-
1
-
-
84895548648
-
Lomitapide and mipomersen: Two first-in-class drugs for reducing lowdensity lipoprotein cholesterol in patients with homozygous familial hypercholesterolemia
-
Rader DJ, Kastelein JJ. 2014. Lomitapide and mipomersen: two first-in-class drugs for reducing lowdensity lipoprotein cholesterol in patients with homozygous familial hypercholesterolemia. Circulation 129:1022-32
-
(2014)
Circulation
, vol.129
, pp. 1022-1032
-
-
Rader, D.J.1
Kastelein, J.J.2
-
2
-
-
77949512140
-
RNA targeting therapeutics:molecular mechanisms of antisense oligonucleotides as a therapeutic platform
-
Bennett CF, Swayze EE. 2010.RNA targeting therapeutics:molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Ann. Rev. Pharmacol. Toxicol. 50:259-93
-
(2010)
Ann. Rev. Pharmacol. Toxicol
, vol.50
, pp. 259-293
-
-
Bennett, C.F.1
Swayze, E.E.2
-
3
-
-
85009118442
-
-
NS Templeton Boca Raton, FL CRC Press
-
Bennett CF, Swayze E, Henry S, Geary R. 2015. Antisense oligonucleotide-based therapeutics. In Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, ed. NS Templeton, pp. 467-92. Boca Raton, FL: CRC Press
-
(2015)
Antisense Oligonucleotide-based Therapeutics Gene and Cell Therapy: Therapeutic Mechanisms and Strategies
, pp. 467-492
-
-
Bennett, C.F.1
Swayze, E.2
Henry, S.3
Geary, R.4
-
4
-
-
84940746287
-
-
ed. NS Templeton Boca Raton, FL CRC Press
-
Akinc A, Betterncourt BR, Maier MA. 2015. Development of RNAi therapeutics. In Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, ed. NS Templeton, pp. 493-520. Boca Raton, FL: CRC Press
-
(2015)
Development of RNAi therapeutics Gene and Cell Therapy: Therapeutic Mechanisms and Strategies
, pp. 493-520
-
-
Akinc, A.1
Betterncourt, B.R.2
Maier, M.A.3
-
5
-
-
85009115942
-
Therapeutic applications of morpholino oligonucleotides
-
ed. NS Templeton Boca Raton, FL CRC Press
-
Soto-Pantoja DR, Isenberg JS, Roberts DD. 2015. Therapeutic applications of morpholino oligonucleotides. In Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, ed. NS Templeton, pp. 521-41. Boca Raton, FL: CRC Press
-
(2015)
Gene and Cell Therapy: Therapeutic Mechanisms and Strategies
, pp. 521-541
-
-
Soto-Pantoja, D.R.1
Isenberg, J.S.2
Roberts, D.D.3
-
6
-
-
85057669819
-
The RNase H mechanism
-
ed. ST Crooke Boca Raton, FL CRC Press
-
Lima WF, Wu H, Crooke ST. 2008. The RNase H mechanism. In Antisense Drug Technology: Principles, Strategies, and Applications, ed. ST Crooke, pp. 47-74. Boca Raton, FL: CRC Press
-
(2008)
Antisense Drug Technology: Principles, Strategies, and Applications
, pp. 47-74
-
-
Lima, W.F.1
Wu, H.2
Crooke, S.T.3
-
7
-
-
33645075443
-
Mir-122 regulation of lipid metabolism revealed by in vivo antisense targeting
-
Esau C, Davis S, Murray SF, Yu XX, Pandey SK, et al. 2006. miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting. Cell Metab. 3:87-98
-
(2006)
Cell Metab
, vol.3
, pp. 87-98
-
-
Esau, C.1
Davis, S.2
Murray, S.F.3
Yu, X.X.4
Pandey, S.K.5
-
8
-
-
84881029988
-
Therapeutic silencing of microRNA-33 inhibits the progression of atherosclerosis in Ldlr/mice-brief report
-
Rotllan N, Raḿrez CM, Aryal B, Esau CC, Fernández-Hernando C. 2013. Therapeutic silencing of microRNA-33 inhibits the progression of atherosclerosis in Ldlr/mice-brief report. Arterioscler. Thromb. Vasc. Biol. 33:1973-77
-
(2013)
Arterioscler. Thromb. Vasc. Biol
, vol.33
, pp. 1973-1977
-
-
Rotllan, N.R.1
-
9
-
-
84981294965
-
Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames
-
Liang X-H, Shen W, Sun H, Migawa MT, Vickers TA, Crooker ST. 2016. Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames. Nat. Biotechnol. 34:875-880
-
(2016)
Nat. Biotechnol
, vol.34
, pp. 875-880
-
-
Liang, X.-H.1
Shen, W.2
Sun, H.3
Migawa, M.T.4
Vickers, T.A.5
Crooker, S.T.6
-
11
-
-
0029664865
-
Oligonucleotide N3-P5-phosphoramidates as antisense agents
-
Gryaznov S, Skorski T, Cucco C, Nieborowska-Skorska M, Chiu CY, et al. 1996. Oligonucleotide N3-P5-phosphoramidates as antisense agents. Nucleic Acids Res. 24:1508-14
-
(1996)
Nucleic Acids Res
, vol.24
, pp. 1508-1514
-
-
Gryaznov, S.1
Skorski, T.2
Cucco, C.3
Nieborowska-Skorska, M.4
Chiu, C.Y.5
-
12
-
-
0033812002
-
Heterocyclic modifications of oligonucleotides and antisense technology
-
Herdewijn P. 2000. Heterocyclic modifications of oligonucleotides and antisense technology. Antisense Nucleic Acid Drug Dev. 10:297-310
-
(2000)
Antisense Nucleic Acid Drug Dev
, vol.10
, pp. 297-310
-
-
Herdewijn, P.1
-
13
-
-
0030727765
-
The ups and downs of nucleic acid duplex stability: Structure-stability studies on chemically-modified DNA:RNA duplexes
-
Freier SM, Altmann K-H. 1997. The ups and downs of nucleic acid duplex stability: structure-stability studies on chemically-modified DNA:RNA duplexes. Nucleic Acids Res. 25:4429-43
-
(1997)
Nucleic Acids Res
, vol.25
, pp. 4429-4443
-
-
Freier, S.M.1
Altmann, K.-H.2
-
14
-
-
0028931102
-
CpG motifs in bacterial DNA trigger direct B-cell activation
-
Krieg AM, Yi AK, Matson S, Waldschmidt TJ, Bishop GA, et al. 1995. CpG motifs in bacterial DNA trigger direct B-cell activation. Nature 374:546-49
-
(1995)
Nature
, vol.374
, pp. 546-549
-
-
Krieg, A.M.1
Yi, A.K.2
Matson, S.3
Waldschmidt, T.J.4
Bishop, G.A.5
-
15
-
-
0026783155
-
Oligodeoxynucleotides containing C-5 propyne analogs of 2-deoxyuridine and 2-deoxycytidine
-
Froehler BC, Wadwani S, Terhorst TJ, Gerrard SR. 1992. Oligodeoxynucleotides containing C-5 propyne analogs of 2-deoxyuridine and 2-deoxycytidine. Tetrahedron Lett. 33:5307-10
-
(1992)
Tetrahedron Lett
, vol.33
, pp. 5307-5310
-
-
Froehler, B.C.1
Wadwani, S.2
Terhorst, T.J.3
Gerrard, S.R.4
-
16
-
-
24144492298
-
Evaluation of C-5 propynyl pyrmidine-containing oligonucleotides in vitro and in vivo
-
Shen L, Siwkowski A, Wancewicz EV, Lesnik EA, Butler M, et al. 2003. Evaluation of C-5 propynyl pyrmidine-containing oligonucleotides in vitro and in vivo. Antisense Nucleic Acid Drug Dev. 13:129-42
-
(2003)
Antisense Nucleic Acid Drug Dev
, vol.13
, pp. 129-142
-
-
Shen, L.1
Siwkowski, A.2
Wancewicz, E.V.3
Lesnik, E.A.4
Butler, M.5
-
17
-
-
0027168725
-
Evaluation of 2-modified oligonucleotides containing 2-deoxy gaps as antisense inhibitors of gene expression
-
Monia BP, Lesnik EA, Gonzalez C, LimaWF, McGeeD, et al. 1993. Evaluation of 2-modified oligonucleotides containing 2-deoxy gaps as antisense inhibitors of gene expression. J. Biol. Chem. 268:14514-22
-
(1993)
J. Biol. Chem
, vol.268
, pp. 14514-14522
-
-
Monia, B.P.1
Lesnik, E.A.2
Gonzalez, C.3
Lima, W.F.4
McGee, D.5
-
18
-
-
33646543312
-
A phase i safety and dose escalation trial of docetaxel combined with GEMR-231, a second generation antisense oligonucleotide targeting protein kinase A R1in patients with advanced solid cancers
-
Goel S, Desai K, Macapinlac M, Wadler S, Goldberg G, et al. 2006. A phase I safety and dose escalation trial of docetaxel combined with GEMR-231, a second generation antisense oligonucleotide targeting protein kinase A R1in patients with advanced solid cancers. Investig. New Drugs 24:125-34
-
(2006)
Investig. New Drugs
, vol.24
, pp. 125-134
-
-
Goel, S.1
Desai, K.2
Macapinlac, M.3
Wadler, S.4
Goldberg, G.5
-
19
-
-
84907991204
-
Safety and efficacy of drisapersen for the treatment ofDuchenne muscular dystrophy (DEMANDII): An exploratory, randomised, placebocontrolled phase 2 study
