. USA , vol.75 , pp. 280-284
    • Zamecnik, P.C.1    Stephenson, M.L.2
  • 14
    • 77949512140 scopus 로고    scopus 로고
    • RNA targeting therapeutics: Molecular mechanisms of antisense oligonucleotides as a therapeutic platform
    • Bennett, C. F., Swayze, E. E. RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu. Rev. Pharmacol. Toxicol. 50, 259-293 (2010).
    • (2010) Annu. Rev. Pharmacol. Toxicol. , vol.50 , pp. 259-293
    • Bennett, C.F.1    Swayze, E.E.2
  • 15
    • 84915763945 scopus 로고    scopus 로고
    • Phosphorothioates essential components of therapeutic oligonucleotides
    • Eckstein, F. Phosphorothioates, essential components of therapeutic oligonucleotides. Nucleic Acid Ther. 24, 374-387 (2014).
    • (2014) Nucleic Acid Ther. , vol.24 , pp. 374-387
    • Eckstein, F.1
  • 16
    • 85014812999 scopus 로고    scopus 로고
    • Antisense Drug Technology: Principles, strategies, applications
    • Edited by Stanley T. Crooke
    • Fluiter, K. Antisense Drug Technology: Principles, Strategies, Applications. Edited by Stanley T. Crooke. ChemMedChem 4, 879 (2009).
    • (2009) ChemMedChem , vol.4 , pp. 879
    • Fluiter, K.1
  • 17
    • 84937252963 scopus 로고    scopus 로고
    • Pharmacokinetics biodistribution and cell uptake of antisense oligonucleotides
    • Geary, R. S., Norris, D., Yu, R., Bennett, C. F. Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides. Adv. Drug Deliv. Rev. 87, 46-51 (2015).
    • (2015) Adv. Drug Deliv. Rev. , vol.87 , pp. 46-51
    • Geary, R.S.1    Norris, D.2    Yu, R.3    Bennett, C.F.4
  • 18
    • 79959479529 scopus 로고    scopus 로고
    • Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes
    • Koller, E., et al. Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes. Nucleic Acids Res. 39, 4795-4807 (2011).
    • (2011) Nucleic Acids Res. , vol.39 , pp. 4795-4807
    • Koller, E.1
  • 19
    • 0029077840 scopus 로고
    • Characterization of fully 2-modified oligoribonucleotide hetero-and homoduplex hybridization and nuclease sensitivity
    • Cummins, L. L., et al. Characterization of fully 2-modified oligoribonucleotide hetero-and homoduplex hybridization and nuclease sensitivity. Nucleic Acids Res. 23, 2019-2024 (1995).
    • (1995) Nucleic Acids Res. , vol.23 , pp. 2019-2024
    • Cummins, L.L.1
  • 20
    • 15844400778 scopus 로고    scopus 로고
    • Nuclease resistance and antisense activity of modified oligonucleotides targeted to Ha-ras
    • Monia, B. P., Johnston, J. F., Sasmor, H., Cummins, L. L. Nuclease resistance and antisense activity of modified oligonucleotides targeted to Ha-ras. J. Biol. Chem. 271, 14533-14540 (1996).
    • (1996) J. Biol. Chem. , vol.271 , pp. 14533-14540
    • Monia, B.P.1    Johnston, J.F.2    Sasmor, H.3    Cummins, L.L.4
  • 21
    • 33644620908 scopus 로고    scopus 로고
    • Chemical modification of siRNAs to improve serum stability without loss of efficacy
    • Choung, S., Kim, Y. J., Kim, S., Park, H. O., Choi, Y. C. Chemical modification of siRNAs to improve serum stability without loss of efficacy. Biochem. Biophys. Res. Commun. 342, 919-927 (2006).
    • (2006) Biochem. Biophys. Res. Commun. , vol.342 , pp. 919-927
    • Choung, S.1    Kim, Y.J.2    Kim, S.3    Park, H.O.4    Choi, Y.C.5
  • 22
    • 34548108038 scopus 로고    scopus 로고
    • 2-O-methyl-modified RNAs act as TLR7 antagonists
    • Robbins, M., et al. 2-O-methyl-modified RNAs act as TLR7 antagonists. Mol. Ther. 15, 1663-1669 (2007).
    • (2007) Mol. Ther. , vol.15 , pp. 1663-1669
    • Robbins, M.1
  • 23
    • 0032750621 scopus 로고    scopus 로고
    • 2-carbohydrate modifications in antisense oligonucleotide therapy: Importance of conformation, configuration and conjugation
    • Manoharan, M. 2-carbohydrate modifications in antisense oligonucleotide therapy: importance of conformation, configuration and conjugation. Biochim. Biophys. Acta 1489, 117-130 (1999).
    • (1999) Biochim. Biophys. Acta , vol.1489 , pp. 117-130
    • Manoharan, M.1
  • 24
    • 0030727765 scopus 로고    scopus 로고
    • The ups and downs of nucleic acid duplex stability: Structure-stability studies on chemically-modified DNA:RNA duplexes
    • Freier, S. M., Altmann, K.-H. The ups and downs of nucleic acid duplex stability: structure-stability studies on chemically-modified DNA:RNA duplexes. Nucleic Acids Res. 25, 4429-4443 (1997).
    • (1997) Nucleic Acids Res. , vol.25 , pp. 4429-4443
    • Freier, S.M.1    Altmann, K.-H.2
  • 25
    • 1842835804 scopus 로고    scopus 로고
    • Flexible and frozen sugar-modified nucleic acids-modulation of biological activity through furanose ring dynamics in the antisense strand
    • Mangos, M. M., Damha, M. J. Flexible and frozen sugar-modified nucleic acids-modulation of biological activity through furanose ring dynamics in the antisense strand. Curr. Top. Med. Chem. 2, 1147-1171 (2002).
    • (2002) Curr. Top. Med. Chem. , vol.2 , pp. 1147-1171
    • Mangos, M.M.1    Damha, M.J.2
  • 26
    • 80052913097 scopus 로고    scopus 로고
    • An overview of sugar-modified oligonucleotides for antisense therapeutics
    • Prakash, T. P. An overview of sugar-modified oligonucleotides for antisense therapeutics. Chem. Biodivers. 8, 1616-1641 (2011).
    • (2011) Chem. Biodivers. , vol.8 , pp. 1616-1641
    • Prakash, T.P.1
  • 27
    • 21744453184 scopus 로고    scopus 로고
    • Probing the influence of stereoelectronic effects on the biophysical properties of oligonucleotides: Comprehensive analysis of the RNA affinity, nuclease resistance, crystal structure of ten 2-O-ribonucleic acid modifications
    • Egli, M., et al. Probing the influence of stereoelectronic effects on the biophysical properties of oligonucleotides: comprehensive analysis of the RNA affinity, nuclease resistance, crystal structure of ten 2-O-ribonucleic acid modifications. Biochemistry 44, 9045-9057 (2005).
