메뉴 건너뛰기




Volumn , Issue , 2016, Pages 659-672

Therapeutic nucleic acids: current clinical status

Author keywords

antisense oligonucleotides; aptamers; gene therapy; ribozymes; RNA decoys

Indexed keywords

ALIPOGENE TIPARVOVEC; ANTISENSE OLIGONUCLEOTIDE; APTAMER; CIRCULAR RNA; DNA; FOMIVIRSEN; GENDICINE; MICRORNA; MIPOMERSEN; MIRAVIRSEN; NUCLEIC ACID; RIBOZYME; RNA; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

EID: 84982933442     PISSN: 03065251     EISSN: 13652125     Source Type: Journal    
DOI: 10.1111/bcp.12987     Document Type: Review
Times cited : (182)

References (112)
  • 1
    • 84909580167 scopus 로고    scopus 로고
    • The expanding view of RNA and DNA function
    • Breaker RR, Joyce GF. The expanding view of RNA and DNA function. Chem Biol 2014; 21: 1059–65.
    • (2014) Chem Biol , vol.21 , pp. 1059-1065
    • Breaker, R.R.1    Joyce, G.F.2
  • 2
    • 79959810780 scopus 로고    scopus 로고
    • Chemistry of nucleic acids: impacts in multiple fields
    • Khakshoor O, Kool ET. Chemistry of nucleic acids: impacts in multiple fields. Chem Commun 2011; 47: 7018–24.
    • (2011) Chem Commun , vol.47 , pp. 7018-7024
    • Khakshoor, O.1    Kool, E.T.2
  • 3
    • 0036636074 scopus 로고    scopus 로고
    • Nucleic-acid therapeutics: basic principles and recent applications
    • Opalinska JB, Gewirtz AM. Nucleic-acid therapeutics: basic principles and recent applications. Nat Rev Drug Discov 2002; 1: 503–14.
    • (2002) Nat Rev Drug Discov , vol.1 , pp. 503-514
    • Opalinska, J.B.1    Gewirtz, A.M.2
  • 4
    • 84953439628 scopus 로고    scopus 로고
    • Critical considerations for developing nucleic acid macromolecule based drug products
    • Muralidhara BK, Baid R, Bishop SM, Huang M, Wang W, Nema S. Critical considerations for developing nucleic acid macromolecule based drug products. Drug Discov Today 2016; 21: 430–44.
    • (2016) Drug Discov Today , vol.21 , pp. 430-444
    • Muralidhara, B.K.1    Baid, R.2    Bishop, S.M.3    Huang, M.4    Wang, W.5    Nema, S.6
  • 5
    • 85028832423 scopus 로고    scopus 로고
    • Nanodiamonds: the intersection of nanotechnology, drug development and personalized medicine
    • Ho D, Wang C-H K, Chow EK-H. Nanodiamonds: the intersection of nanotechnology, drug development and personalized medicine. Sci Adv 2015; 1: e1500439.
    • (2015) Sci Adv , vol.1
    • Ho, D.1    Wang C-H, K.2    Chow, E.K.-H.3
  • 6
    • 0034945428 scopus 로고    scopus 로고
    • Antisense oligonucleotides in cutaneous therapy
    • Wraight CJ, White PJ. Antisense oligonucleotides in cutaneous therapy. Pharmacol Ther 2001; 90: 89–104.
    • (2001) Pharmacol Ther , vol.90 , pp. 89-104
    • Wraight, C.J.1    White, P.J.2
  • 7
    • 79958706454 scopus 로고    scopus 로고
    • Gene therapy: nucleic acids as drugs. Action, mechanisms and delivery into the cell
    • Cavagnari BM. Gene therapy: nucleic acids as drugs. Action, mechanisms and delivery into the cell. Arch Argent Pediatr 2011; 109: 237–44.
    • (2011) Arch Argent Pediatr , vol.109 , pp. 237-244
    • Cavagnari, B.M.1
  • 8
    • 84935005894 scopus 로고    scopus 로고
    • Short-interference RNAs: becoming medicines
    • Martinez T, Jimenez AI, Paneda C. Short-interference RNAs: becoming medicines. EXCLI J 2015; 14: 714–46.
    • (2015) EXCLI J , vol.14 , pp. 714-746
    • Martinez, T.1    Jimenez, A.I.2    Paneda, C.3
  • 9
    • 84941903146 scopus 로고    scopus 로고
    • Overview of the regulatory oversight implemented by the French regulatory authorities for the clinical investigation of gene therapy and cell therapy products
    • Lucas-Samuel S, Ferry N, Trouvin JH. Overview of the regulatory oversight implemented by the French regulatory authorities for the clinical investigation of gene therapy and cell therapy products. Adv Exp Med Biol 2015; 871: 73–85.
    • (2015) Adv Exp Med Biol , vol.871 , pp. 73-85
    • Lucas-Samuel, S.1    Ferry, N.2    Trouvin, J.H.3
  • 10
    • 84856431819 scopus 로고    scopus 로고
    • RNA therapeutics: beyond RNA interference and antisense oligonucleotides
    • Kole R, Krainer AR, Altman S. RNA therapeutics: beyond RNA interference and antisense oligonucleotides. Nat Rev Drug Discov 2012; 11: 125–40.
    • (2012) Nat Rev Drug Discov , vol.11 , pp. 125-140
    • Kole, R.1    Krainer, A.R.2    Altman, S.3
  • 11
    • 11844254846 scopus 로고    scopus 로고
    • Secondary structure prediction of interacting RNA molecules
    • Andronescu M, Zhang ZC, Condon A. Secondary structure prediction of interacting RNA molecules. J Mol Biol 2005; 345: 987–1001.