-
Voit T, Topaloglu H, Straub V, Muntoni F, Deconinck N, et al. 2014. Safety and efficacy of drisapersen for the treatment ofDuchenne muscular dystrophy (DEMANDII): an exploratory, randomised, placebocontrolled phase 2 study. Lancet Neurol. 13:987-96
-
(2014)
Lancet Neurol
, vol.13
, pp. 987-996
-
-
Voit, T.1
Topaloglu, H.2
Straub, V.3
Muntoni, F.4
Deconinck, N.5
-
20
-
-
9144247189
-
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
-
Soutschek J, Akinc A, Bramlage B, Charisse K, Constien R, et al. 2004. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432:173-78
-
(2004)
Nature
, vol.432
, pp. 173-178
-
-
Soutschek, J.1
Akinc, A.2
Bramlage, B.3
Charisse, K.4
Constien, R.5
-
21
-
-
33646185371
-
RNAi-Mediated gene silencing in Non-Human primates
-
ZimmermannTS, Lee AC, Akinc A, Bramlage B, Bumcrot D, et al. 2006. RNAi-mediated gene silencing in non-human primates. Nature 441:111-14
-
(2006)
Nature
, vol.441
, pp. 111-114
-
-
Zimmermann, T.S.1
Lee, A.C.2
Akinc, A.3
Bramlage, B.4
Bumcrot, D.5
-
22
-
-
33745614074
-
Position-specific chemical modification of siRNAs reduces Off-Target transcript silencing
-
Jackson AL, Burchard J, Leake D, Reynolds A, Schelter J, et al. 2006. Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing. RNA 12:1197-205
-
(2006)
RNA
, vol.12
, pp. 1197-1205
-
-
Jackson, A.L.1
Burchard, J.2
Leake, D.3
Reynolds, A.4
Schelter, J.5
-
23
-
-
13944261143
-
Fully 2-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA
-
Allerson CR, Sioufi N, Jarres R, Prakash TP, Naik N, et al. 2005. Fully 2-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA. J. Med. Chem. 48:901-4
-
(2005)
J. Med. Chem
, vol.48
, pp. 901-904
-
-
Allerson, C.R.1
Sioufi, N.2
Jarres, R.3
Prakash, T.P.4
Naik, N.5
-
24
-
-
84918578407
-
MultivalentN-acetylgalactosamineconjugated siRNAlocalizes in hepatocytes and elicits robust RNAi-mediated gene silencing
-
Nair JK, Willoughby JLS, Chan A, Charisse K, Alam MR, et al. 2014. MultivalentN-acetylgalactosamineconjugated siRNAlocalizes in hepatocytes and elicits robust RNAi-mediated gene silencing. J. Am.Chem. Soc. 136:16958-61
-
(2014)
J. Am.Chem. Soc
, vol.136
, pp. 16958-16961
-
-
Nair, J.K.1
Willoughby, J.L.S.2
Chan, A.3
Charisse, K.4
Alam, M.R.5
-
25
-
-
0033965192
-
Chemically modified oligonucleotides exhibit decreased immune stimulation in mice
-
Henry S, Stecker K, Brooks D, Monteith D, Conklin B, Bennett CF. 2000. Chemically modified oligonucleotides exhibit decreased immune stimulation in mice. J. Pharmacol. Exp. Ther. 292:468-79
-
(2000)
J. Pharmacol. Exp. Ther
, vol.292
, pp. 468-479
-
-
Henry, S.1
Stecker, K.2
Brooks, D.3
Monteith, D.4
Conklin, B.5
Bennett, C.F.6
-
26
-
-
84880668457
-
Clinical pharmacological properties of mipomersen (Kynamro), a second generation antisense inhibitor of apolipoprotein B
-
Crooke ST, Geary RS. 2013. Clinical pharmacological properties of mipomersen (Kynamro), a second generation antisense inhibitor of apolipoprotein B. Br. J. Clin. Pharmacol. 76:269-76
-
(2013)
Br. J. Clin. Pharmacol
, vol.76
, pp. 269-276
-
-
Crooke, S.T.1
Geary, R.S.2
-
27
-
-
84960517144
-
Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy
-
Chiriboga CA, Swoboda KJ, Darras BT, Iannaccone ST, Montes J, et al. 2016. Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy. Neurology 86:890-97
-
(2016)
Neurology
, vol.86
, pp. 890-897
-
-
Chiriboga, C.A.1
Swoboda, K.J.2
Darras, B.T.3
Iannaccone, S.T.4
Montes, J.5
-
28
-
-
84903546492
-
Pharmacology of a central nervous system delivered 2-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates
-
Rigo F, Chun SJ, Norris DA, Hung G, Lee S, et al. 2014. Pharmacology of a central nervous system delivered 2-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates. J. Pharmacol. Exp. Ther. 350:46-55
-
(2014)
J. Pharmacol. Exp. Ther
, vol.350
, pp. 46-55
-
-
Rigo, F.1
Chun, S.J.2
Norris, D.A.3
Hung, G.4
Lee, S.5
-
29
-
-
84976444883
-
Suppressing transthyretin production in mice, monkeys and humans using 2nd-generation antisense oligonucleotides
-
Ackermann EJ, Guo S, BensonMD, Booten S, Freier S, et al. 2016. Suppressing transthyretin production in mice, monkeys and humans using 2nd-generation antisense oligonucleotides. Amyloid. 23:148-57
-
(2016)
Amyloid
, vol.23
, pp. 148-157
-
-
Ackermann, E.J.1
Guo, S.2
Bensonmd Booten, S.3
Freier, S.4
-
30
-
-
84865682650
-
Single-Stranded siRNAs activate RNAi in animals
-
Lima WF, Prakash TP, Murray HM, Kinberger GA, Li W, et al. 2012. Single-stranded siRNAs activate RNAi in animals. Cell 150:883-94
-
(2012)
Cell
, vol.150
, pp. 883-894
-
-
Lima, W.F.1
Prakash, T.P.2
Murray, H.M.3
Kinberger, G.A.4
Li, W.5
-
31
-
-
0030862285
-
Synthesis of 2-O,4-C-methyleneuridine and-cytidine Novel bicyclic nucleosides having a fixed C3,-endo sugar puckering
-
Obika S, Nanbu D, Hari Y, Morio K-I, In Y, et al. 1997. Synthesis of 2-O,4-C-methyleneuridine and-cytidine. Novel bicyclic nucleosides having a fixed C3,-endo sugar puckering. Tetrahedron Lett. 38:8735-38
-
(1997)
Tetrahedron Lett
, vol.38
, pp. 8735-8738
-
-
Obika, S.1
Nanbu, D.2
Hari, Y.3
Morio, K.-I.Y.4
-
32
-
-
0032909507
-
Synthesis of 3-C-and 4-C-branched oligodeoxynucleotides and the development of locked nucleic acid (LNA)
-
Wengel J. 1999. Synthesis of 3-C-and 4-C-branched oligodeoxynucleotides and the development of locked nucleic acid (LNA). Acc. Chem. Res. 32:301-10
-
(1999)
Acc. Chem. Res
, vol.32
, pp. 301-310
-
-
Wengel, J.1
-
33
-
-
0036567147
-
Design of antisense oligonucleotides stabilized by locked nucleic acids
-
Kurreck J, Wyszko E, Gillen C, Erdmann VA. 2002. Design of antisense oligonucleotides stabilized by locked nucleic acids. Nucleic Acids Res. 30:1911-18
-
(2002)
Nucleic Acids Res
, vol.30
, pp. 1911-1918
-
-
Kurreck, J.1
Wyszko, E.2
Gillen, C.3
Erdmann, V.A.4
-
34
-
-
84926429679
-
Unnatural nucleoside analogs for antisense therapy
-
ed. S Hanessian Weinheim, Ger Wiley-VCH
-
Seth PP, Swayze EE. 2014. Unnatural nucleoside analogs for antisense therapy. In Natural Products in Medicinal Chemistry, ed. S Hanessian, pp. 403-40. Weinheim, Ger.: Wiley-VCH
-
(2014)
Natural Products in Medicinal Chemistry
, pp. 403-440
-
-
Seth, P.P.1
Swayze, E.E.2
-
35
-
-
20444479844
-
On the in vitro and in vivo properties of four locked nucleic acid nucleotides incorporated into an anti-H-Ras antisense oligonucleotide
-
Fluiter K, Frieden M, Vreijling J, Rosenbohm C, De Wissel MB, et al. 2005. On the in vitro and in vivo properties of four locked nucleic acid nucleotides incorporated into an anti-H-Ras antisense oligonucleotide. ChemBioChem 6:1104-9
-
(2005)
ChemBioChem
, vol.6
, pp. 1104-1109
-
-
Fluiter, K.1
Frieden, M.2
Vreijling, J.3
Rosenbohm, C.4
De Wissel, M.B.5
-
36
-
-
0037033135
-
2-O,4-C-ethylene-bridged nucleic acids (ENA): Highly nuclease-resistant and thermodynamically stable oligonucleotides for antisense drug
-
Morita K, Hasegawa C, Kaneko M, Tsutsumi S, Sone J, et al. 2002. 2-O,4-C-ethylene-bridged nucleic acids (ENA): highly nuclease-resistant and thermodynamically stable oligonucleotides for antisense drug. Bioorg. Med. Chem. Lett. 12:73-76
-
(2002)
Bioorg. Med. Chem. Lett
, vol.12
, pp. 73-76
-
-
Morita, K.1
Hasegawa, C.2
Kaneko, M.3
Tsutsumi, S.4
Sone, J.5
-
37
-
-
84899807328
-
Sequence motifs associated with hepatotoxicity of locked nucleic acid-modified antisense oligonucleotides