    • (2005) Biochemistry , vol.44 , pp. 9045-9057
    • Egli, M.1
  • 28
    • 0029099305 scopus 로고
    • A New access to 2-O-alkylated ribonucleosides and properties of 2-O-alkylated oligoribonucleotides
    • Martin, P. A New access to 2-O-alkylated ribonucleosides and properties of 2-O-alkylated oligoribonucleotides. Helv. Chim. Acta 78, 486-504 (1995).
    • (1995) Helv. Chim. Acta , vol.78 , pp. 486-504
    • Martin, P.1
  • 29
    • 0001120760 scopus 로고    scopus 로고
    • Preorganization of DNA: Design principles for improving nucleic acid recognition by synthetic oligonucleotides
    • Kool, E. T. Preorganization of DNA: Design principles for improving nucleic acid recognition by synthetic oligonucleotides. Chem. Rev. 97, 1473-1488 (1997).
    • (1997) Chem. Rev. , vol.97 , pp. 1473-1488
    • Kool, E.T.1
  • 30
    • 80055018807 scopus 로고    scopus 로고
    • Stability and mismatch discrimination of locked nucleic acid-DNA duplexes
    • Owczarzy, R., You, Y., Groth, C. L., Tataurov, A. V. Stability and mismatch discrimination of locked nucleic acid-DNA duplexes. Biochemistry 50, 9352-9367 (2011).
    • (2011) Biochemistry , vol.50 , pp. 9352-9367
    • Owczarzy, R.1    You, Y.2    Groth, C.L.3    Tataurov, A.V.4
  • 31
    • 0032473890 scopus 로고    scopus 로고
    • LNA (locked nucleic acids): Synthesis of the adenine, cytosine, guanine, 5-methylcytosine, thymine and uracil bicyclonucleoside monomers, oligomerisation, unprecedented nucleic acid recognition
    • Koshkin, A. A., et al. LNA (locked nucleic acids): synthesis of the adenine, cytosine, guanine, 5-methylcytosine, thymine and uracil bicyclonucleoside monomers, oligomerisation, unprecedented nucleic acid recognition. Tetrahedron 54, 3607-3630 (1998).
    • (1998) Tetrahedron , vol.54 , pp. 3607-3630
    • Koshkin, A.A.1
  • 32
    • 0032560835 scopus 로고    scopus 로고
    • Stability and structural features of the duplexes containing nucleoside analogues with a fixed N-type conformation, 2-O, 4-C-methyleneribonucleosides
    • Obika, S., et al. Stability and structural features of the duplexes containing nucleoside analogues with a fixed N-type conformation, 2-O, 4-C-methyleneribonucleosides. Tetrahedr. Lett. 39, 5401-5404 (1998).
    • (1998) Tetrahedr. Lett. , vol.39 , pp. 5401-5404
    • Obika, S.1
  • 33
    • 0343065617 scopus 로고    scopus 로고
    • LNA (locked nucleic acids) synthesis and high-affinity nucleic acid recognition
    • Singh, S. K., Nielsen, P., Koshkin, A. A., Wengel, J. LNA (locked nucleic acids): synthesis and high-affinity nucleic acid recognition. Chem. Commun. (Camb. ) 1998 455-456 (1998).
    • (1998) Chem. Commun. (Camb. ) , vol.1998 , pp. 455-456
    • Singh, S.K.1    Nielsen, P.2    Koshkin, A.A.3    Wengel, J.4
  • 34
    • 84878634770 scopus 로고    scopus 로고
    • Locked nucleic acid: Tighter is different
    • Watts, J. K. Locked nucleic acid: tighter is different. Chem. Commun. (Camb. ) 49, 5618-5620 (2013).
    • (2013) Chem. Commun. (Camb. ) , vol.49 , pp. 5618-5620
    • Watts, J.K.1
  • 35
    • 1242297789 scopus 로고    scopus 로고
    • Nuclear antisense effects in cyclophilin A pre-mRNA splicing by oligonucleotides: A comparison of tricyclo-DNA with LNA
    • Ittig, D., Liu, S., Renneberg, D., Schümperli, D., Leumann, C. J. Nuclear antisense effects in cyclophilin A pre-mRNA splicing by oligonucleotides: a comparison of tricyclo-DNA with LNA. Nucleic Acids Res. 32, 346-353 (2004).
    • (2004) Nucleic Acids Res. , vol.32 , pp. 346-353
    • Ittig, D.1    Liu, S.2    Renneberg, D.3    Schümperli, D.4    Leumann, C.J.5
  • 36
    • 84925507915 scopus 로고    scopus 로고
    • Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers
    • Goyenvalle, A., et al. Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers. Nat. Med. 21, 270-275 (2015).
    • (2015) Nat. Med. , vol.21 , pp. 270-275
    • Goyenvalle, A.1
  • 37
  • 38
    • 84894522337 scopus 로고    scopus 로고
    • A kinetic model explains why shorter and less affine enzyme-recruiting oligonucleotides can be more potent
    • Pedersen, L., Hagedorn, P. H., Lindholm, M. W., Lindow, M. A kinetic model explains why shorter and less affine enzyme-recruiting oligonucleotides can be more potent. Mol. Ther. Nucleic Acids 3, e149 (2014).
    • (2014) Mol. Ther. Nucleic Acids , vol.3 , pp. e149
    • Pedersen, L.1    Hagedorn, P.H.2    Lindholm, M.W.3    Lindow, M.4
  • 39
    • 78649877710 scopus 로고    scopus 로고
    • Short locked nucleic acid antisense oligonucleotides potently reduce apolipoprotein B mRNA and serum cholesterol in mice and non-human primates
    • Straarup, E. M., et al. Short locked nucleic acid antisense oligonucleotides potently reduce apolipoprotein B mRNA and serum cholesterol in mice and non-human primates. Nucleic Acids Res. 38, 7100-7111 (2010).
    • (2010) Nucleic Acids Res. , vol.38 , pp. 7100-7111
    • Straarup, E.M.1
  • 40
    • 59449093219 scopus 로고    scopus 로고
    • Short antisense oligonucleotides with novel 2-4 conformationaly restricted nucleoside analogues show improved potency without increased toxicity in animals
    • Seth, P. P., et al. Short antisense oligonucleotides with novel 2-4 conformationaly restricted nucleoside analogues show improved potency without increased toxicity in animals. J. Med. Chem. 52, 10-13 (2009).