    • (2005) J Mol Biol , vol.345 , pp. 987-1001
    • Andronescu, M.1    Zhang, Z.C.2    Condon, A.3
  • 13
    • 80052228547 scopus 로고    scopus 로고
    • Nucleic acid aptamers: clinical applications and promising new horizons
    • Ni X, Castanares M, Mukherjee A, Lupold SE. Nucleic acid aptamers: clinical applications and promising new horizons. Curr Med Chem 2011; 18: 4206–14.
    • (2011) Curr Med Chem , vol.18 , pp. 4206-4214
    • Ni, X.1    Castanares, M.2    Mukherjee, A.3    Lupold, S.E.4
  • 14
    • 84927717726 scopus 로고    scopus 로고
    • Aptamer selection technology and recent advances
    • Blind M, Blank M. Aptamer selection technology and recent advances. Mol Ther Nucleic Acids 2015; 4: e223.
    • (2015) Mol Ther Nucleic Acids , vol.4
    • Blind, M.1    Blank, M.2
  • 16
  • 17
    • 84904111483 scopus 로고    scopus 로고
    • Nucleic acid aptamers: research tools in disease diagnostics and therapeutics
    • Santosh B, Yadava PK. Nucleic acid aptamers: research tools in disease diagnostics and therapeutics. BioMed Res Int 2014; 2014: 540451.
    • (2014) BioMed Res Int , vol.2014 , pp. 540451
    • Santosh, B.1    Yadava, P.K.2
  • 18
    • 84929302116 scopus 로고    scopus 로고
    • Nucleic acid aptamers as potential therapeutic and diagnostic agents for lymphoma
    • Shum KT, Zhou J, Rossi JJ. Nucleic acid aptamers as potential therapeutic and diagnostic agents for lymphoma. J Cancer Ther 2013; 4: 872–90.
    • (2013) J Cancer Ther , vol.4 , pp. 872-890
    • Shum, K.T.1    Zhou, J.2    Rossi, J.J.3
  • 19
    • 33846914204 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: RNA aptamers
    • Que-Gewirth NS, Sullenger BA. Gene therapy progress and prospects: RNA aptamers. Gene Ther 2007; 14: 283–91.
    • (2007) Gene Ther , vol.14 , pp. 283-291
    • Que-Gewirth, N.S.1    Sullenger, B.A.2
  • 20
    • 33745211255 scopus 로고    scopus 로고
    • Antisense oligonucleotides: from design to therapeutic application
    • Chan JHP, Lim S, Wong WSF. Antisense oligonucleotides: from design to therapeutic application. Clin Exp Pharmacol Physiol 2006; 33: 533–40.
    • (2006) Clin Exp Pharmacol Physiol , vol.33 , pp. 533-540
    • Chan, J.H.P.1    Lim, S.2    Wong, W.S.F.3
  • 22
    • 84924538557 scopus 로고    scopus 로고
    • Synthesis, biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages
    • Wan WB, Migawa MT, Vasquez G, Murray HM, Nichols JG, Gaus H, et al. Synthesis, biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages. Nucleic Acids Res 2014; 42: 13456–68.
    • (2014) Nucleic Acids Res , vol.42 , pp. 13456-13468
    • Wan, W.B.1    Migawa, M.T.2    Vasquez, G.3    Murray, H.M.4    Nichols, J.G.5    Gaus, H.6
  • 23
    • 77949512140 scopus 로고    scopus 로고
    • RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform
    • Bennett CF, Swayze EE. RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol 2010; 50: 259–93.
    • (2010) Annu Rev Pharmacol Toxicol , vol.50 , pp. 259-293
    • Bennett, C.F.1    Swayze, E.E.2
  • 24
    • 67649271530 scopus 로고    scopus 로고
    • Antisense oligonucleotide pharmacokinetics and metabolism
    • Geary RS. Antisense oligonucleotide pharmacokinetics and metabolism. Expert Opin Drug Metab Toxicol 2009; 5: 381–91.
    • (2009) Expert Opin Drug Metab Toxicol , vol.5 , pp. 381-391
    • Geary, R.S.1
  • 25
    • 84937252483 scopus 로고    scopus 로고
    • Antisense oligonucleotides in therapy for neurodegenerative disorders
    • Evers MM, Toonen LJA, van Roon-Mom WMC. Antisense oligonucleotides in therapy for neurodegenerative disorders. Adv Drug Deliv Rev 2015; 87: 90–103.
    • (2015) Adv Drug Deliv Rev , vol.87 , pp. 90-103
    • Evers, M.M.1    Toonen, L.J.A.2    van Roon-Mom, W.M.C.3
  • 26
    • 84872432062 scopus 로고    scopus 로고
    • Clinical pharmacokinetics of second generation antisense oligonucleotides
    • Yu RZ, Grundy JS, Geary RS. Clinical pharmacokinetics of second generation antisense oligonucleotides. Expert Opin Drug Metab Toxicol 2013; 9: 169–82.
    • (2013) Expert Opin Drug Metab Toxicol , vol.9 , pp. 169-182
    • Yu, R.Z.1    Grundy, J.S.2    Geary, R.S.3
  • 27
    • 67349213986 scopus 로고    scopus 로고
    • Advances in antisense oligonucleotide development for target identification, validation, and as novel therapeutics
    • Mansoor M, Melendez AJ. Advances in antisense oligonucleotide development for target identification, validation, and as novel therapeutics. Gene Regul Syst Bio 2008; 2: 275–95.
    • (2008) Gene Regul Syst Bio , vol.2 , pp. 275-295
    • Mansoor, M.1    Melendez, A.J.2
  • 28
    • 84988044461 scopus 로고    scopus 로고
    • Antisense approval provides boost to the field
    • Tse MT. Antisense approval provides boost to the field. Drug Discov 2013; 12: 179.