-
Burdick AD, Sciabola S, Mantena SR, Hollingshead BD, Stanton R, et al. 2014. Sequence motifs associated with hepatotoxicity of locked nucleic acid-modified antisense oligonucleotides. Nucleic Acids Res. 42:4882-489
-
(2014)
Nucleic Acids Res
, vol.42
, pp. 4882-5489
-
-
Burdick, A.D.1
Sciabola, S.2
Mantena, S.R.3
Hollingshead, B.D.4
Stanton, R.5
-
38
-
-
33846902312
-
Antisense oligonucleotides containing locked nucleic acid (LNA) improve potency but cause significant hepatotoxicity in animals
-
Swayze EE, Siwkowski AM, Wancewicz EV, Migawa MT, Wyrzykiewicz TK, et al. 2007. Antisense oligonucleotides containing locked nucleic acid (LNA) improve potency but cause significant hepatotoxicity in animals. Nucleic Acids Res. 35:687-700
-
(2007)
Nucleic Acids Res
, vol.35
, pp. 687-700
-
-
Swayze, E.E.1
Siwkowski, A.M.2
Wancewicz, E.V.3
Migawa, M.T.4
Wyrzykiewicz, T.K.5
-
39
-
-
77649230488
-
Antisense oligonucleotides containing conformationally constrained 2-,4-(N-methoxy)aminomethylene and 2-,4-aminooxymethylene and 2-O,4-C-aminomethylene bridged nucleoside analogues show improved potency in animal models
-
Prakash TP, Siwkowski A, Allerson CR, Migawa MT, Lee S, et al. 2010. Antisense oligonucleotides containing conformationally constrained 2-,4-(N-methoxy)aminomethylene and 2-,4-aminooxymethylene and 2-O,4-C-aminomethylene bridged nucleoside analogues show improved potency in animal models. J. Med. Chem. 53:1636-50
-
(2010)
J. Med. Chem
, vol.53
, pp. 1636-1650
-
-
Prakash, T.P.1
Siwkowski, A.2
Allerson, C.R.3
Migawa, M.T.4
Lee, S.5
-
40
-
-
84868347571
-
Structure activity relationships of-L-LNA modified phosphorothioate gapmer antisense oligonucleotides in animals
-
Seth PP, Jazayeri A, Yu J, Allerson CR, Bhat B, Swayze EE. 2012. Structure activity relationships of-L-LNA modified phosphorothioate gapmer antisense oligonucleotides in animals. Mol. Ther. Nucleic Acids 1:e47
-
(2012)
Mol. Ther. Nucleic Acids
, vol.1
, pp. e47
-
-
Seth, P.P.1
Jazayeri, A.2
Yu, J.3
Allerson, C.R.4
Bhat, B.5
Swayze, E.E.6
-
41
-
-
84864199342
-
Structure and nuclease resistance of 2-,4-constrained 2-O-methoxyethyl (cMOE) and 2-O-ethyl (cEt) modified DNAs
-
Pallan PS, Allerson CR, Berdeja A, Seth PP, Swayze EE, et al. 2012. Structure and nuclease resistance of 2-,4-constrained 2-O-methoxyethyl (cMOE) and 2-O-ethyl (cEt) modified DNAs. Chem. Commun. 48:8195-97
-
(2012)
Chem. Commun
, vol.48
, pp. 8195-8197
-
-
Pallan, P.S.1
Allerson, C.R.2
Berdeja, A.3
Seth, P.P.4
Swayze, E.E.5
-
42
-
-
80054756151
-
Synthesis, improved antisense activity and structural rationale for the divergent RNA affinities of 3-fluoro hexitol nucleic acid (FHNA and Ara-FHNA) modified oligonucleotides
-
Egli M, Pallan PS, Allerson CR, Prakash TP, Berdeja A, et al. 2011. Synthesis, improved antisense activity and structural rationale for the divergent RNA affinities of 3-fluoro hexitol nucleic acid (FHNA and Ara-FHNA) modified oligonucleotides. J. Am. Chem. Soc. 133:16642-49
-
(2011)
J. Am. Chem. Soc
, vol.133
, pp. 16642-16649
-
-
Egli, M.1
Pallan, P.S.2
Allerson, C.R.3
Prakash, T.P.4
Berdeja, A.5
-
43
-
-
78649899322
-
Configuration of the 5-methyl group modulates the biophysical and biological properties of locked nucleic acid (LNA) oligonucleotides
-
Seth PP, Allerson CR, Siwkowski A, Vasquez G, Berdeja A, et al. 2010. Configuration of the 5-methyl group modulates the biophysical and biological properties of locked nucleic acid (LNA) oligonucleotides. J. Med. Chem. 53:8309-18
-
(2010)
J. Med. Chem
, vol.53
, pp. 8309-8318
-
-
Seth, P.P.1
Allerson, C.R.2
Siwkowski, A.3
Vasquez, G.4
Berdeja, A.5
-
44
-
-
84878247636
-
Preclinical evaluation of the toxicological effects of a novel constrained ethylmodified antisense compound targeting signal transducer and activator of transcription 3 in mice and cynomolgus monkeys
-
Burel SA, Han SR, Lee HS, Norris DA, Lee BS, et al. 2013. Preclinical evaluation of the toxicological effects of a novel constrained ethylmodified antisense compound targeting signal transducer and activator of transcription 3 in mice and cynomolgus monkeys. Nucleic Acid Ther. 23:213-27
-
(2013)
Nucleic Acid Ther
, vol.23
, pp. 213-227
-
-
Burel, S.A.1
Han, S.R.2
Lee, H.S.3
Norris, D.A.4
Lee, B.S.5
-
45
-
-
84946543730
-
Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1
-
Pandey SK, Wheeler TM, Justice SL, Kim A, Younis H, et al. 2015. Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1. J. Pharmacol. Exp. Ther. 355:329-40
-
(2015)
J. Pharmacol. Exp. Ther
, vol.355
, pp. 329-340
-
-
Pandey, S.K.1
Wheeler, T.M.2
Justice, S.L.3
Kim, A.4
Younis, H.5
-
46
-
-
12644302203
-
Pharmacokinetic properties of several novel oligonucleotide analogs in mice
-
Crooke ST, Graham MJ, Zuckerman JE, BrooksD, Conklin BS, et al. 1996. Pharmacokinetic properties of several novel oligonucleotide analogs in mice. J. Pharmacol. Exp. Ther. 277:923-37
-
(1996)
J. Pharmacol. Exp. Ther
, vol.277
, pp. 923-937
-
-
Crooke, S.T.1
Graham, M.J.2
Zuckerman, J.E.3
Brooks, D.4
Conklin, B.S.5
-
47
-
-
28444469246
-
Silencing of microRNAs in vivo with antagomirs
-
Krutzfeldt J, Rajewsky N, Braich R, Rajeev KG, Tuschl T, et al. 2005. Silencing of microRNAs in vivo with "antagomirs." Nature 438:685-89
-
(2005)
Nature
, vol.438
, pp. 685-689
-
-
Krutzfeldt, J.1
Rajewsky, N.2
Braich, R.3
Rajeev, K.G.4
Tuschl, T.5
-
48
-
-
23444453219
-
Lipid modification of GRN163, an N3-P5-thio-phosphoramidate oligonucleotide, enhances the potency of telomerase inhibition
-
Herbert BS, Gellert GC, Hochreiter A, Pongracz K, Wright WE, et al. 2005. Lipid modification of GRN163, an N3-P5-thio-phosphoramidate oligonucleotide, enhances the potency of telomerase inhibition. Oncogene 24:5262-68
-
(2005)
Oncogene
, vol.24
, pp. 5262-5268
-
-
Herbert, B.S.1
Gellert, G.C.2
Hochreiter, A.3
Pongracz, K.4
Wright, W.E.5
-
49
-
-
41149179660
-
Efficient in vivo delivery of siRNA to the liver by conjugation of-tocopherol
-
Nishina K, Unno T, Uno Y, Kubodera T, Kanouchi T, et al. 2008. Efficient in vivo delivery of siRNA to the liver by conjugation of-tocopherol. Mol. Ther. 16:734-40
-
(2008)
Mol. Ther
, vol.16
, pp. 734-740
-
-
Nishina, K.1
Unno, T.2
Uno, Y.3
Kubodera, T.4
Kanouchi, T.5
-
50
-
-
84938917501
-
DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing
-
Nishina K, Piao W, Yoshida-Tanaka K, Sujino Y, Nishina T, et al. 2015. DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing. Nat. Commun. 6:7969
-
(2015)
Nat. Commun
, vol.6
, pp. 7969
-
-
Nishina, K.1
Piao, W.2
Yoshida-Tanaka, K.3
Sujino, Y.4
Nishina, T.5
-
51
-
-
84905584822
-
Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice
-
Prakash TP, Graham MJ, Yu J, Carty R, Low A, et al. 2014. Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice. Nucleic Acids Res. 42:8796-807
-
(2014)
Nucleic Acids Res
, vol.42
, pp. 8796-8807
-
-
Prakash, T.P.1
Graham, M.J.2
Yu, J.3
Carty, R.4
Low, A.5
-
52
-
-
84962171168
-
Comprehensive structure-activity relationship of triantennary N-acetylgalactosamine conjugated antisense oligonucleotides for targeted delivery to hepatocytes
-
Prakash TP, Yu J, Migawa MT, Kinberger GA, WanWB, et al. 2016. Comprehensive structure-activity relationship of triantennary N-acetylgalactosamine conjugated antisense oligonucleotides for targeted delivery to hepatocytes. J. Med. Chem. 59:2718-33