    • (2009) J. Med. Chem. , vol.52 , pp. 10-13
    • Seth, P.P.1
  • 41
    • 33846902312 scopus 로고    scopus 로고
    • Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals
    • Swayze, E. E., et al. Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals. Nucleic Acids Res. 35, 687-700 (2007).
    • (2007) Nucleic Acids Res. , vol.35 , pp. 687-700
    • Swayze, E.E.1
  • 42
    • 84949911119 scopus 로고    scopus 로고
    • In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization
    • Kamola, P. J., et al. In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization. Nucleic Acids Res. 43, 8638-8650 (2015).
    • (2015) Nucleic Acids Res. , vol.43 , pp. 8638-8650
    • Kamola, P.J.1
  • 43
    • 84963812653 scopus 로고    scopus 로고
    • Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts
    • Burel, S. A., et al. Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts. Nucleic Acids Res. 44, 2093-2109 (2016).
    • (2016) Nucleic Acids Res. , vol.44 , pp. 2093-2109
    • Burel, S.A.1
  • 44
    • 84979738435 scopus 로고    scopus 로고
    • Ribonuclease H1-dependent hepatotoxicity caused by locked nucleic acid-modified gapmer antisense oligonucleotides
    • Kasuya, T., et al. Ribonuclease H1-dependent hepatotoxicity caused by locked nucleic acid-modified gapmer antisense oligonucleotides. Sci. Rep. 6, 30377 (2016).
    • (2016) Sci. Rep. , vol.6 , pp. 30377
    • Kasuya, T.1
  • 45
    • 84941894875 scopus 로고    scopus 로고
    • Allele-selective inhibition of mutant huntingtin with 2-thio-and C5-triazolylphenyl-deoxythymidine-modified antisense oligonucleotides
    • Stergaard, M. E., et al. Allele-selective inhibition of mutant huntingtin with 2-thio-and C5-triazolylphenyl-deoxythymidine-modified antisense oligonucleotides. Nucleic Acid Ther. 25, 266-274 (2015).
    • (2015) Nucleic Acid Ther. , vol.25 , pp. 266-274
    • Stergaard, M.E.1
  • 46
    • 84927177598 scopus 로고    scopus 로고
    • In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides
    • Southwell, A. L., et al. In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides. Mol. Ther. 22, 2093-2106 (2014).
    • (2014) Mol. Ther. , vol.22 , pp. 2093-2106
    • Southwell, A.L.1
  • 47
    • 84890057093 scopus 로고    scopus 로고
    • Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS
    • ∅stergaard, M. E., et al. Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS. Nucleic Acids Res. 41, 9634-9650 (2013).
    • (2013) Nucleic Acids Res. , vol.41 , pp. 9634-9650
    • Stergaard, M.E.1
  • 48
    • 0032539186 scopus 로고    scopus 로고
    • Hybrids of RNA and arabinonucleic acids (ANA and 2F-ANA) are substrates of Ribonuclease H
    • Damha, M. J., et al. Hybrids of RNA and arabinonucleic acids (ANA and 2F-ANA) are substrates of Ribonuclease H. J. Am. Chem. Soc. 120, 12976-12977 (1998).
    • (1998) J. Am. Chem. Soc. , vol.120 , pp. 12976-12977
    • Damha, M.J.1
  • 49
    • 46249131334 scopus 로고    scopus 로고
    • 2F-arabinonucleic acids (2F-ANA)-history, properties, new frontiers
    • Watts, J. K., Damha, M. J. 2F-arabinonucleic acids (2F-ANA)-history, properties, new frontiers. Can. J. Chem. 86, 641-656 (2008).
    • (2008) Can. J. Chem. , vol.86 , pp. 641-656
    • Watts, J.K.1    Damha, M.J.2
  • 50
    • 0037460131 scopus 로고    scopus 로고
    • Efficient RNase H-directed cleavage of RNA promoted by antisense DNA or 2F-ANA constructs containing acyclic nucleotide inserts
    • Mangos, M. M., et al. Efficient RNase H-directed cleavage of RNA promoted by antisense DNA or 2F-ANA constructs containing acyclic nucleotide inserts. J. Am. Chem. Soc. 125, 654-661 (2003).
    • (2003) J. Am. Chem. Soc. , vol.125 , pp. 654-661
    • Mangos, M.M.1
  • 51
    • 0037119693 scopus 로고    scopus 로고
    • Oligonucleotides comprised of alternating 2-deoxy-2-fluoro-d-arabinonucleosides and d-2-deoxyribonucleosides (2F-ANA/DNA 'altimers') induce efficient RNA cleavage mediated by RNase H
    • Min, K.-L., Viazovkina, E., Galarneau, A., Parniak, M. A., Damha, M. J. Oligonucleotides comprised of alternating 2-deoxy-2-fluoro-d-arabinonucleosides and d-2-deoxyribonucleosides (2F-ANA/DNA 'altimers') induce efficient RNA cleavage mediated by RNase H. Bioorg. Med. Chem. Lett. 12, 2651-2654 (2002).
    • (2002) Bioorg. Med. Chem. Lett. , vol.12 , pp. 2651-2654
    • Min, K.-L.1    Viazovkina, E.2    Galarneau, A.3    Parniak, M.A.4    Damha, M.J.5
  • 52
    • 84890805770 scopus 로고    scopus 로고
    • Eteplirsen for the treatment of Duchenne muscular dystrophy
    • Mendell, J. R., et al. Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann. Neurol. 74, 637-647 (2013).
    • (2013) Ann. Neurol. , vol.74 , pp. 637-647
    • Mendell, J.R.1
  • 53
    • 84960517144 scopus 로고    scopus 로고
    • Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy
    • Chiriboga, C. A., et al. Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy. Neurology 86, 890-897 (2016).
    • (2016) Neurology , vol.86 , pp. 890-897
    • Chiriboga, C.A.1
  • 54
    • 85011327851 scopus 로고    scopus 로고
    • Big win possible for Ionis/Biogen antisense drug in muscular atrophy
    • Garber, K. Big win possible for Ionis/Biogen antisense drug in muscular atrophy. Nat. Biotechnol. 34, 1002-1003 (2016).
    • (2016) Nat. Biotechnol. , vol.34 , pp. 1002-1003
    • Garber, K.1
  • 55
    • 84905503012 scopus 로고    scopus 로고
    • Therapeutic targeting of microRNAs: Current status and future challenges
    • Li, Z., Rana, T. M. Therapeutic targeting of microRNAs: current status and future challenges. Nat. Rev. Drug Discov. 13, 622-638 (2014).
    • (2014) Nat. Rev. Drug Discov. , vol.13 , pp. 622-638
    • Li, Z.1    Rana, T.M.2
  • 56
    • 77955469975 scopus 로고    scopus 로고
    • A direct comparison of anti-microRNA oligonucleotide potency
    • Lennox, K. A., Behlke, M. A. A direct comparison of anti-microRNA oligonucleotide potency. Pharm. Res. 27, 1788-1799 (2010).