    • (2013) Drug Discov , vol.12 , pp. 179
    • Tse, M.T.1
  • 30
    • 84894047761 scopus 로고    scopus 로고
    • Mipomersen (Kynamro). A novel antisense oligonucleotide inhibitor for the management of homozygous familial hypercholesterolemia
    • Wong E, Goldberg T. Mipomersen (Kynamro). A novel antisense oligonucleotide inhibitor for the management of homozygous familial hypercholesterolemia. Pharm Therapeut 2014; 39: 119–22.
    • (2014) Pharm Therapeut , vol.39 , pp. 119-122
    • Wong, E.1    Goldberg, T.2
  • 31
    • 0034945407 scopus 로고    scopus 로고
    • Technology evaluation: fomivirsen, Isis Pharmaceuticals Inc./CIBA vision
    • Orr RM. Technology evaluation: fomivirsen, Isis Pharmaceuticals Inc./CIBA vision. Curr Opin Mol Ther 2001; 3: 288–94.
    • (2001) Curr Opin Mol Ther , vol.3 , pp. 288-294
    • Orr, R.M.1
  • 32
  • 33
    • 0036094531 scopus 로고    scopus 로고
    • Fomivirsen: clinical pharmacology and potential drug interactions
    • Geary RS, Henry SP, Grillone LR. Fomivirsen: clinical pharmacology and potential drug interactions. Clin Pharmacokinet 2002; 41: 255–60.
    • (2002) Clin Pharmacokinet , vol.41 , pp. 255-260
    • Geary, R.S.1    Henry, S.P.2    Grillone, L.R.3
  • 34
    • 0036211366 scopus 로고    scopus 로고
    • Randomized dose-comparison studies of intravitreous fomivirsen for treatment of cytomegalovirus retinitis that has reactivated or is persistently active despite other therapies in patients with AIDS
    • The Vitravene study group. Randomized dose-comparison studies of intravitreous fomivirsen for treatment of cytomegalovirus retinitis that has reactivated or is persistently active despite other therapies in patients with AIDS. Am J Ophthalmol 2002; 133: 475–83.
    • (2002) Am J Ophthalmol , vol.133 , pp. 475-483
  • 35
    • 0036202847 scopus 로고    scopus 로고
    • Safety of intravitreous fomivirsen for treatment of cytomegalovirus retinitis in patients with AIDS
    • Vitravene Study Group 2. Safety of intravitreous fomivirsen for treatment of cytomegalovirus retinitis in patients with AIDS. Am J Ophthalmol 2002; 133: 484–98.
    • (2002) Am J Ophthalmol , vol.133 , pp. 484-498
  • 36
    • 0033825806 scopus 로고    scopus 로고
    • Reversible bull's-eye maculopathy associated with intravitreal fomivirsen therapy for cytomegalovirus retinitis
    • Stone TW, Jaffe GJ. Reversible bull's-eye maculopathy associated with intravitreal fomivirsen therapy for cytomegalovirus retinitis. Am J Ophthalmol 2000; 130: 242–3.
    • (2000) Am J Ophthalmol , vol.130 , pp. 242-243
    • Stone, T.W.1    Jaffe, G.J.2
  • 37
    • 33750207033 scopus 로고    scopus 로고
    • Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B
    • Kastelein JJ, Wedel MK, Baker BF, Su J, Bradley JD, Yu RZ, et al. Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B. Circulation 2006; 114: 1729–35.
    • (2006) Circulation , vol.114 , pp. 1729-1735
    • Kastelein, J.J.1    Wedel, M.K.2    Baker, B.F.3    Su, J.4    Bradley, J.D.5    Yu, R.Z.6
  • 38
    • 79956267846 scopus 로고    scopus 로고
    • Familial hypercholesterolemia: screening, diagnosis and management of pediatric and adult patients: clinical guidance from the National Lipid Association Expert Panel on Familial Hypercholesterolemia
    • Goldberg AC, Hopkins PN, Toth PP, Ballantyne CM, Rader DJ, Robinson JG, et al. Familial hypercholesterolemia: screening, diagnosis and management of pediatric and adult patients: clinical guidance from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Clin Lipidol 2011; 5: 133–40.
    • (2011) J Clin Lipidol , vol.5 , pp. 133-140
    • Goldberg, A.C.1    Hopkins, P.N.2    Toth, P.P.3    Ballantyne, C.M.4    Rader, D.J.5    Robinson, J.G.6
  • 39
    • 77949485460 scopus 로고    scopus 로고
    • Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolemia: a randomised, double-blind, placebo-controlled trial
    • Raal FJ, Santos RD, Blom DJ, Marais AD, Charng MJ, Cromwell WC, et al. Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolemia: a randomised, double-blind, placebo-controlled trial. Lancet 2010; 375: 998–1006.
    • (2010) Lancet , vol.375 , pp. 998-1006
    • Raal, F.J.1    Santos, R.D.2    Blom, D.J.3    Marais, A.D.4    Charng, M.J.5    Cromwell, W.C.6
  • 40
    • 84869039132 scopus 로고    scopus 로고
    • Randomized, placebo-controlled trial of mipomersen in patients with severe hypercholesterolemia receiving maximally tolerated lipid-lowering therapy
    • McGowan MP, Tardif JC, Ceska R, Burgess LJ, Soran H, Gouni-Berthold I, et al. Randomized, placebo-controlled trial of mipomersen in patients with severe hypercholesterolemia receiving maximally tolerated lipid-lowering therapy. PLoS One 2012; 7: 133–40.