-
(2016)
J. Med. Chem
, vol.59
, pp. 2718-2733
-
-
Prakash, T.P.1
Yu, J.2
Migawa, M.T.3
Kinberger, G.A.4
Wan, W.B.5
-
53
-
-
84937252963
-
Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides
-
Geary RS, Norris D, Yu R, Bennett CF. 2015. Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides. Adv. Drug Deliv. Rev. 87:46-51
-
(2015)
Adv. Drug Deliv. Rev
, vol.87
, pp. 46-51
-
-
Geary, R.S.1
Norris, D.2
Yu, R.3
Bennett, C.F.4
-
54
-
-
84885566527
-
Basic principles of the pharmacokinetics of antisense oligonucleotide drugs
-
ed. ST Crooke Boca Raton, FL CRC Press
-
Levin AA, Yu RZ, Geary RS. 2008. Basic principles of the pharmacokinetics of antisense oligonucleotide drugs. In Antisense Drug Technology: Principles, Strategies, and Applications, ed. ST Crooke, pp. 183-216. Boca Raton, FL: CRC Press
-
(2008)
Antisense Drug Technology: Principles, Strategies, and Applications
, pp. 183-216
-
-
Levin, A.A.1
Yu, R.Z.2
Geary, R.S.3
-
55
-
-
79959479529
-
Mechanisms of singlestranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes
-
Koller E, Vincent TM, Chappell A, De S, Manoharan M, Bennett CF. 2011. Mechanisms of singlestranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes. Nucleic Acids Res. 39:4795-807
-
(2011)
Nucleic Acids Res
, vol.39
, pp. 4795-4807
-
-
Koller, E.1
Vincent, T.M.2
Chappell, A.3
De S Manoharan, M.4
Bennett, C.F.5
-
56
-
-
84862663712
-
Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis
-
Kordasiewicz HB, StanekLM, Wancewicz EV, MazurC, McAlonis MM, et al. 2012. Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron 74:1031-44
-
(2012)
Neuron
, vol.74
, pp. 1031-1044
-
-
Kordasiewicz, H.B.1
Stanek, L.M.2
Wancewicz, E.V.3
Mazur, C.4
McAlonis, M.M.5
-
57
-
-
59749087965
-
Cross-Species comparison of in vivo PK/PD relationships for second-generation antisense oligonucleotides targeting apolipoprotein B-100
-
Yu RZ, Lemonidis KM, Graham MJ, Matson JE, Crooke RM, et al. 2009. Cross-species comparison of in vivo PK/PD relationships for second-generation antisense oligonucleotides targeting apolipoprotein B-100. Biochem. Pharmacol. 77:910-19
-
(2009)
Biochem. Pharmacol
, vol.77
, pp. 910-919
-
-
Yu, R.Z.1
Lemonidis, K.M.2
Graham, M.J.3
Matson, J.E.4
Crooke, R.M.5
-
58
-
-
84890319075
-
Characterization of target mRNA reduction through in situ RNA hybridization in multiple organ systems following systemic antisense treatment in animals
-
Hung G, Xiao X, Peralta R, Bhattacharjee G, Murray S, et al. 2013. Characterization of target mRNA reduction through in situ RNA hybridization in multiple organ systems following systemic antisense treatment in animals. Nucleic Acid Ther. 23:369-78
-
(2013)
Nucleic Acid Ther
, vol.23
, pp. 369-378
-
-
Hung, G.1
Xiao, X.2
Peralta, R.3
Bhattacharjee, G.4
Murray, S.5
-
59
-
-
2642556483
-
Oral bioavailability and multiple dose tolerability of an antisense oligonucleotide tablet formulated with sodium caprate
-
Raoof AA, Chiu P, Ramtoola Z, Cumming IK, Teng CL, et al. 2004. Oral bioavailability and multiple dose tolerability of an antisense oligonucleotide tablet formulated with sodium caprate. J. Pharm. Sci. 93:1431-39
-
(2004)
J. Pharm. Sci
, vol.93
, pp. 1431-1439
-
-
Raoof, A.A.1
Chiu, P.2
Ramtoola, Z.3
Cumming, I.K.4
Teng, C.L.5
-
60
-
-
0033763150
-
Pharmacokinetic and toxicity profile of a phosphorothioate oligonucleotide following inhalation delivery to lung in mice
-
Templin MV, Levin AA, Graham MJ, Aberg PM, Axelsson BI, et al. 2000. Pharmacokinetic and toxicity profile of a phosphorothioate oligonucleotide following inhalation delivery to lung in mice. Antisense Nucleic Acid Drug Dev. 10:359-68
-
(2000)
Antisense Nucleic Acid Drug Dev
, vol.10
, pp. 359-368
-
-
Templin, M.V.1
Levin, A.A.2
Graham, M.J.3
Aberg, P.M.4
Axelsson, B.I.5
-
61
-
-
33646237136
-
Bioavailability and therapeutic activity of alicaforsen (ISIS 2302) administered as a rectal retention enema to subjects with active ulcerative colitis
-
Miner PB Jr., Wedel MK, Xia S, Baker BF, Geary RS, Matson J. 2006. Bioavailability and therapeutic activity of alicaforsen (ISIS 2302) administered as a rectal retention enema to subjects with active ulcerative colitis. Aliment. Pharmacol. Ther. 23:1427-34
-
(2006)
Aliment. Pharmacol. Ther
, vol.23
, pp. 1427-1434
-
-
Miner, P.B.1
Wedel, M.K.2
Xia, S.3
Baker, B.F.4
Geary, R.S.5
Matson, J.6
-
62
-
-
38349171887
-
Oral delivery of antisense oligonucleotides in man
-
Tillman LG, Geary RS, Hardee GE. 2008. Oral delivery of antisense oligonucleotides in man. J. Pharm. Sci. 97:225-36
-
(2008)
J. Pharm. Sci
, vol.97
, pp. 225-236
-
-
Tillman, L.G.1
Geary, R.S.2
Hardee, G.E.3
-
63
-
-
85002982666
-
-
ed. ST Crooke Boca Raton, FL CRC Press
-
Iversen PL. 2008. Morpholinos. In Antisense Drug Technology: Principles, Strategies, and Applications, ed. ST Crooke, pp. 565-82. Boca Raton, FL: CRC Press
-
(2008)
Morpholinos Antisense Drug Technology: Principles, Strategies, and Applications
, pp. 565-582
-
-
Iversen, P.L.1
-
64
-
-
0035989848
-
Pharmacokinetics and tissue distribution of a peptide nucleic acid after intravenous administration
-
McMahon BM, Mays D, Lipsky J, Stewart JA, Fauq A, Richelson E. 2002. Pharmacokinetics and tissue distribution of a peptide nucleic acid after intravenous administration. Antisense Nucleic Acid Drug Dev. 12:65-73
-
(2002)
Antisense Nucleic Acid Drug Dev
, vol.12
, pp. 65-73
-
-
McMahon, B.M.1
Mays, D.2
Lipsky, J.3
Stewart, J.A.4
Fauq, A.5
Richelson, E.6
-
65
-
-
84904901858
-
Toxicological and pharmacokinetic properties of QPI-1007, a chemically modified synthetic siRNA targeting caspase 2 mRNA, following intravitreal injection
-
Solano EC, Kornbrust DJ, Beaudry A, Foy JW, Schneider DJ, Thompson JD. 2014. Toxicological and pharmacokinetic properties of QPI-1007, a chemically modified synthetic siRNA targeting caspase 2 mRNA, following intravitreal injection. Nucleic Acid Ther. 24:258-66
-
(2014)
Nucleic Acid Ther
, vol.24
, pp. 258-266
-
-
Solano, E.C.1
Kornbrust, D.J.2
Beaudry, A.3
Foy, J.W.4
Schneider, D.J.5
Thompson, J.D.6
-
66
-
-
84904872039
-
Toxicological properties of 2-O-methoxyethyl chimeric antisense inhibitors in animals and man
-
ed. ST Crooke Boca Raton, FL CRC Press
-
Henry SP, Kim T-W, Kramer-Strickland K, Zanardi TA, Fey RA, Levin AA. 2008. Toxicological properties of 2-O-methoxyethyl chimeric antisense inhibitors in animals and man. In Antisense Drug Technology: Principles, Strategies, and Applications, ed. ST Crooke, pp. 327-63. Boca Raton, FL: CRC Press
-
(2008)
Antisense Drug Technology: Principles, Strategies, and Applications
, pp. 327-363
-
-
Henry, S.P.1
Kim, T.-W.2
Kramer-Strickland, K.3
Zanardi, T.A.4
Fey, R.A.5
Levin, A.A.6
-
67
-
-
0002582104
-
Toxicity of antisense oligonucleotides
-
ed. ST Crooke New York Marcel Dekker. 1st ed
-
Levin AA, Henry SP, Monteith D, Templin MV. 2001. Toxicity of antisense oligonucleotides. In Antisense Drug Technology: Principles, Strategies, and Applications, ed. ST Crooke, pp. 201-67. New York: Marcel Dekker. 1st ed
-
(2001)
Antisense Drug Technology: Principles, Strategies, and Applications
, pp. 201-267
-
-
Levin, A.A.1
Henry, S.P.2
Monteith, D.3
Templin, M.V.4
-
68
-
-
58749104364
-
The promises and pitfalls of RNA-Interference-Based therapeutics
-