    • (2010) Pharm. Res. , vol.27 , pp. 1788-1799
    • Lennox, K.A.1    Behlke, M.A.2
  • 57
    • 84891765459 scopus 로고    scopus 로고
    • Miravirsen (SPC3649) can inhibit the biogenesis of miR-122
    • Gebert, L. F., et al. Miravirsen (SPC3649) can inhibit the biogenesis of miR-122. Nucleic Acids Res. 42, 609-621 (2014).
    • (2014) Nucleic Acids Res. , vol.42 , pp. 609-621
    • Gebert, L.F.1
  • 58
    • 79953202455 scopus 로고    scopus 로고
    • Silencing of microRNA families by seed-targeting tiny LNAs
    • Obad, S., et al. Silencing of microRNA families by seed-targeting tiny LNAs. Nat. Genet. 43, 371-378 (2011).
    • (2011) Nat. Genet. , vol.43 , pp. 371-378
    • Obad, S.1
  • 59
    • 84867619259 scopus 로고    scopus 로고
    • Pharmacodynamics and subchronic toxicity in mice and monkeys of ISIS 388626, a second-generation antisense oligonucleotide that targets human sodium glucose cotransporter 2
    • Zanardi, T. A., et al. Pharmacodynamics and subchronic toxicity in mice and monkeys of ISIS 388626, a second-generation antisense oligonucleotide that targets human sodium glucose cotransporter 2. J. Pharmacol. Exp. Ther. 343, 489-496 (2012).
    • (2012) J. Pharmacol. Exp. Ther. , vol.343 , pp. 489-496
    • Zanardi, T.A.1
  • 60
    • 84959387101 scopus 로고    scopus 로고
    • Enhancing antisense efficacy with multimers and multi-targeting oligonucleotides (MTOs) using cleavable linkers
    • Subramanian, R. R., et al. Enhancing antisense efficacy with multimers and multi-targeting oligonucleotides (MTOs) using cleavable linkers. Nucleic Acids Res. 43, 9123-9132 (2015).
    • (2015) Nucleic Acids Res. , vol.43 , pp. 9123-9132
    • Subramanian, R.R.1
  • 61
    • 79955394091 scopus 로고    scopus 로고
    • Novel oligonucleotides containing two 3-ends complementary to target mRNA show optimal gene-silencing activity
    • Bhagat, L., et al. Novel oligonucleotides containing two 3-ends complementary to target mRNA show optimal gene-silencing activity. J. Med. Chem. 54, 3027-3036 (2011).
    • (2011) J. Med. Chem. , vol.54 , pp. 3027-3036
    • Bhagat, L.1
  • 62
    • 0037108281 scopus 로고    scopus 로고
    • Stereodefined phosphorothioate analogues of DNA: Relative thermodynamic stability of the model PS-DNA/DNA and PS-DNA/RNA complexes
    • Boczkowska, M., Guga, P., Stec, W. J. Stereodefined phosphorothioate analogues of DNA: relative thermodynamic stability of the model PS-DNA/DNA and PS-DNA/RNA complexes. Biochemistry 41, 12483-12487 (2002).
    • (2002) Biochemistry , vol.41 , pp. 12483-12487
    • Boczkowska, M.1    Guga, P.2    Stec, W.J.3
  • 63
    • 0029594158 scopus 로고
    • Stereodifferentiation-The effect of P chirality of oligo(nucleoside phosphorothioates) on the activity of bacterial RNase H
    • Koziolkiewicz, M., Krakowiak, A., Kwinkowski, M., Boczkowska, M., Stec, W. J. Stereodifferentiation-the effect of P chirality of oligo(nucleoside phosphorothioates) on the activity of bacterial RNase H. Nucleic Acids Res. 23, 5000-5005 (1995).
    • (1995) Nucleic Acids Res. , vol.23 , pp. 5000-5005
    • Koziolkiewicz, M.1    Krakowiak, A.2    Kwinkowski, M.3    Boczkowska, M.4    Stec, W.J.5
  • 64
    • 0031457916 scopus 로고    scopus 로고
    • Stereodependent inhibition of plasminogen activator inhibitor type 1 by phosphorothioate oligonucleotides: Proof of sequence specificity in cell culture and in vivo rat experiments
    • Stec, W. J., et al. Stereodependent inhibition of plasminogen activator inhibitor type 1 by phosphorothioate oligonucleotides: proof of sequence specificity in cell culture and in vivo rat experiments. Antisense Nucleic Acid Drug Dev. 7, 567-573 (1997).
    • (1997) Antisense Nucleic Acid Drug Dev. , vol.7 , pp. 567-573
    • Stec, W.J.1
  • 65
    • 84915817112 scopus 로고    scopus 로고
    • Meeting report: 10th annual meeting of the Oligonucleotide Therapeutics Society
    • Gagnon, K. T., Watts, J. K. Meeting report: 10th annual meeting of the Oligonucleotide Therapeutics Society. Nucl. Acid Ther. 24, 428-434 (2014).
    • (2014) Nucl. Acid Ther. , vol.24 , pp. 428-434
    • Gagnon, K.T.1    Watts, J.K.2
  • 66
    • 0032545933 scopus 로고    scopus 로고
    • Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
    • Fire, A., et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391, 806-811 (1998).
    • (1998) Nature , vol.391 , pp. 806-811
    • Fire, A.1
  • 67
    • 0035942736 scopus 로고    scopus 로고
    • Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
    • Elbashir, S. M., et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411, 494-498 (2001).
    • (2001) Nature , vol.411 , pp. 494-498
    • Elbashir, S.M.1
  • 68
    • 77952688315 scopus 로고    scopus 로고
    • A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus
    • DeVincenzo, J., et al. A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus. Proc. Natl. Acad. Sci. USA 107, 8800-8805 (2010).
    • (2010) Proc. Natl. Acad. Sci. USA , vol.107 , pp. 8800-8805
    • DeVincenzo, J.1
  • 70
    • 33845780974 scopus 로고    scopus 로고
    • RNA learns from antisense
    • Corey, D. R. RNA learns from antisense. Nat. Chem. Biol. 3, 8-11 (2007).
    • (2007) Nat. Chem. Biol. , vol.3 , pp. 8-11
    • Corey, D.R.1
  • 71
    • 1942533474 scopus 로고    scopus 로고
    • In vivo activity of nuclease-resistant siRNAs
    • Layzer, J. M., et al. In vivo activity of nuclease-resistant siRNAs. RNA 10, 766-771 (2004).