    • (2012) PLoS One , vol.7 , pp. 133-140
    • McGowan, M.P.1    Tardif, J.C.2    Ceska, R.3    Burgess, L.J.4    Soran, H.5    Gouni-Berthold, I.6
  • 41
    • 84868518061 scopus 로고    scopus 로고
    • Apolipoprotein B synthesis inhibition with mipomersen in heterozygous familial hypercholesterolemia: results of a randomized, double-blind, placebo-controlled trial to assess efficacy and safety as add-on therapy in patients with coronary artery disease
    • Stein EA, Dufour R, Gagne C, Gaudet D, East C, Donovan JM, et al. Apolipoprotein B synthesis inhibition with mipomersen in heterozygous familial hypercholesterolemia: results of a randomized, double-blind, placebo-controlled trial to assess efficacy and safety as add-on therapy in patients with coronary artery disease. Circulation 2012; 126: 2283–92.
    • (2012) Circulation , vol.126 , pp. 2283-2292
    • Stein, E.A.1    Dufour, R.2    Gagne, C.3    Gaudet, D.4    East, C.5    Donovan, J.M.6
  • 43
    • 17644401347 scopus 로고    scopus 로고
    • Cross-linked small polyethylenimines: while still nontoxic, deliver DNA efficiently to mammalian cells in vitro and in vivo
    • Thomas M, Ge Q, Lu JJ, Chen J, Klibanov AM. Cross-linked small polyethylenimines: while still nontoxic, deliver DNA efficiently to mammalian cells in vitro and in vivo. Pharm Res 2005; 22: 373–80.
    • (2005) Pharm Res , vol.22 , pp. 373-380
    • Thomas, M.1    Ge, Q.2    Lu, J.J.3    Chen, J.4    Klibanov, A.M.5
  • 45
    • 84868371203 scopus 로고    scopus 로고
    • Oligonucleotide delivery to the lung: waiting to inhale
    • Kjems J, Howard KA. Oligonucleotide delivery to the lung: waiting to inhale. Mol Ther Nucleic Acids 2012; 1: e1.
    • (2012) Mol Ther Nucleic Acids , vol.1
    • Kjems, J.1    Howard, K.A.2
  • 47
    • 84897018235 scopus 로고    scopus 로고
    • Human gene therapy: a brief overview of the genetic revolution
    • Misra S. Human gene therapy: a brief overview of the genetic revolution. JAPI 2013; 61: 127–33.
    • (2013) JAPI , vol.61 , pp. 127-133
    • Misra, S.1
  • 48
    • 3042786281 scopus 로고    scopus 로고
    • Promoters and control elements: designing expression cassettes for gene therapy
    • Papadakis ED, Nicklin SA, Baker AH, White SJ. Promoters and control elements: designing expression cassettes for gene therapy. Curr Gene Ther 2004; 4: 89–113.
    • (2004) Curr Gene Ther , vol.4 , pp. 89-113
    • Papadakis, E.D.1    Nicklin, S.A.2    Baker, A.H.3    White, S.J.4
  • 49
    • 84875341955 scopus 로고    scopus 로고
    • Viral and nonviral delivery systems for gene delivery
    • Nayerossadat N, Maedeh T, Ali PA. Viral and nonviral delivery systems for gene delivery. Adv Biomed Res 2012; 1: 27.
    • (2012) Adv Biomed Res , vol.1 , pp. 27
    • Nayerossadat, N.1    Maedeh, T.2    Ali, P.A.3
  • 50
    • 33846202524 scopus 로고    scopus 로고
    • Gene therapy: human gemline genetics modifications – assessing the scientific, socioethical, and religious issues
    • Mathews QL, Curiel DT. Gene therapy: human gemline genetics modifications – assessing the scientific, socioethical, and religious issues. South Med J 2007; 100: 98–100.
    • (2007) South Med J , vol.100 , pp. 98-100
    • Mathews, Q.L.1    Curiel, D.T.2
  • 51
    • 0035967623 scopus 로고    scopus 로고
    • Death but one unintended consequence of gene therapy trial
    • Sibbald B. Death but one unintended consequence of gene therapy trial. CMAJ 2001; 164: 1612.
    • (2001) CMAJ , vol.164 , pp. 1612
    • Sibbald, B.1
  • 53
    • 24944454988 scopus 로고    scopus 로고
    • Current status of Gendicine in China: recombinant human Ad-p53 agent for treatment of cancers
    • Peng H. Current status of Gendicine in China: recombinant human Ad-p53 agent for treatment of cancers. Hum Gene Ther 2005; 16: 1016–27.
    • (2005) Hum Gene Ther , vol.16 , pp. 1016-1027
    • Peng, H.1
  • 54
    • 33644928077 scopus 로고    scopus 로고
    • China's gene therapy drug
    • Krimsky S. China's gene therapy drug. Genewatch 2005; 18: 10–3.
    • (2005) Genewatch , vol.18 , pp. 10-13
    • Krimsky, S.1
  • 55
    • 84901643074 scopus 로고    scopus 로고
    • Recombinant adenovirus-p53 (Gencidine) sensitizes a pancreatic carcinoma cell line to radiation
    • Li J, Pan J, Zhu X, Su Y, Bao L, Qiu S, et al. Recombinant adenovirus-p53 (Gencidine) sensitizes a pancreatic carcinoma cell line to radiation. Chin J Can Res 2013; 25: 715–21.
    • (2013) Chin J Can Res , vol.25 , pp. 715-721
    • Li, J.1    Pan, J.2    Zhu, X.3    Su, Y.4    Bao, L.5    Qiu, S.6
  • 56
    • 84866899464 scopus 로고    scopus 로고
    • Endgame: Glybera finally recommended for approval as the first gene therapy drug in the European Union
    • Yla-Herttuala S. Endgame: Glybera finally recommended for approval as the first gene therapy drug in the European Union. Mol Ther 2012; 20: 1831–2.
    • (2012) Mol Ther , vol.20 , pp. 1831-1832
    • Yla-Herttuala, S.1
  • 57
    • 84982897517 scopus 로고    scopus 로고
    • United States Food and Drug Administration. Guidance for industry. Gene therapy for clinical trials – observing subjects for delayed adverse events. Available at: http://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/ucm078719.pdf (last accessed 22 December 2015).