Castanotto D, Rossi JJ. 2009. The promises and pitfalls of RNA-interference-based therapeutics. Nature 457:426-33
-
(2009)
Nature
, vol.457
, pp. 426-433
-
-
Castanotto, D.1
Rossi, J.J.2
-
69
-
-
84963812653
-
Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts
-
Burel SA, Hart CE, Cauntay P, Hsiao J, Machemer T, et al. 2016. Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts. Nucleic Acids Res. 44:2093-109
-
(2016)
Nucleic Acids Res
, vol.44
, pp. 2093-2109
-
-
Burel, S.A.1
Hart, C.E.2
Cauntay, P.3
Hsiao, J.4
Machemer, T.5
-
70
-
-
84981311948
-
Considerations for the characterization and interpretation of results related to alternative complement activation in monkeys associated with oligonucleotide-based therapeutics
-
Henry SP, Seguin R, Cavagnaro J, Berman C, Tepper J, Kornbrust D. 2016. Considerations for the characterization and interpretation of results related to alternative complement activation in monkeys associated with oligonucleotide-based therapeutics. Nucleic Acid Ther. 26:210-15
-
(2016)
Nucleic Acid Ther
, vol.26
, pp. 210-215
-
-
Henry, S.P.1
Seguin, R.2
Cavagnaro, J.3
Berman, C.4
Tepper, J.5
Kornbrust, D.6
-
71
-
-
33745612464
-
Widespread siRNA Off-Target transcript silencing mediated by seed region sequence complementarity
-
Jackson AL, Burchard J, Schelter J, Chau BN, Cleary M, et al. 2006. Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity. RNA 12:1179-87
-
(2006)
RNA
, vol.12
, pp. 1179-1187
-
-
Jackson, A.L.1
Burchard, J.2
Schelter, J.3
Chau, B.N.4
Cleary, M.5
-
72
-
-
74049124186
-
Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application
-
Jackson AL, Linsley PS. 2010. Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application. Nat. Rev. Drug Discov. 9:57-67
-
(2010)
Nat. Rev. Drug Discov
, vol.9
, pp. 57-67
-
-
Jackson, A.L.1
Linsley, P.S.2
-
73
-
-
0037685280
-
Expression profiling reveals off-target gene regulation by RNAi
-
Jackson AL, Bartz SR, Schelter J, Kobayashi SV, Burchard J, et al. 2003. Expression profiling reveals off-target gene regulation by RNAi. Nat. Biotechnol. 21:635-37
-
(2003)
Nat. Biotechnol
, vol.21
, pp. 635-637
-
-
Jackson, A.L.1
Bartz, S.R.2
Schelter, J.3
Kobayashi, S.V.4
Burchard, J.5
-
74
-
-
0035942332
-
Phosphorothioate oligonucleotides inhibit the intrinsic tenase complex by an allosteric mechanism
-
Sheehan JP, Phan TM. 2001. Phosphorothioate oligonucleotides inhibit the intrinsic tenase complex by an allosteric mechanism. Biochemistry 40:4980-89
-
(2001)
Biochemistry
, vol.40
, pp. 4980-4989
-
-
Sheehan, J.P.1
Phan, T.M.2
-
75
-
-
0030922755
-
Activation of the alternative pathway of complement by a phosphorothioate oligonucleotide: Potential mechanism of action
-
Henry SP, Giclas PC, Leeds J, PangburnM, Auletta C, et al. 1997. Activation of the alternative pathway of complement by a phosphorothioate oligonucleotide: potential mechanism of action. J. Pharmacol. Exp. Ther. 281:810-16
-
(1997)
J. Pharmacol. Exp. Ther
, vol.281
, pp. 810-816
-
-
Henry, S.P.1
Giclas, P.C.2
Leeds, J.3
Pangburn, M.4
Auletta, C.5
-
76
-
-
0030842828
-
Immune stimulation- A class effect of phosphorothioate oligodeoxynucleotides in rodents
-
Monteith DK, Henry SP, Howard RB, Flournoy S, Levin AA, et al. 1997. Immune stimulation- A class effect of phosphorothioate oligodeoxynucleotides in rodents. Anticancer Drug Des. 12:421-32
-
(1997)
Anticancer Drug des
, vol.12
, pp. 421-432
-
-
Monteith, D.K.1
Henry, S.P.2
Howard, R.B.3
Flournoy, S.4
Levin, A.A.5
-
77
-
-
23944493932
-
Non-CpG-Containing antisense 2-methoxyethyl oligonucleotides activate a proinflammatory response independent of Toll-like receptor 9 or myeloid differentiation factor
-
Senn JJ, Burel S, Henry SP. 2005. Non-CpG-containing antisense 2-methoxyethyl oligonucleotides activate a proinflammatory response independent of Toll-like receptor 9 or myeloid differentiation factor J. Pharmacol. Exp. Ther. 314:972-79
-
(2005)
J. Pharmacol. Exp. Ther
, vol.314
, pp. 972-979
-
-
Senn, J.J.1
Burel, S.2
Henry, S.P.3
-
78
-
-
84863259511
-
An overview of the clinical safety experience of first-and second-generation antisense oligonucleotides
-
Boca Raton, FL CRC Press
-
Kwoh JT. 2008. An overview of the clinical safety experience of first-and second-generation antisense oligonucleotides. In Antisense Drug Technology: Principles, Strategies, and Applications, ed. ST Crooke, pp. 365-99. Boca Raton, FL: CRC Press
-
(2008)
Antisense Drug Technology: Principles, Strategies, and Applications, Ed. ST Crooke
, pp. 365-399
-
-
Kwoh, J.T.1
-
79
-
-
84980407562
-
Integrated safety assessment of 2-Omethoxyethyl chimeric antisense oligonucleotides in nonhuman primates and healthy human volunteers
-
Crooke ST, Baker BF, Kwoh TJ, Cheng W, Schulz DJ, et al. 2016. Integrated safety assessment of 2-Omethoxyethyl chimeric antisense oligonucleotides in nonhuman primates and healthy human volunteers. Mol. Ther. 10:1771-82
-
(2016)
Mol. Ther
, vol.10
, pp. 1771-1782
-
-
Crooke, S.T.1
Baker, B.F.2
Kwoh, T.J.3
Cheng, W.4
Schulz, D.J.5
-
81
-
-
85053956820
-
Discovery and development of RNAi therapeutics
-
ed. ST Crooke. Boca Raton, FL CRC Press
-
de FougerollesAR, Maraganore JM. 2007. Discovery and development of RNAi therapeutics. In Antisense Drug Technology: Principles, Strategies, and Applications, ed. ST Crooke. Boca Raton, FL: CRC Press
-
(2007)
Antisense Drug Technology: Principles, Strategies, and Applications
-
-
De Fougerolles, A.R.1
Maraganore, J.M.2
-
82
-
-
84908266291
-
Development of RNAi technology for targeted therapy- A track of siRNA based agents to RNAi therapeutics
-
Zhou Y, Zhang C, LiangW. 2014. Development of RNAi technology for targeted therapy- A track of siRNA based agents to RNAi therapeutics. J. Control. Release 193:270-81
-
(2014)
J. Control. Release
, vol.193
, pp. 270-281
-
-
Zhou, Y.1
Zhang, C.2
Liang, W.3
-
83
-
-
0032617560
-
Fomiversen sodium approved to treat CMV retinitis
-
Piascik P. 1999. Fomiversen sodium approved to treat CMV retinitis. J. Am. Pharm. Assoc. 39:84-85
-
(1999)
J. Am. Pharm. Assoc
, vol.39
, pp. 84-85
-
-
Piascik, P.1
-
84
-
-
84925541081
-
Clinical and preclinical pharmacokinetics and pharmacodynamics of mipomersen (Kynamro): A second-generation antisense oligonucleotide inhibitor of apolipoprotein B
-
GearyRS, BakerBF, Crooke ST. 2015. Clinical and preclinical pharmacokinetics and pharmacodynamics of mipomersen (Kynamro): a second-generation antisense oligonucleotide inhibitor of apolipoprotein B. Clin. Pharmacokinet. 54:133-46
-
(2015)
Clin. Pharmacokinet
, vol.54
, pp. 133-146
-
-
Geary, R.S.1
Baker, B.F.2
Crooke, S.T.3
-
85
-
-
84963959871
-
Pediatric experience with mipomersen as adjunctive therapy for homozygous familial hypercholesterolemia
-
Raal FJ, Braamskamp MJ, Selvey SL, Sensinger CH, Kastelein JJP. 2016. Pediatric experience with mipomersen as adjunctive therapy for homozygous familial hypercholesterolemia. J. Clin. Lipidol. 10:860-69
-
(2016)
J. Clin. Lipidol
, vol.10
, pp. 860-869
-
-
Raal, F.J.1
Braamskamp, M.J.2
Selvey, S.L.3
Sensinger, C.H.4
Kastelein, J.J.P.5
-
86
-
-
84921866616
-
Mipomersen, an antisense oligonucleotide to apolipoprotein B-100, reduces lipoprotein(a) in various populations with hypercholesterolemia: Results of 4 phase III trials
-