    • (2004) RNA , vol.10 , pp. 766-771
    • Layzer, J.M.1
  • 72
    • 52049090811 scopus 로고    scopus 로고
    • Chemically modified siRNA: Tools and applications
    • Watts, J. K., Deleavey, G. F., Damha, M. J. Chemically modified siRNA: tools and applications. Drug Discov. Today 13, 842-855 (2008).
    • (2008) Drug Discov. Today , vol.13 , pp. 842-855
    • Watts, J.K.1    Deleavey, G.F.2    Damha, M.J.3
  • 73
    • 33745614074 scopus 로고    scopus 로고
    • Position-specific chemical modification of siRNAs reduces off-target" transcript silencing
    • Jackson, A. L., et al. Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing. RNA 12, 1197-1205 (2006).
    • (2006) RNA , vol.12 , pp. 1197-1205
    • Jackson, A.L.1
  • 74
    • 34447306000 scopus 로고    scopus 로고
    • Effect of siRNA nuclease stability on the in vitro and in vivo kinetics of siRNA-mediated gene silencing
    • Bartlett, D. W., Davis, M. E. Effect of siRNA nuclease stability on the in vitro and in vivo kinetics of siRNA-mediated gene silencing. Biotechnol. Bioeng. 97, 909-921 (2007).
    • (2007) Biotechnol. Bioeng. , vol.97 , pp. 909-921
    • Bartlett, D.W.1    Davis, M.E.2
  • 75
    • 13944261143 scopus 로고    scopus 로고
    • Fully 2-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA
    • Allerson, C. R., et al. Fully 2-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA. J. Med. Chem. 48, 901-904 (2005).
    • (2005) J. Med. Chem. , vol.48 , pp. 901-904
    • Allerson, C.R.1
  • 76
    • 21244497878 scopus 로고    scopus 로고
    • Positional effect of chemical modifications on short interference RNA activity in mammalian cells
    • Prakash, T. P., et al. Positional effect of chemical modifications on short interference RNA activity in mammalian cells. J. Med. Chem. 48, 4247-4253 (2005).
    • (2005) J. Med. Chem. , vol.48 , pp. 4247-4253
    • Prakash, T.P.1
  • 77
    • 20044396377 scopus 로고    scopus 로고
    • Activity of stabilized short interfering RNA in a mouse model of hepatitis B virus replication
    • Morrissey, D. V., et al. Activity of stabilized short interfering RNA in a mouse model of hepatitis B virus replication. Hepatology 41, 1349-1356 (2005).
    • (2005) Hepatology , vol.41 , pp. 1349-1356
    • Morrissey, D.V.1
  • 78
    • 77955233988 scopus 로고    scopus 로고
    • Synergistic effects between analogs of DNA and RNA improve the potency of siRNA-mediated gene silencing
    • Deleavey, G. F., et al. Synergistic effects between analogs of DNA and RNA improve the potency of siRNA-mediated gene silencing. Nucleic Acids Res. 38, 4547-4557 (2010).
    • (2010) Nucleic Acids Res. , vol.38 , pp. 4547-4557
    • Deleavey, G.F.1
  • 80
    • 84956528338 scopus 로고    scopus 로고
    • SiRNAmod: A database of experimentally validated chemically modified siRNAs
    • Dar, S. A., Thakur, A., Qureshi, A., Kumar, M. siRNAmod: A database of experimentally validated chemically modified siRNAs. Sci. Rep. 6, 20031 (2016).
    • (2016) Sci. Rep. , vol.6 , pp. 20031
    • Dar, S.A.1    Thakur, A.2    Qureshi, A.3    Kumar, M.4
  • 82
    • 9144247189 scopus 로고    scopus 로고
    • Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
    • Soutschek, J., et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432, 173-178 (2004).
    • (2004) Nature , vol.432 , pp. 173-178
    • Soutschek, J.1
  • 83
    • 0038606999 scopus 로고    scopus 로고
    • Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells
    • Czauderna, F., et al. Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells. Nucleic Acids Res. 31, 2705-2716 (2003).
    • (2003) Nucleic Acids Res. , vol.31 , pp. 2705-2716
    • Czauderna, F.1
  • 84
    • 79952066733 scopus 로고    scopus 로고
    • Unique gene-silencing and structural properties of 2-fluoro-modified siRNAs
    • Manoharan, M., et al. Unique gene-silencing and structural properties of 2-fluoro-modified siRNAs. Angew. Chem. Int. Ed. Engl. 50, 2284-2288 (2011).
    • (2011) Angew. Chem. Int. Ed. Engl. , vol.50 , pp. 2284-2288
    • Manoharan, M.1
  • 85
    • 84941072684 scopus 로고    scopus 로고
    • Systematic evaluation of antibody-mediated siRNA delivery using an industrial platform of THIOMAB-siRNA conjugates
    • Cuellar, T. L., et al. Systematic evaluation of antibody-mediated siRNA delivery using an industrial platform of THIOMAB-siRNA conjugates. Nucleic Acids Res. 43, 1189-1203 (2015).
    • (2015) Nucleic Acids Res. , vol.43 , pp. 1189-1203
    • Cuellar, T.L.1
  • 86
    • 27744590896 scopus 로고    scopus 로고
    • Passenger-strand cleavage facilitates assembly of siRNA into Ago2-containing RNAi enzyme complexes
    • Matranga, C., Tomari, Y., Shin, C., Bartel, D. P., Zamore, P. D. Passenger-strand cleavage facilitates assembly of siRNA into Ago2-containing RNAi enzyme complexes. Cell 123, 607-620 (2005).
    • (2005) Cell , vol.123 , pp. 607-620
    • Matranga, C.1    Tomari, Y.2    Shin, C.3    Bartel, D.P.4    Zamore, P.D.5
  • 88
    • 78651281223 scopus 로고    scopus 로고
    • Modulation of thermal stability can enhance the potency of siRNA
    • Addepalli, H., et al. Modulation of thermal stability can enhance the potency of siRNA. Nucleic Acids Res. 38, 7320-7331 (2010).
    • (2010) Nucleic Acids Res. , vol.38 , pp. 7320-7331
    • Addepalli, H.1
  • 90
    • 0142165224 scopus 로고    scopus 로고
    • Functional siRNAs and miRNAs exhibit strand bias
    • Khvorova, A., Reynolds, A., Jayasena, S. D. Functional siRNAs and miRNAs exhibit strand bias. Cell 115, 209-216 (2003).
    • (2003) Cell , vol.115 , pp. 209-216
    • Khvorova, A.1    Reynolds, A.2    Jayasena, S.D.3
  • 91
    • 84934293424 scopus 로고    scopus 로고
    • Single-molecule imaging reveals that Argonaute reshapes the binding properties of its nucleic acid guides
    • Salomon, W. E., Jolly, S. M., Moore, M. J., Zamore, P. D., Serebrov, V. Single-molecule imaging reveals that Argonaute reshapes the binding properties of its nucleic acid guides. Cell 162, 84-95 (2015).