  • 59
    • 84961844230 scopus 로고    scopus 로고
    • Correa S, Dreaden EC, Gu L, Hammond PT. Engineering nanolayered particles for modular drug delivery. J Control Release 2016. doi:10.1016/j.jconrel.2016.01.040.
  • 61
    • 84949033739 scopus 로고    scopus 로고
    • Therapeutic potential of cell penetrating peptides (CPPs) and cationic polymers for chronic hepatitis B
    • Ndeboko B, Lemamy GJ, Nielsen PE, Cova L. Therapeutic potential of cell penetrating peptides (CPPs) and cationic polymers for chronic hepatitis B. Int J Mol Sci 2015; 16: 28230–41.
    • (2015) Int J Mol Sci , vol.16 , pp. 28230-28241
    • Ndeboko, B.1    Lemamy, G.J.2    Nielsen, P.E.3    Cova, L.4
  • 62
    • 84876538553 scopus 로고    scopus 로고
    • The RNA–protein world
    • Altman S. The RNA–protein world. RNA 2013; 19: 589–90.
    • (2013) RNA , vol.19 , pp. 589-590
    • Altman, S.1
  • 64
    • 0032545933 scopus 로고    scopus 로고
    • Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
    • Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998; 391: 806–11.
    • (1998) Nature , vol.391 , pp. 806-811
    • Fire, A.1    Xu, S.2    Montgomery, M.K.3    Kostas, S.A.4    Driver, S.E.5    Mello, C.C.6
  • 65
    • 0035942736 scopus 로고    scopus 로고
    • Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
    • Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001; 411: 494–8.
    • (2001) Nature , vol.411 , pp. 494-498
    • Elbashir, S.M.1    Harborth, J.2    Lendeckel, W.3    Yalcin, A.4    Weber, K.5    Tuschl, T.6
  • 67
    • 84880144292 scopus 로고    scopus 로고
    • Recent developments in nanoparticle-based siRNA delivery for cancer therapy
    • Lee JM, Yoon TJ, Cho YS. Recent developments in nanoparticle-based siRNA delivery for cancer therapy. BioMed Res Int 2013; 2013: 782041.
    • (2013) BioMed Res Int , vol.2013 , pp. 782041
    • Lee, J.M.1    Yoon, T.J.2    Cho, Y.S.3
  • 68
    • 0034123448 scopus 로고    scopus 로고
    • Fas and Fas ligand: strong co-expression in human hepatocytes surrounding hepatocellular carcinoma; can cancer induce suicide in peritumoural cells?
    • Roskams T, Libbrecht L, Van Damme B, Desmet V. Fas and Fas ligand: strong co-expression in human hepatocytes surrounding hepatocellular carcinoma; can cancer induce suicide in peritumoural cells? J Pathol 2000; 191: 150–3.
    • (2000) J Pathol , vol.191 , pp. 150-153
    • Roskams, T.1    Libbrecht, L.2    Van Damme, B.3    Desmet, V.4
  • 69
    • 0037348329 scopus 로고    scopus 로고
    • RNA interference targeting Fas protects mice from fulminant hepatitis
    • Song E, Lee SK, Wang J, Ince N, Ouyang N, Min J, et al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat Med 2003; 9: 347–51.
    • (2003) Nat Med , vol.9 , pp. 347-351
    • Song, E.1    Lee, S.K.2    Wang, J.3    Ince, N.4    Ouyang, N.5    Min, J.6
  • 70
    • 82755161958 scopus 로고    scopus 로고
    • Gene silencing by siRNAs and antisense oligonucleotides in the laboratory and the clinic
    • Watts JK, Corey DR. Gene silencing by siRNAs and antisense oligonucleotides in the laboratory and the clinic. J Pathol 2012; 226: 365–79.
    • (2012) J Pathol , vol.226 , pp. 365-379
    • Watts, J.K.1    Corey, D.R.2
  • 71
    • 38749136602 scopus 로고    scopus 로고
    • Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV)
    • DeVincenzo J, Cehelsky JE, Alvarez R, Elbashir S, Harborth J, Toudjarska I, et al. Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV). Antiviral Res 2008; 77: 225–31.
    • (2008) Antiviral Res , vol.77 , pp. 225-231
    • DeVincenzo, J.1    Cehelsky, J.E.2    Alvarez, R.3    Elbashir, S.4    Harborth, J.5    Toudjarska, I.6
  • 73
    • 79951827479 scopus 로고    scopus 로고
    • RNA interference, RSV and lung transplantation – a promising future for siRNA therapeutics
    • Astor TL. RNA interference, RSV and lung transplantation – a promising future for siRNA therapeutics. Am J Respir Crit Care Med 2011; 183: 427–8.
    • (2011) Am J Respir Crit Care Med , vol.183 , pp. 427-428
    • Astor, T.L.1
  • 74
  • 75
    • 84858379476 scopus 로고    scopus 로고
    • MicroRNAs in stress signaling and human disease
    • Mendell JT, Olson EN. MicroRNAs in stress signaling and human disease. Cell 2012; 148: 1172–87.
    • (2012) Cell , vol.148 , pp. 1172-1187
    • Mendell, J.T.1    Olson, E.N.2
  • 76
    • 84920116302 scopus 로고    scopus 로고
    • In vitro antiviral activity and preclinical and clinical resistance profile of Miravirsen, a novel anti-hepatitis C virus therapeutic targeting the human factor miR-122
    • Ottosen S, Parsley TB, Yang L, Zeh K, van Doorn LJ, van der Veer E, et al. In vitro antiviral activity and preclinical and clinical resistance profile of Miravirsen, a novel anti-hepatitis C virus therapeutic targeting the human factor miR-122. Antimicrob Agents Chemother 2015; 59: 599–608.