Santos RD, Raal FJ, Catapano AL, Witztum JL, Steinhagen-Thiessen E, Tsimikas S. 2015. Mipomersen, an antisense oligonucleotide to apolipoprotein B-100, reduces lipoprotein(a) in various populations with hypercholesterolemia: results of 4 phase III trials. Arterioscler. Thromb. Vasc. Biol. 35:689-99
-
(2015)
Arterioscler. Thromb. Vasc. Biol
, vol.35
, pp. 689-699
-
-
Santos, R.D.1
Raal, F.J.2
Catapano, A.L.3
Witztum, J.L.4
Steinhagen-Thiessen, E.5
Tsimikas, S.6
-
87
-
-
84958104510
-
No effect on QT intervals of mipomersen, a 2-O-methoxyethyl modified antisense oligonucleotide targeting ApoB-100 mRNA, in a phase i dose escalation placebo-controlled study, and confirmed by a thorough QT (tQT) study, in healthy subjects
-
Yu RZ, Gunawan R, Li Z, Mittleman RS, Mahmood A, et al. 2016. No effect on QT intervals of mipomersen, a 2-O-methoxyethyl modified antisense oligonucleotide targeting ApoB-100 mRNA, in a phase I dose escalation placebo-controlled study, and confirmed by a thorough QT (tQT) study, in healthy subjects. Eur. J. Clin. Pharmacol. 72:267-75
-
(2016)
Eur. J. Clin. Pharmacol
, vol.72
, pp. 267-275
-
-
Yu, R.Z.1
Gunawan, R.2
Li, Z.3
Mittleman, R.S.4
Mahmood, A.5
-
88
-
-
84878637167
-
Antisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, nonhuman primates, and humans
-
Graham MJ, Lee RG, Bell TA III, Fu W, Mullick AE, et al. 2013. Antisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, nonhuman primates, and humans. Circ. Res. 112:1479-90
-
(2013)
Circ. Res
, vol.112
, pp. 1479-1490
-
-
Graham, M.J.1
Lee, R.G.2
Bell, T.A.3
Fu, W.4
Mullick, A.E.5
-
89
-
-
58149262866
-
A null mutation in human APOC3 confers a favorable plasma lipid profile and apparent cardioprotection
-
Pollin TI, Damcott CM, Shen H, Ott SH, Shelton J, et al. 2008. A null mutation in human APOC3 confers a favorable plasma lipid profile and apparent cardioprotection. Science 322:1702-5
-
(2008)
Science
, vol.322
, pp. 1702-1705
-
-
Pollin, T.I.1
Damcott, C.M.2
Shen, H.3
Ott, S.H.4
Shelton, J.5
-
90
-
-
84918803944
-
Targeting APOC3 in the familial chylomicronemia syndrome
-
Gaudet D, Brisson D, Tremblay K, Alexander VJ, Singleton W, et al. 2014. Targeting APOC3 in the familial chylomicronemia syndrome. N. Engl. J. Med. 371:2200-6
-
(2014)
N. Engl. J. Med
, vol.371
, pp. 2200-2206
-
-
Gaudet, D.1
Brisson, D.2
Tremblay, K.3
Alexander, V.J.4
Singleton, W.5
-
91
-
-
84938401608
-
Antisense inhibition of apolipoprotein C-III in patients with hypertriglyceridemia
-
Gaudet D, Alexander VJ, Baker BF, Brisson D, Tremblay K, et al. 2015. Antisense inhibition of apolipoprotein C-III in patients with hypertriglyceridemia. N. Engl. J. Med. 373:438-47
-
(2015)
N. Engl. J. Med
, vol.373
, pp. 438-447
-
-
Gaudet, D.1
Alexander, V.J.2
Baker, B.F.3
Brisson, D.4
Tremblay, K.5
-
92
-
-
84920984860
-
Factor XI antisense oligonucleotide for prevention of venous thrombosis
-
Buller HR, Bethune C, Bhanot S, Gailani D, Monia BP, et al. 2015. Factor XI antisense oligonucleotide for prevention of venous thrombosis. N. Engl. J. Med. 372:232-40
-
(2015)
N. Engl. J. Med
, vol.372
, pp. 232-240
-
-
Buller, H.R.1
Bethune, C.2
Bhanot, S.3
Gailani, D.4
Monia, B.P.5
-
93
-
-
84859439270
-
Phase i clinical trial of Smad7 knockdown using antisense oligonucleotide in patients with active Crohn's disease
-
Monteleone G, Fantini MC, Onali S, Zorzi F, Sancesario G, et al. 2012. Phase I clinical trial of Smad7 knockdown using antisense oligonucleotide in patients with active Crohn's disease. Mol. Ther. 20:870-76
-
(2012)
Mol. Ther
, vol.20
, pp. 870-876
-
-
Monteleone, G.1
Fantini, M.C.2
Onali, S.3
Zorzi, F.4
Sancesario, G.5
-
94
-
-
84925064199
-
Mongersen, an oral SMAD7 antisense oligonucleotide, and Crohn's disease
-
Monteleone G, Neurath MF, Ardizzone S, Di Sabatino A, Fantini MC, et al. 2015. Mongersen, an oral SMAD7 antisense oligonucleotide, and Crohn's disease. N. Engl. J. Med. 372:1104-13
-
(2015)
N. Engl. J. Med
, vol.372
, pp. 1104-1113
-
-
Monteleone, G.1
Neurath, M.F.2
Ardizzone, S.3
Di Sabatino, A.4
Fantini, M.C.5
-
95
-
-
33846804162
-
A randomized, double-masked, placebo-controlled study of alicaforsen, an antisense inhibitor of intercellular adhesion molecule 1, for the treatment of subjects with active Crohn's disease
-
Yacyshyn BR, CheyWY, WedelMK, Yu RZ, Paul D, Cheung E. 2007. A randomized, double-masked, placebo-controlled study of alicaforsen, an antisense inhibitor of intercellular adhesion molecule 1, for the treatment of subjects with active Crohn's disease. Clin. Gastroenterol. Hepatol. 5:215-20
-
(2007)
Clin. Gastroenterol. Hepatol
, vol.5
, pp. 215-220
-
-
Yacyshyn, B.R.1
Chey, W.Y.2
Wedel, M.K.3
Yu, R.Z.4
Paul, D.5
Cheung, E.6
-
96
-
-
1342331543
-
Anenema formulation of alicaforsen, an antisense inhibitor of intercellular adhesion molecule-1, in the treatment of chronic, unremitting pouchitis
-
Miner P, Wedel M, Bane B, Bradley J. 2004.Anenema formulation of alicaforsen, an antisense inhibitor of intercellular adhesion molecule-1, in the treatment of chronic, unremitting pouchitis. Aliment. Pharmacol. Ther. 19:281-86
-
(2004)
Aliment. Pharmacol. Ther
, vol.19
, pp. 281-286
-
-
Miner, P.1
Wedel, M.2
Bane, B.3
Bradley, J.4
-
97
-
-
33646232502
-
Safety and efficacy of two dose formulations of alicaforsen enema compared with mesalazine enema for treatment of mild to moderate left-sided ulcerative colitis: A randomized, double-blind, active-controlled trial
-
Miner PB Jr., Wedel MK, Xia S, Baker BF. 2006. Safety and efficacy of two dose formulations of alicaforsen enema compared with mesalazine enema for treatment of mild to moderate left-sided ulcerative colitis: a randomized, double-blind, active-controlled trial. Aliment. Pharmacol. Ther. 23:1403-13
-
(2006)
Aliment. Pharmacol. Ther
, vol.23
, pp. 1403-1413
-
-
Miner, P.B.1
Wedel, M.K.2
Xia, S.3
Baker, B.F.4
-
98
-
-
33646271377
-
A Phase II dose ranging, double-blind, placebo-controlled study of alicaforsen enema in subjects with acute exacerbation of mild to moderate left-sided ulcerative colitis
-
Van Deventer SJH, Wedel MK, Baker BF, Xia S, Chuang E, Miner PB Jr. 2006. A Phase II dose ranging, double-blind, placebo-controlled study of alicaforsen enema in subjects with acute exacerbation of mild to moderate left-sided ulcerative colitis. Aliment. Pharmacol. Ther. 23:1415-25
-
(2006)
Aliment. Pharmacol. Ther
, vol.23
, pp. 1415-1425
-
-
Van Deventer, S.J.H.1
Wedel, M.K.2
Baker, B.F.3
Xia, S.4
Chuang, E.5
Miner, P.B.6
-
99
-
-
84979462428
-
Alicaforsen, an antisense inhibitor of ICAM-1, as treatment for chronic refractory pouchitis after proctocolectomy: A case series
-
Greuter T, Biedermann L, Rogler G, Sauter B, Seibold F. 2016. Alicaforsen, an antisense inhibitor of ICAM-1, as treatment for chronic refractory pouchitis after proctocolectomy: a case series. United Eur. Gastroenterol. J. 4:97-104
-
(2016)
United Eur. Gastroenterol. J
, vol.4
, pp. 97-104
-
-
Greuter, T.1
Biedermann, L.2
Rogler, G.3
Sauter, B.4
Seibold, F.5
-
100
-
-
0026874337
-
Systemic human antisense therapy begins
-
Bayever E, Iversen P, Smith S, Spinolo J, Zon G. 1992. Systemic human antisense therapy begins. Antisense Res. Dev. 2:109-10
-
(1992)
Antisense Res. Dev
, vol.2
, pp. 109-110
-
-
Bayever, E.1
Iversen, P.2
Smith, S.3
Spinolo, J.4
Zon, G.5
-
101
-
-
84868550507
-
Custirsen (OGX-011): A Second-Generation antisense inhibitor of clusterin in development for the treatment of prostate cancer