    • (2015) Cell , vol.162 , pp. 84-95
    • Salomon, W.E.1    Jolly, S.M.2    Moore, M.J.3    Zamore, P.D.4    Serebrov, V.5
  • 92
    • 84979265998 scopus 로고    scopus 로고
    • Structural analysis of human Argonaute-2 bound to a modified siRNA guide
    • Schirle, N. T., et al. Structural analysis of human Argonaute-2 bound to a modified siRNA guide. J. Am. Chem. Soc. 138, 8694-8697 (2016).
    • (2016) J. Am. Chem. Soc. , vol.138 , pp. 8694-8697
    • Schirle, N.T.1
  • 93
    • 84961779012 scopus 로고    scopus 로고
    • Structural basis of duplex thermodynamic stability and enhanced nuclease resistance of 5-C-methyl pyrimidine-modified oligonucleotides
    • Kel'in, A. V., et al. Structural basis of duplex thermodynamic stability and enhanced nuclease resistance of 5-C-methyl pyrimidine-modified oligonucleotides. J. Org. Chem. 81, 2261-2279 (2016).
    • (2016) J. Org. Chem. , vol.81 , pp. 2261-2279
    • Kel'In, A.V.1
  • 94
    • 0037031576 scopus 로고    scopus 로고
    • Single-stranded antisense siRNAs guide target RNA cleavage in RNAi
    • Martinez, J., Patkaniowska, A., Urlaub, H., Lührmann, R., Tuschl, T. Single-stranded antisense siRNAs guide target RNA cleavage in RNAi. Cell 110, 563-574 (2002).
    • (2002) Cell , vol.110 , pp. 563-574
    • Martinez, J.1    Patkaniowska, A.2    Urlaub, H.3    Lührmann, R.4    Tuschl, T.5
  • 95
    • 77953479619 scopus 로고    scopus 로고
    • Structural basis for 5-nucleotide base-specific recognition of guide RNA by human AGO2
    • Frank, F., Sonenberg, N., Nagar, B. Structural basis for 5-nucleotide base-specific recognition of guide RNA by human AGO2. Nature 465, 818-822 (2010).
    • (2010) Nature , vol.465 , pp. 818-822
    • Frank, F.1    Sonenberg, N.2    Nagar, B.3
  • 96
    • 15844371219 scopus 로고    scopus 로고
    • Structural basis for 5-end-specific recognition of guide RNA by the A. Fulgidus Piwi protein
    • Ma, J. B., et al. Structural basis for 5-end-specific recognition of guide RNA by the A. fulgidus Piwi protein. Nature 434, 666-670 (2005).
    • (2005) Nature , vol.434 , pp. 666-670
    • Ma, J.B.1
  • 97
    • 34248563036 scopus 로고    scopus 로고
    • The human RNA kinase hClp1 is active on 3 transfer RNA exons and short interfering RNAs
    • Weitzer, S., Martinez, J. The human RNA kinase hClp1 is active on 3 transfer RNA exons and short interfering RNAs. Nature 447, 222-226 (2007).
    • (2007) Nature , vol.447 , pp. 222-226
    • Weitzer, S.1    Martinez, J.2
  • 98
    • 38649099969 scopus 로고    scopus 로고
    • Strand-specific 5-O-methylation of siRNA duplexes controls guide strand selection and targeting specificity
    • Chen, P. Y., et al. Strand-specific 5-O-methylation of siRNA duplexes controls guide strand selection and targeting specificity. RNA 14, 263-274 (2008).
    • (2008) RNA , vol.14 , pp. 263-274
    • Chen, P.Y.1
  • 99
    • 84868369592 scopus 로고    scopus 로고
    • SiRNA-optimized modifications for enhanced in vivo activity
    • Kenski, D. M., et al. siRNA-optimized modifications for enhanced in vivo activity. Mol. Ther. Nucleic Acids 1, e5 (2012).
    • (2012) Mol. Ther. Nucleic Acids , vol.1 , pp. e5
    • Kenski, D.M.1
  • 100
    • 84964582919 scopus 로고    scopus 로고
    • 5-(E)-vinylphosphonate: A stable phosphate mimic can improve the RNAi Activity of siRNA-GalNAc conjugates
    • Parmar, R., et al. 5-(E)-vinylphosphonate: a stable phosphate mimic can improve the RNAi Activity of siRNA-GalNAc conjugates. ChemBioChem 17, 985-989 (2016).
    • (2016) ChemBioChem , vol.17 , pp. 985-989
    • Parmar, R.1
  • 101
    • 84865688581 scopus 로고    scopus 로고
    • Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression
    • Yu, D., et al. Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. Cell 150, 895-908 (2012).
    • (2012) Cell , vol.150 , pp. 895-908
    • Yu, D.1
  • 102
    • 84865682650 scopus 로고    scopus 로고
    • Single-stranded siRNAs activate RNAi in animals
    • Lima, W. F., et al. Single-stranded siRNAs activate RNAi in animals. Cell 150, 883-894 (2012).
    • (2012) Cell , vol.150 , pp. 883-894
    • Lima, W.F.1
  • 103
    • 84942279838 scopus 로고    scopus 로고
    • Identification of metabolically stable 5-phospha 5(-phosphate analogs that support single-stranded siRNA activity
    • Prakash, T. P., et al. Identification of metabolically stable 5-phospha 5(-phosphate analogs that support single-stranded siRNA activity. Nucleic Acids Res. 43, 2993-3011 (2015).
    • (2015) Nucleic Acids Res. , vol.43 , pp. 2993-3011
    • Prakash, T.P.1
  • 104
    • 77951132901 scopus 로고    scopus 로고
    • Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles
    • Davis, M. E., et al. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature 464, 1067-1070 (2010).
    • (2010) Nature , vol.464 , pp. 1067-1070
    • Davis, M.E.1
  • 105
    • 84883118140 scopus 로고    scopus 로고
    • Safety and efficacy of RNAi therapy for transthyretin amyloidosis
    • Coelho, T., et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N. Engl. J. Med. 369, 819-829 (2013).
    • (2013) N. Engl. J. Med. , vol.369 , pp. 819-829
    • Coelho, T.1
  • 106
    • 84979914402 scopus 로고    scopus 로고
    • The delivery of therapeutic oligonucleotides
    • Juliano, R. L. The delivery of therapeutic oligonucleotides. Nucleic Acids Res. 44, 6518-6548 (2016).
    • (2016) Nucleic Acids Res. , vol.44 , pp. 6518-6548
    • Juliano, R.L.1
  • 107
    • 84918578407 scopus 로고    scopus 로고
    • Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing
    • Nair, J. K., et al. Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing. J. Am. Chem. Soc. 136, 16958-16961 (2014).