    • (2015) Antimicrob Agents Chemother , vol.59 , pp. 599-608
    • Ottosen, S.1    Parsley, T.B.2    Yang, L.3    Zeh, K.4    van Doorn, L.J.5    van der Veer, E.6
  • 77
    • 84858126071 scopus 로고    scopus 로고
    • MicroRNA dysregulation in cancer: diagnostics, monitoring and therapeutics. A comprehensive review
    • Lorio MV, Croce CM. MicroRNA dysregulation in cancer: diagnostics, monitoring and therapeutics. A comprehensive review. EMBO Mol Med 2012; 4: 143–59.
    • (2012) EMBO Mol Med , vol.4 , pp. 143-159
    • Lorio, M.V.1    Croce, C.M.2
  • 78
    • 18744396337 scopus 로고    scopus 로고
    • Frequent deletions and down-regulation of micro- RNA genes miR15 and miR16 at 13q14 in chronic lymphocytic leukemia
    • Calin GA, Dumitru CD, Shimizu M, Bichi R, Zupo S, Noch E, et al. Frequent deletions and down-regulation of micro- RNA genes miR15 and miR16 at 13q14 in chronic lymphocytic leukemia. Proc Natl Acad Sci U S A 2002; 99: 15524–9.
    • (2002) Proc Natl Acad Sci U S A , vol.99 , pp. 15524-15529
    • Calin, G.A.1    Dumitru, C.D.2    Shimizu, M.3    Bichi, R.4    Zupo, S.5    Noch, E.6
  • 80
    • 33646471662 scopus 로고    scopus 로고
    • Pre-B cell proliferation and lymphoblastic leukemia/high-grade lymphoma in E(mu)-miR155 transgenic mice
    • Costinean S, Zanesi N, Pekarsky Y, Tili E, Volinia S, Heerema N, et al. Pre-B cell proliferation and lymphoblastic leukemia/high-grade lymphoma in E(mu)-miR155 transgenic mice. Proc Natl Acad Sci U S A 2006; 103: 7024–9.
    • (2006) Proc Natl Acad Sci U S A , vol.103 , pp. 7024-7029
    • Costinean, S.1    Zanesi, N.2    Pekarsky, Y.3    Tili, E.4    Volinia, S.5    Heerema, N.6
  • 81
    • 84922216394 scopus 로고    scopus 로고
    • A computational approach to identifying gene-microRNA modules in cancer
    • Jin D, Lee H. A computational approach to identifying gene-microRNA modules in cancer. PLoS Comput Biol 2015; 11: e1004042.
    • (2015) PLoS Comput Biol , vol.11
    • Jin, D.1    Lee, H.2
  • 82
    • 84869219338 scopus 로고    scopus 로고
    • miR-34 – a microRNA replacement therapy is headed to the clinic
    • Bader AG. miR-34 – a microRNA replacement therapy is headed to the clinic. Front Genet 2012; 3: 120.
    • (2012) Front Genet , vol.3 , pp. 120
    • Bader, A.G.1
  • 83
    • 84929635695 scopus 로고    scopus 로고
    • The emerging role of miRNAs in combined cancer therapy
    • Yu HW, Cho WC. The emerging role of miRNAs in combined cancer therapy. Expert Opin Biol Ther 2015; 15: 923–5.
    • (2015) Expert Opin Biol Ther , vol.15 , pp. 923-925
    • Yu, H.W.1    Cho, W.C.2
  • 85
    • 46749124698 scopus 로고    scopus 로고
    • Position-dependent function for a tandem microRNA miR-122-binding site located in the hepatitis C virus RNA genome
    • Jopling C, Schütz S, Sarnow P. Position-dependent function for a tandem microRNA miR-122-binding site located in the hepatitis C virus RNA genome. Cell Host Microbe 2008; 4: 77–85.
    • (2008) Cell Host Microbe , vol.4 , pp. 77-85
    • Jopling, C.1    Schütz, S.2    Sarnow, P.3
  • 87
    • 84903717510 scopus 로고    scopus 로고
    • Development of microRNA therapeutics is coming of age
    • Van Rooij E, Kauppinen S. Development of microRNA therapeutics is coming of age. EMBO Mol Med 2014; 6: 851–64.
    • (2014) EMBO Mol Med , vol.6 , pp. 851-864
    • Van Rooij, E.1    Kauppinen, S.2
  • 89
    • 84904440080 scopus 로고    scopus 로고
    • Application of microRNAs in diabetes mellitus
    • Chen H, Lan HY, Roukos DH, Cho WC. Application of microRNAs in diabetes mellitus. J Endocrinol 2014; 222: R1–R10.
    • (2014) J Endocrinol , vol.222 , pp. R1-R10
    • Chen, H.1    Lan, H.Y.2    Roukos, D.H.3    Cho, W.C.4
  • 90
    • 84929301851 scopus 로고    scopus 로고
    • Therapeutic aptamers: developmental potential as anticancer drugs
    • Lee JW, Kim HJ, Heo K. Therapeutic aptamers: developmental potential as anticancer drugs. BMB Rep 2015; 48: 234–7.
    • (2015) BMB Rep , vol.48 , pp. 234-237
    • Lee, J.W.1    Kim, H.J.2    Heo, K.3
  • 91
    • 84876025119 scopus 로고    scopus 로고
    • RNA aptamers and their therapeutic and diagnostic applications
    • Germer K, Leonard M, Zhang X. RNA aptamers and their therapeutic and diagnostic applications. Int J Biochem Mol Biol 2013; 4: 27–40.