-
Zielinski R, Chi KN. 2012. Custirsen (OGX-011): a second-generation antisense inhibitor of clusterin in development for the treatment of prostate cancer. Future Oncol. 8:1239-51
-
(2012)
Future Oncol
, vol.8
, pp. 1239-1251
-
-
Zielinski, R.1
Chi, K.N.2
-
102
-
-
84947724597
-
AZD9150, a next-generation antisense oligonucleotide inhibitor of STAT3 with early evidence of clinical activity in lymphoma and lung cancer
-
Hong D, Kurzrock R, Kim Y, Woessner R, Younes A, et al. 2015. AZD9150, a next-generation antisense oligonucleotide inhibitor of STAT3 with early evidence of clinical activity in lymphoma and lung cancer. Sci. Transl. Med. 7:314-185
-
(2015)
Sci. Transl. Med
, vol.7
, pp. 185-314
-
-
Hong, D.1
Kurzrock, R.2
Kim, Y.3
Woessner, R.4
Younes, A.5
-
103
-
-
84927671644
-
Generation 2.5 antisense oligonucleotides targeting the androgen receptor and its splice variants suppress enzalutamide-resistant prostate cancer cell growth
-
Yamamoto Y, Loriot Y, Beraldi E, Zhang F, Wyatt AW, et al. 2015. Generation 2.5 antisense oligonucleotides targeting the androgen receptor and its splice variants suppress enzalutamide-resistant prostate cancer cell growth. Clin. Cancer Res. 21:1675-87
-
(2015)
Clin. Cancer Res
, vol.21
, pp. 1675-1687
-
-
Yamamoto, Y.1
Loriot, Y.2
Beraldi, E.3
Zhang, F.4
Wyatt, A.W.5
-
104
-
-
84943425235
-
Onset time and durability of huntingtin suppression in rhesus putamen after direct infusion of antihuntingtin siRNA
-
Grondin R, Ge P, Chen Q, Sutherland JE, Zhang Z, et al. 2015. Onset time and durability of huntingtin suppression in rhesus putamen after direct infusion of antihuntingtin siRNA. Mol. Ther. Nucleic Acids 4:e245
-
(2015)
Mol. Ther. Nucleic Acids
, vol.4
, pp. e245
-
-
Grondin, R.1
Ge, P.2
Chen, Q.3
Sutherland, J.E.4
Zhang, Z.5
-
105
-
-
77957877526
-
Synuclein suppression by targeted small interfering RNA in the primate substantia nigra
-
McCormack AL, Mak SK, Henderson JM, Bumcrot D, Farrer MJ, Di Monte DA. 2010.-Synuclein suppression by targeted small interfering RNA in the primate substantia nigra. PLOS ONE 5:e12122
-
(2010)
PLOS ONE
, vol.5
, pp. e12122
-
-
McCormack, A.L.1
Mak, S.K.2
Henderson, J.M.3
Bumcrot, D.4
Farrer, M.J.5
Di Monte, D.A.6
-
106
-
-
84904367458
-
Duchenne and Becker muscular dystrophies
-
Flanigan KM. 2014. Duchenne and Becker muscular dystrophies. Neurol. Clin. 32:671-88
-
(2014)
Neurol. Clin
, vol.32
, pp. 671-688
-
-
Flanigan, K.M.1
-
107
-
-
32244443828
-
Systemic delivery ofmorpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
-
Alter J, Lou F, Rabinowitz A, YinHF, Rosenfeld J, et al. 2006. Systemic delivery ofmorpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat.Med. 12:175-77
-
(2006)
Nat.Med
, vol.12
, pp. 175-177
-
-
Alter, J.1
Lou, F.2
Rabinowitz, A.3
Yinhf Rosenfeld, J.4
-
108
-
-
0037447517
-
Therapeutic Antisense-Induced exon skipping in cultured muscle cells from six different DMD patients
-
Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, den Dunnen JT, et al. 2003. Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum. Mol. Genet. 12:907-14
-
(2003)
Hum. Mol. Genet
, vol.12
, pp. 907-914
-
-
Aartsma-Rus, A.1
Janson, A.A.2
Kaman, W.E.3
Bremmer-Bout, M.4
Den Dunnen, J.T.5
-
109
-
-
34848904544
-
Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin premRNA splicing in human muscle
-
Arechavala-Gomeza V, Graham IR, Popplewell LJ, Adams AM, Aartsma-Rus A, et al. 2007. Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin premRNA splicing in human muscle. Hum. Gene Ther. 18:798-810
-
(2007)
Hum. Gene Ther
, vol.18
, pp. 798-810
-
-
Arechavala-Gomeza, V.1
Graham, I.R.2
Popplewell, L.J.3
Adams, A.M.4
Aartsma-Rus, A.5
-
110
-
-
79955158683
-
Systemic administration of PRO051 in Duchenne's muscular dystrophy
-
GoemansNM, TuliniusM, van den Akker JT, Burm BE, Ekhart PF, et al. 2011. Systemic administration of PRO051 in Duchenne's muscular dystrophy. N. Engl. J. Med. 364:1513-22
-
(2011)
N. Engl. J. Med
, vol.364
, pp. 1513-1522
-
-
Goemans, N.M.1
Tulinius, M.2
Vanden Akker, J.T.3
Burm, B.E.4
Ekhart, P.F.5
-
111
-
-
84890805770
-
Eteplirsen for the treatment of Duchenne muscular dystrophy
-
Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, et al. 2013. Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann. Neurol. 74:637-47
-
(2013)
Ann. Neurol
, vol.74
, pp. 637-647
-
-
Mendell, J.R.1
Rodino-Klapac, L.R.2
Sahenk, Z.3
Roush, K.4
Bird, L.5
-
112
-
-
84856546632
-
Restoration of the dystrophinassociated glycoprotein complex after exon skipping therapy inDuchenne muscular dystrophy
-
Cirak S, Feng L, AnthonyK, Arechavala-Gomeza V, Torelli S, et al. 2012. Restoration of the dystrophinassociated glycoprotein complex after exon skipping therapy inDuchenne muscular dystrophy. Mol. Ther. 20:462-67
-
(2012)
Mol. Ther
, vol.20
, pp. 462-467
-
-
Cirak, S.1
Feng, L.2
Anthony, K.3
Arechavala-Gomeza, V.4
Torelli, S.5
-
113
-
-
84958106352
-
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
-
Mendell JR, Goemans N, Lowes LP, Alfano LN, Berry K, et al. 2016. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Ann. Neurol. 79:257-71
-
(2016)
Ann. Neurol
, vol.79
, pp. 257-271
-
-
Mendell, J.R.1
Goemans, N.2
Lowes, L.P.3
Alfano, L.N.4
Berry, K.5
-
114
-
-
0026566108
-
Molecular basis of myotnic Dystrophy: Expansion of a trinucleotides (CTG) repeat at the 3-end of a transcript encoding a protein kinase family member
-
Brook JD, McCurrach ME, Harley HG, Buckler AJ, Church D, et al. 1992. Molecular basis of myotnic dystrophy: expansion of a trinucleotides (CTG) repeat at the 3-end of a transcript encoding a protein kinase family member. Cell 68:799-808
-
(1992)
Cell
, vol.68
, pp. 799-808
-
-
Brook, J.D.1
McCurrach, M.E.2
Harley, H.G.3
Buckler, A.J.4
Church, D.5
-
115
-
-
0034783271
-
Muscleblind localizes to nuclear foci of aberrant RNA in myotonic dystrophy types 1 and 2
-
Mankodi A, Urbinati CR, YuanQP, Moxley RT, Sansone V, et al. 2001. Muscleblind localizes to nuclear foci of aberrant RNA in myotonic dystrophy types 1 and 2. Hum. Mol. Genet. 10:2165-70
-
(2001)
Hum. Mol. Genet
, vol.10
, pp. 2165-2170
-
-
Mankodi, A.1
Urbinati, C.R.2
Yuan, Q.P.3
Moxley, R.T.4
Sansone, V.5
-
116
-
-
69549126597
-
Triplet-Repeat Oligonucleotide-Mediated reversal of RNA toxicity in myotonic dystrophy
-
Mulders SA, van den BroekWJ, Wheeler TM, Croes HJ, van Kuik-Romeijn P, et al. 2009. Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy. PNAS 106:13915-20
-
(2009)
PNAS
, vol.106
, pp. 13915-13920
-
-
Mulders, S.A.1
Vanden Broek, W.J.2
Wheeler, T.M.3
Croes, H.J.4
Van Kuik-Romeijn, P.5
-
117
-
-
67650828361
-
Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA
-
Wheeler TM, Sobczak K, Lueck JD, Osborn RJ, Lin X, et al. 2009. Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA. Science 325:336-39
-
(2009)
Science
, vol.325
, pp. 336-339
-
-
Wheeler, T.M.1
Sobczak, K.2
Lueck, J.D.3
Osborn, R.J.4
Lin, X.5
-
118
-
-
84864439453
-
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
-
Wheeler TM, Leger AJ, Pandey SK, MacLeod AR, Nakamori M, et al. 2012. Targeting nuclear RNA for in vivo correction of myotonic dystrophy. Nature 488:111-15
-
(2012)
Nature
, vol.488
, pp. 111-115
-
-
Wheeler, T.M.1
Leger, A.J.2
Pandey, S.K.3
MacLeod, A.R.4
Nakamori, M.5
-
119
-
-