    • (2014) J. Am. Chem. Soc. , vol.136 , pp. 16958-16961
    • Nair, J.K.1
  • 108
    • 84964940266 scopus 로고    scopus 로고
    • Synergistic effect of phosphorothioate, 5-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA
    • Prakash, T. P., et al. Synergistic effect of phosphorothioate, 5-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA. Bioorg. Med. Chem. Lett. 26, 2817-2820 (2016).
    • (2016) Bioorg. Med. Chem. Lett. , vol.26 , pp. 2817-2820
    • Prakash, T.P.1
  • 109
    • 84913591370 scopus 로고    scopus 로고
    • Synthesis of multi-galactose-conjugated 2-O-methyl oligoribonucleotides and their in vivo imaging with positron emission tomography
    • Mäkilä, J., et al. Synthesis of multi-galactose-conjugated 2-O-methyl oligoribonucleotides and their in vivo imaging with positron emission tomography. Bioorg. Med. Chem. 22, 6806-6813 (2014).
    • (2014) Bioorg. Med. Chem. , vol.22 , pp. 6806-6813
    • Mäkilä, J.1
  • 110
    • 85008485863 scopus 로고    scopus 로고
    • A highly durable RNAi therapeutic inhibitor of PCSK9
    • Fitzgerald, K., et al. A highly durable RNAi therapeutic inhibitor of PCSK9. N. Engl. J. Med. 376, 41-51 (2017).
    • (2017) N. Engl. J. Med. , vol.376 , pp. 41-51
    • Fitzgerald, K.1
  • 111
    • 84936792294 scopus 로고    scopus 로고
    • 2-fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF
    • Shen, W., Liang, X. H., Sun, H., Crooke, S. T. 2-fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF. Nucleic Acids Res. 43, 4569-4578 (2015).
    • (2015) Nucleic Acids Res. , vol.43 , pp. 4569-4578
    • Shen, W.1    Liang, X.H.2    Sun, H.3    Crooke, S.T.4
  • 112
    • 85011325677 scopus 로고    scopus 로고
    • Impact of oligonucleotide structure, chemistry, delivery method on in vitro cytotoxicity
    • Janas, M. M., et al. Impact of oligonucleotide structure, chemistry, delivery method on in vitro cytotoxicity. Nucleic Acid Ther. http://dx. doi. org/10. 1089/nat. 2016/0639 (2016).
    • (2016) Nucleic Acid Ther.
    • Janas, M.M.1
  • 113
    • 84929574453 scopus 로고    scopus 로고
    • IRNA conjugates carrying sequentially assembled trivalent N-acetylgalactosamine linked through nucleosides elicit robust gene silencing in vivo in hepatocytes
    • Matsuda, S., et al. iRNA conjugates carrying sequentially assembled trivalent N-acetylgalactosamine linked through nucleosides elicit robust gene silencing in vivo in hepatocytes. ACS Chem. Biol. 10, 1181-1187 (2015).
    • (2015) ACS Chem. Biol. , vol.10 , pp. 1181-1187
    • Matsuda, S.1
  • 114
    • 84926320054 scopus 로고    scopus 로고
    • Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-acetylgalactosamine elicits robust gene silencing in vivo
    • Rajeev, K. G., et al. Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-acetylgalactosamine elicits robust gene silencing in vivo. ChemBioChem 16, 903-908 (2015).
    • (2015) ChemBioChem , vol.16 , pp. 903-908
    • Rajeev, K.G.1
  • 115
    • 84937764228 scopus 로고    scopus 로고
    • An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia
    • Sehgal, A., et al. An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia. Nat. Med. 21, 492-497 (2015).
    • (2015) Nat. Med. , vol.21 , pp. 492-497
    • Sehgal, A.1
  • 116
    • 84905584822 scopus 로고    scopus 로고
    • Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice
    • Prakash, T. P., et al. Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice. Nucleic Acids Res. 42, 8796-8807 (2014).
    • (2014) Nucleic Acids Res. , vol.42 , pp. 8796-8807
    • Prakash, T.P.1
  • 117
    • 84965106860 scopus 로고    scopus 로고
    • Disposition and pharmacology of a GalNAc3-conjugated ASO targeting human lipoprotein (a) in mice
    • Yu, R. Z., et al. Disposition and pharmacology of a GalNAc3-conjugated ASO targeting human lipoprotein (a) in mice. Mol. Ther. Nucleic Acids 5, e317 (2016).
    • (2016) Mol. Ther. Nucleic Acids , vol.5 , pp. e317
    • Yu, R.Z.1
  • 118
    • 84994784124 scopus 로고    scopus 로고
    • Disposition and pharmacokinetics of a GalNAc3-conjugated antisense oligonucleotide targeting human lipoprotein (a) in monkeys
    • Yu, R. Z., et al. Disposition and pharmacokinetics of a GalNAc3-conjugated antisense oligonucleotide targeting human lipoprotein (a) in monkeys. Nucleic Acid Ther. 26, 372-380 (2016).
    • (2016) Nucleic Acid Ther. , vol.26 , pp. 372-380
    • Yu, R.Z.1
  • 119
    • 35148815670 scopus 로고    scopus 로고
    • Mechanisms and optimization of in vivo delivery of lipophilic siRNAs
    • Wolfrum, C., et al. Mechanisms and optimization of in vivo delivery of lipophilic siRNAs. Nat. Biotechnol. 25, 1149-1157 (2007).
    • (2007) Nat. Biotechnol. , vol.25 , pp. 1149-1157
    • Wolfrum, C.1
  • 120
    • 84890082930 scopus 로고    scopus 로고
    • Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye
    • Byrne, M., et al. Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye. J. Ocul. Pharmacol. Ther. 29, 855-864 (2013).
    • (2013) J. Ocul. Pharmacol. Ther. , vol.29 , pp. 855-864
    • Byrne, M.1
  • 121
    • 84948734979 scopus 로고    scopus 로고
    • Hydrophobically modified siRNAs silence huntingtin mRNA in primary neurons and mouse brain
    • Alterman, J. F., et al. Hydrophobically modified siRNAs silence huntingtin mRNA in primary neurons and mouse brain. Mol. Ther. Nucleic Acids 4, e266 (2015).
    • (2015) Mol. Ther. Nucleic Acids , vol.4 , pp. e266
    • Alterman, J.F.1
  • 122
    • 85015168960 scopus 로고    scopus 로고
    • Docosahexaenoic acid conjugation enhances distribution and safety of siRNA upon local administration in mouse brain
    • Nikan, M., et al. Docosahexaenoic acid conjugation enhances distribution and safety of siRNA upon local administration in mouse brain. Mol. Ther. Nucleic Acids 5, e344 (2016).