    • (2013) Int J Biochem Mol Biol , vol.4 , pp. 27-40
    • Germer, K.1    Leonard, M.2    Zhang, X.3
  • 92
    • 0033168686 scopus 로고    scopus 로고
    • A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34(+) cells from the bone marrow of human immunodeficiency virus-1-infected children
    • Kohn DB, Bauer G, Rice CR, Rothschild JC, Carbonaro DA, Valdez P, et al. A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34(+) cells from the bone marrow of human immunodeficiency virus-1-infected children. Blood 1999; 94: 368–71.
    • (1999) Blood , vol.94 , pp. 368-371
    • Kohn, D.B.1    Bauer, G.2    Rice, C.R.3    Rothschild, J.C.4    Carbonaro, D.A.5    Valdez, P.6
  • 93
    • 33845658622 scopus 로고    scopus 로고
    • Anti-VEGF aptamer (pegaptanib) therapy for ocular vascular diseases
    • Ng EW, Adamis AP. Anti-VEGF aptamer (pegaptanib) therapy for ocular vascular diseases. Ann N Y Acad Sci 2006; 1082: 151–71.
    • (2006) Ann N Y Acad Sci , vol.1082 , pp. 151-171
    • Ng, E.W.1    Adamis, A.P.2
  • 94
    • 79954606458 scopus 로고    scopus 로고
    • CXCL 12 (SDF1 alpha)- CXCR4/CXCR7 pathway inhibition: an emerging sensitizer for anticancer therapies?
    • Duda DG, Kozin V, Kirkpatrick ND, Xu L, Fukumura D, Jain RK. CXCL 12 (SDF1 alpha)- CXCR4/CXCR7 pathway inhibition: an emerging sensitizer for anticancer therapies? Clin Cancer Res 2011; 8: 2074–80.
    • (2011) Clin Cancer Res , vol.8 , pp. 2074-2080
    • Duda, D.G.1    Kozin, V.2    Kirkpatrick, N.D.3    Xu, L.4    Fukumura, D.5    Jain, R.K.6
  • 95
    • 78349279253 scopus 로고    scopus 로고
    • Insulin resistance, inflammation, and obesity: role of monocyte chemoattractant protein-1 (or CCL2) in the regulation of metabolism
    • Rull A, Camps J, Alonso-Villaverde C, Joven J. Insulin resistance, inflammation, and obesity: role of monocyte chemoattractant protein-1 (or CCL2) in the regulation of metabolism. Mediators Inflamm 2010; 2010: 326580.
    • (2010) Mediators Inflamm , vol.2010 , pp. 326580
    • Rull, A.1    Camps, J.2    Alonso-Villaverde, C.3    Joven, J.4
  • 96
    • 84857576006 scopus 로고    scopus 로고
    • RNA aptamers and spiegelmers: synthesis, purification, and post-synthetic PEG conjugation
    • Hoffmann S, Hoos J, Klussmann S, Vonhoff S. RNA aptamers and spiegelmers: synthesis, purification, and post-synthetic PEG conjugation. Curr Protoc Nucleic Acid Chem 2011; 46: 1–30.
    • (2011) Curr Protoc Nucleic Acid Chem , vol.46 , pp. 1-30
    • Hoffmann, S.1    Hoos, J.2    Klussmann, S.3    Vonhoff, S.4
  • 97
    • 84922431782 scopus 로고    scopus 로고
    • Turning mirror-image oligonucleotides into drugs: the evolution of spiegelmer therapeutics
    • Vater A, Klussmann S. Turning mirror-image oligonucleotides into drugs: the evolution of spiegelmer therapeutics. Drug Discov Today 2015; 1: 147–55.
    • (2015) Drug Discov Today , vol.1 , pp. 147-155
    • Vater, A.1    Klussmann, S.2
  • 98
    • 72449209766 scopus 로고    scopus 로고
    • REG-1, a regimen comprising RB-006, a Factor IXa antagonist, and its oligonucleotide active control agent RB-007 for the potential treatment of arterial thrombosis
    • Becker RC, Chan MY. REG-1, a regimen comprising RB-006, a Factor IXa antagonist, and its oligonucleotide active control agent RB-007 for the potential treatment of arterial thrombosis. Curr Opin Mol Ther 2009; 11: 707–15.
    • (2009) Curr Opin Mol Ther , vol.11 , pp. 707-715
    • Becker, R.C.1    Chan, M.Y.2
  • 99
    • 79851499067 scopus 로고    scopus 로고
    • A randomized, partially blinded, multicenter, active-controlled, dose-ranging study assessing the safety, efficacy, and pharmacodynamics of the REG1 anticoagulation system in patients with acute coronary syndromes: design and rationale of the RADAR Phase IIb trial
    • Povsic TJ, Cohen MG, Mehran R, Buller CE, Bode C, Cornel JH, et al. A randomized, partially blinded, multicenter, active-controlled, dose-ranging study assessing the safety, efficacy, and pharmacodynamics of the REG1 anticoagulation system in patients with acute coronary syndromes: design and rationale of the RADAR Phase IIb trial. Am Heart J 2011; 161: 261–8.
    • (2011) Am Heart J , vol.161 , pp. 261-268
    • Povsic, T.J.1    Cohen, M.G.2    Mehran, R.3    Buller, C.E.4    Bode, C.5    Cornel, J.H.6
  • 100
    • 84921518137 scopus 로고    scopus 로고
    • Ribozymes: nucleic acid enzymes with potential pharmaceutical applications: a review
    • Abera G, Berhanu G, Tekewe A. Ribozymes: nucleic acid enzymes with potential pharmaceutical applications: a review. Pharmacophore 2012; 3: 164–78.
    • (2012) Pharmacophore , vol.3 , pp. 164-178
    • Abera, G.1    Berhanu, G.2    Tekewe, A.3
  • 102
    • 0035290919 scopus 로고    scopus 로고
    • Angiozyme: a novel angiogenesis inhibitor
    • Weng DE, Usman N. Angiozyme: a novel angiogenesis inhibitor. Curr Oncol Rep 2001; 3: 141–6.
    • (2001) Curr Oncol Rep , vol.3 , pp. 141-146
    • Weng, D.E.1    Usman, N.2
  • 103
    • 0242694722 scopus 로고    scopus 로고
    • Preliminary analysis of a phase II study of HEPTAZYMETM, a nuclease resistant ribozyme targeting hepatitis C virus (HCV) RNA
    • Tong M, Schiff E, Jensen DM. Preliminary analysis of a phase II study of HEPTAZYMETM, a nuclease resistant ribozyme targeting hepatitis C virus (HCV) RNA. Hepatology 2002; 36: 360A.
    • (2002) Hepatology , vol.36 , pp. 360A
    • Tong, M.1    Schiff, E.2    Jensen, D.M.3
  • 104
    • 84965000033 scopus 로고    scopus 로고
    • Circular RNAs are long-lived and display only minimal early alterations in response to a growth factor
    • Enuka Y, Lauriola M, Feldman ME, Sas-Chen A, Ulitsky I, Yarden Y. Circular RNAs are long-lived and display only minimal early alterations in response to a growth factor. Nucleic Acids Res 2016; 44: 1370–83.
    • (2016) Nucleic Acids Res , vol.44 , pp. 1370-1383
    • Enuka, Y.1    Lauriola, M.2    Feldman, M.E.3    Sas-Chen, A.4    Ulitsky, I.5    Yarden, Y.6
  • 105
    • 84924274183 scopus 로고    scopus 로고
    • Exon-intron circular RNAs regulate transcription in the nucleus
    • Li Z, Huang C, Bao C, Chen L, Lin M, Wang X, et al. Exon-intron circular RNAs regulate transcription in the nucleus. Nat Struct Mol Biol 2015; 22: 256–64.
    • (2015) Nat Struct Mol Biol , vol.22 , pp. 256-264
    • Li, Z.1    Huang, C.2    Bao, C.3    Chen, L.4    Lin, M.5    Wang, X.6
  • 106
    • 84911491114 scopus 로고    scopus 로고
    • Circular RNAs: diversity of form and function
    • Lasda E, Parker R. Circular RNAs: diversity of form and function. RNA 2014; 20: 1829–42.
    • (2014) RNA , vol.20 , pp. 1829-1842
    • Lasda, E.1    Parker, R.2
  • 107
    • 80455143733 scopus 로고    scopus 로고
    • miRNA-dependent gene silencing involving Ago2-mediated cleavage of a circular antisense RNA
    • Hansen TB, Wiklund ED, Bramsen JB, Villadsen SB, Statham AL, Clark SJ, et al. miRNA-dependent gene silencing involving Ago2-mediated cleavage of a circular antisense RNA. EMBO J 2011; 30: 4414–22.
    • (2011) EMBO J , vol.30 , pp. 4414-4422
    • Hansen, T.B.1    Wiklund, E.D.2    Bramsen, J.B.3    Villadsen, S.B.4    Statham, A.L.5    Clark, S.J.6
  • 108
    • 84900322651 scopus 로고    scopus 로고
    • Detecting and characterizing circular RNAs
    • Jeck WR, Sharpless NE. Detecting and characterizing circular RNAs. Nat Biotechnol 2014; 32: 453–61.
    • (2014) Nat Biotechnol , vol.32 , pp. 453-461
    • Jeck, W.R.1    Sharpless, N.E.2
  • 109
    • 84901838697 scopus 로고    scopus 로고
    • Assessing the ceRNA hypothesis with quantitative measurements of miRNA and target abundance
    • Denzler R, Agarwal V, Stefano J, Bartel DP, Stoffel M. Assessing the ceRNA hypothesis with quantitative measurements of miRNA and target abundance. Mol Cell 2014; 54: 766–76.
    • (2014) Mol Cell , vol.54 , pp. 766-776
    • Denzler, R.1    Agarwal, V.2    Stefano, J.3    Bartel, D.P.4    Stoffel, M.5
  • 110
    • 78650696753 scopus 로고    scopus 로고
    • Expression of linear and novel circular forms of an INK4/ARF-associated non-coding RNA correlates with atherosclerosis risk
    • Burd CE, Jeck WR, Liu Y, Sanoff HK, Wang Z, Sharpless NE. Expression of linear and novel circular forms of an INK4/ARF-associated non-coding RNA correlates with atherosclerosis risk. PLoS Genet 2010; 6: e1001233.
    • (2010) PLoS Genet , vol.6
    • Burd, C.E.1    Jeck, W.R.2    Liu, Y.3    Sanoff, H.K.4    Wang, Z.5    Sharpless, N.E.6
  • 111
    • 84937638960 scopus 로고    scopus 로고
    • Circular RNA: a new star of noncoding of RNAs
    • Qu S, Yang X, Li X, Wang J, Gao Y, Shang R, et al. Circular RNA: a new star of noncoding of RNAs. Cancer Lett 2015; 365: 141–8.
    • (2015) Cancer Lett , vol.365 , pp. 141-148
    • Qu, S.1    Yang, X.2    Li, X.3    Wang, J.4    Gao, Y.5    Shang, R.6
  • 112
    • 84922973651 scopus 로고    scopus 로고
    • Using circular RNA as a novel type of biomarker in the screening of gastric cancer
    • Li P, Chen S, Chen H, Mo X, Li T, Shao Y, et al. Using circular RNA as a novel type of biomarker in the screening of gastric cancer. Clin Chim Acta 2015; 444: 132–6.
    • (2015) Clin Chim Acta , vol.444 , pp. 132-136
    • Li, P.1    Chen, S.2    Chen, H.3    Mo, X.4    Li, T.5    Shao, Y.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.