    • (2016) Mol. Ther. Nucleic Acids , vol.5 , pp. e344
    • Nikan, M.1
  • 123
    • 84865442084 scopus 로고    scopus 로고
    • Toxicological and pharmacokinetic properties of chemically modified siRNAs targeting p53 RNA following intravenous administration
    • Thompson, J. D., et al. Toxicological and pharmacokinetic properties of chemically modified siRNAs targeting p53 RNA following intravenous administration. Nucleic Acid Ther. 22, 255-264 (2012).
    • (2012) Nucleic Acid Ther. , vol.22 , pp. 255-264
    • Thompson, J.D.1
  • 124
    • 65249087358 scopus 로고    scopus 로고
    • G3139, an anti-Bcl-2 antisense oligomer that binds heparin-binding growth factors and collagen I, alters in vitro endothelial cell growth and tubular morphogenesis
    • Stein, C. A., et al. G3139, an anti-Bcl-2 antisense oligomer that binds heparin-binding growth factors and collagen I, alters in vitro endothelial cell growth and tubular morphogenesis. Clin. Cancer Res. 15, 2797-2807 (2009).
    • (2009) Clin. Cancer Res. , vol.15 , pp. 2797-2807
    • Stein, C.A.1
  • 125
    • 85014793731 scopus 로고    scopus 로고
    • Oligonucleotide therapies for disorders of the nervous system
    • Khorkova, O., Wahlestedt, C. Oligonucleotide therapies for disorders of the nervous system. Nat. Biotechnol. 35, 250-264 (2017).
    • (2017) Nat. Biotechnol. , vol.35 , pp. 250-264
    • Khorkova, O.1    Wahlestedt, C.2
  • 126
    • 84966430344 scopus 로고    scopus 로고
    • Cellular localization of long non-coding RNAs affects silencing by RNAi more than by antisense oligonucleotides
    • Lennox, K. A., Behlke, M. A. Cellular localization of long non-coding RNAs affects silencing by RNAi more than by antisense oligonucleotides. Nucleic Acids Res. 44, 863-877 (2016).
    • (2016) Nucleic Acids Res. , vol.44 , pp. 863-877
    • Lennox, K.A.1    Behlke, M.A.2
  • 127
    • 78649379362 scopus 로고    scopus 로고
    • Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat
    • Gagnon, K. T., et al. Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat. Biochemistry 49, 10166-10178 (2010).
    • (2010) Biochemistry , vol.49 , pp. 10166-10178
    • Gagnon, K.T.1
  • 128
    • 84876416274 scopus 로고    scopus 로고
    • The rough endoplasmatic reticulum is a central nucleation site of siRNA-mediated RNA silencing
    • Stalder, L., et al. The rough endoplasmatic reticulum is a central nucleation site of siRNA-mediated RNA silencing. EMBO J. 32, 1115-1127 (2013).
    • (2013) EMBO J. , vol.32 , pp. 1115-1127
    • Stalder, L.1
  • 129
    • 78649683035 scopus 로고    scopus 로고
    • Quantitative evaluation of siRNA delivery in vivo
    • Pei, Y., et al. Quantitative evaluation of siRNA delivery in vivo. RNA 16, 2553-2563 (2010).
    • (2010) RNA , vol.16 , pp. 2553-2563
    • Pei, Y.1
  • 130
    • 49449117338 scopus 로고    scopus 로고
    • Antisense transcripts are targets for activating small RNAs
    • Schwartz, J. C., et al. Antisense transcripts are targets for activating small RNAs. Nat. Struct. Mol. Biol. 15, 842-848 (2008).
    • (2008) Nat. Struct. Mol. Biol. , vol.15 , pp. 842-848
    • Schwartz, J.C.1
  • 131
    • 84860353437 scopus 로고    scopus 로고
    • Inhibition of natural antisense transcripts in vivo results in gene-specific transcriptional upregulation
    • Modarresi, F., et al. Inhibition of natural antisense transcripts in vivo results in gene-specific transcriptional upregulation. Nat. Biotechnol. 30, 453-459 (2012).
    • (2012) Nat. Biotechnol. , vol.30 , pp. 453-459
    • Modarresi, F.1
  • 132
  • 133
    • 84956900108 scopus 로고    scopus 로고
    • Activating frataxin expression by repeat-targeted nucleic acids
    • Li, L., Matsui, M., Corey, D. R. Activating frataxin expression by repeat-targeted nucleic acids. Nat. Commun. 7, 10606 (2016).
    • (2016) Nat. Commun. , vol.7 , pp. 10606
    • Li, L.1    Matsui, M.2    Corey, D.R.3
  • 134
    • 84957563051 scopus 로고    scopus 로고
    • Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay
    • Nomakuchi, T. T., Rigo, F., Aznarez, I., Krainer, A. R. Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay. Nat. Biotechnol. 34, 164-166 (2016).
    • (2016) Nat. Biotechnol. , vol.34 , pp. 164-166
    • Nomakuchi, T.T.1    Rigo, F.2    Aznarez, I.3    Krainer, A.R.4
  • 135
    • 84981294965 scopus 로고    scopus 로고
    • Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames
    • Liang, X. H., et al. Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames. Nat. Biotechnol. 34, 875-880 (2016).
    • (2016) Nat. Biotechnol. , vol.34 , pp. 875-880
    • Liang, X.H.1
  • 136
    • 84877244539 scopus 로고    scopus 로고
    • Biotech comes to its 'antisenses' after hard-won drug approval
    • 19 February 2013
    • Jiang, K. Biotech comes to its 'antisenses' after hard-won drug approval. Spoonful of Medicine http://blogs. nature. com/spoonful/2013/02/biotech-comes-to-its-antisenses-after-hard-won-drug-approval. html (19 February 2013).
    • Spoonful of Medicine
    • Jiang, K.1
  • 137
    • 85014850900 scopus 로고    scopus 로고
    • Recommended international nonproprietary names for pharmaceutical substances (INN): List 76
    • World Health Organzation.
    • World Health Organzation. Recommended international nonproprietary names for pharmaceutical substances (INN): list 76. WHO Drug Information 30, 477-544 (2016).
    • (2016) WHO Drug Information , vol.30 , pp. 477-544
  • 138
    • 85008330554 scopus 로고    scopus 로고
    • Oligonucleotide therapeutics-A new class of cholesterol-lowering drugs
    • Khvorova, A. Oligonucleotide therapeutics-a new class of cholesterol-lowering drugs. N. Engl. J. Med. 376, 4-7 (2017).
    • (2017) N. Engl. J. Med. , vol.376 , pp. 4-7
    • Khvorova, A.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.