84946543730
-
Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1
-
Pandey SK, Wheeler TM, Justice SL, Kim A, Younis HS, et al. 2015. Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1. J. Pharmacol. Exp. Ther. 355:329-40
-
(2015)
J. Pharmacol. Exp. Ther
, vol.355
, pp. 329-340
-
-
Pandey, S.K.1
Wheeler, T.M.2
Justice, S.L.3
Kim, A.4
Younis, H.S.5
-
120
-
-
84961062552
-
Diagnosis, prognosis, and therapy of transthyretin amyloidosis
-
Gertz MA, Benson MD, Dyck PJ, Grogan M, Coelho T, et al. 2015. Diagnosis, prognosis, and therapy of transthyretin amyloidosis. J. Am. Coll. Cardiol. 66:2451-66
-
(2015)
J. Am. Coll. Cardiol
, vol.66
, pp. 2451-2466
-
-
Gertz, M.A.1
Benson, M.D.2
Dyck, P.J.3
Grogan, M.4
Coelho, T.5
-
121
-
-
84883118140
-
Safety and efficacy of RNAi therapy for transthyretin amyloidosis
-
Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, et al. 2013. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N. Engl. J. Med. 369:819-29
-
(2013)
N. Engl. J. Med
, vol.369
, pp. 819-829
-
-
Coelho, T.1
Adams, D.2
Silva, A.3
Lozeron, P.4
Hawkins, P.N.5
-
122
-
-
84940759062
-
Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: A phase II multi-dose study
-
Suhr OB, Coelho T, Buades J, Pouget J, Conceicao I, et al. 2015. Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study. Orphanet J. Rare Dis. 10:109
-
(2015)
Orphanet J. Rare Dis
, vol.10
, pp. 109
-
-
Suhr, O.B.1
Coelho, T.2
Buades, J.3
Pouget, J.4
Conceicao, I.5
-
123
-
-
84990179844
-
Treatment of transthyretin (TTR) amyloid cardiomyopathy with an antisense oligonucleotide inhibitor of TTR synthesis
-
Benson MD, Ackermann EJ, Monia BP. 2015. Treatment of transthyretin (TTR) amyloid cardiomyopathy with an antisense oligonucleotide inhibitor of TTR synthesis. Orphanet J. Rare Dis. 10:7
-
(2015)
Orphanet J. Rare Dis
, vol.10
, pp. 7
-
-
Benson, M.D.1
Ackermann, E.J.2
Monia, B.P.3
-
124
-
-
0030981541
-
Correlation between severity and SMN protein level in spinal muscular atrophy
-
Lefebvre S, Burlet P, Liu Q, Bertrandy S, Clermont O, et al. 1997. Correlation between severity and SMN protein level in spinal muscular atrophy. Nat. Genet. 16:265-69
-
(1997)
Nat. Genet
, vol.16
, pp. 265-269
-
-
Lefebvre, S.1
Burlet, P.2
Liu, Q.3
Bertrandy, S.4
Clermont, O.5
-
125
-
-
79952348568
-
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
-
Passini MA, Bu J, Richards AM, Kinnecom C, Sardi SP, et al. 2011. Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy. Sci. Transl. Med. 3:72-18
-
(2011)
Sci. Transl. Med
, vol.3
, pp. 18-72
-
-
Passini, M.A.1
Bu, J.2
Richards, A.M.3
Kinnecom, C.4
Sardi, S.P.5
-
126
-
-
77955894067
-
Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
-
Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, et al. 2010. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev. 24:1634-44
-
(2010)
Genes Dev
, vol.24
, pp. 1634-1644
-
-
Hua, Y.1
Sahashi, K.2
Hung, G.3
Rigo, F.4
Passini, M.A.5
-
127
-
-
84969287518
-
Intrathecal injections in children with spinal muscular atrophy: Nusinersen clinical trial experience
-
Haché M, Swoboda KJ, Sethna N, Farrow-Gillespie A, Khandji A, et al. 2016. Intrathecal injections in children with spinal muscular atrophy: nusinersen clinical trial experience. J. Child Neurol. 31:899-906
-
(2016)
J. Child Neurol
, vol.31
, pp. 899-906
-
-
Haché, M.1
Swoboda, K.J.2
Sethna, N.3
Farrow-Gillespie, A.4
Khandji, A.5
-
128
-
-
85009088263
-
Nusinersen treatment of infantile-onset spinal muscular atrophy
-
In press
-
Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, et al. 2016. Nusinersen treatment of infantile-onset spinal muscular atrophy. Lancet. In press
-
(2016)
Lancet
-
-
Finkel, R.S.1
Chiriboga, C.A.2
Vajsar, J.3
Day, J.W.4
Montes, J.5
-
129
-
-
24644483623
-
Modulation of hepatitis C virus RNA abundance by a liver-specific microRNA
-
Jopling CL, Yi M, Lancaster AM, Lemon SM, Sarnow P. 2005. Modulation of hepatitis C virus RNA abundance by a liver-specific microRNA. Science 309:1577-81
-
(2005)
Science
, vol.309
, pp. 1577-1581
-
-
Jopling, C.L.1
Yi, M.2
Lancaster, A.M.3
Lemon, S.M.4
Sarnow, P.5
-
130
-
-
84873459086
-
Sensitive detection of hepatocellular injury in chronic hepatitisCpatients with circulating hepatocyte-derived microRNA-122
-
van der Meer AJ, FaridWR, SonneveldMJ, de Ruiter PE, Boonstra A, et al. 2013. Sensitive detection of hepatocellular injury in chronic hepatitisCpatients with circulating hepatocyte-derived microRNA-122. J. Viral Hepat. 20:158-66
-
(2013)
J. Viral Hepat
, vol.20
, pp. 158-166
-
-
Vander Meer, A.J.1
Farid, W.R.2
Sonneveld, M.J.3
De Ruiter, P.E.4
Boonstra, A.5
-
131
-
-
74249112787
-
Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection
-
Lanford RE, Hildebrandt-Eriksen ES, Petri A, Persson R, Lindow M, et al. 2010. Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection. Science 327:198-201
-
(2010)
Science
, vol.327
, pp. 198-201
-
-
Lanford, R.E.1
Hildebrandt-Eriksen, E.S.2
Petri, A.3
Persson, R.4
Lindow, M.5
-
132
-
-
84877258007
-
Treatment ofHCV infection by targeting microRNA
-
JanssenHLA, ReesinkHW, Lawitz EJ, Zeuzem S, Rodriguez-TorresM, et al. 2013. Treatment ofHCV infection by targeting microRNA. N. Engl. J. Med. 368:1685-94
-
(2013)
N. Engl. J. Med
, vol.368
, pp. 1685-1694
-
-
Janssen, H.L.A.1
Reesink, H.W.2
Lawitz, E.J.3
Zeuzem, S.4
Rodriguez-Torres, M.5
-
133
-
-
84907484544
-
Long-Term safety and efficacy of microRNA-targeted therapy in chronic hepatitis C patients
-
van der Ree MH, van der Meer AJ, de Bruijne J, Maan R, van Vliet A, et al. 2014. Long-term safety and efficacy of microRNA-targeted therapy in chronic hepatitis C patients. Antiviral Res. 111:53-59
-
(2014)
Antiviral Res
, vol.111
, pp. 53-59
-
-
Vander Ree, M.H.1
Vander Meer, A.J.2
De Bruijne, J.3
Maan, R.4
Van Vliet, A.5
-
134
-
-
85006071413
-
LO7: A single subcutaneous dose of 2 mg/kg or 4 mg/kg of RG-101, a GalNAc-conjugated oligonucleotide with antagonist activity against miR-122, results in significant viral load reductions in chronic hepatitis C patients
-
van der Meer AJ, de Vree ML, Stelma F, Willemse S, van der Valk M, et al. 2015. LO7: a single subcutaneous dose of 2 mg/kg or 4 mg/kg of RG-101, a GalNAc-conjugated oligonucleotide with antagonist activity against miR-122, results in significant viral load reductions in chronic hepatitis C patients. J. Hepatol. 62:S261
-
(2015)
J. Hepatol
, vol.62
, pp. S261
-
-
Vander Meer, A.J.1
De Vree, M.L.2
Stelma, F.3
Willemse, S.4
Vander Valk, M.5
-
135
-
-
84944152398
-
Antisense therapy targeting Apolipoprotein(a): A randomised, double-blind, placebo-controlled phase 1 study
-
Tsimikas S, Viney NJ, Hughes SG, SingletonW, Graham MJ, et al. 2015. Antisense therapy targeting apolipoprotein(a): a randomised, double-blind, placebo-controlled phase 1 study. Lancet 386:1472-83
-
(2015)
Lancet
, vol.386
, pp. 1472-1483
-
-
Tsimikas, S.1
Viney, N.J.2
Hughes, S.G.3
Singleton, W.4
Graham, M.J.5
-
136
-
-
37549034298
-
Local dystrophin restoration with antisense oligonucleotide PRO051
-
vanDeutekom JC, Janson AA, Ginjaar IB, FrankhuizenWS, Aartsma-Rus A, et al. 2007. Local dystrophin restoration with antisense oligonucleotide PRO051. N. Engl. J. Med. 357:2677-86
-
(2007)
N. Engl. J. Med
, vol.357
, pp. 2677-2686
-
-
Van Deutekom, J.C.1
Janson, A.A.2
Ginjaar, I.B.3
Frankhuizen, W.S.4
Aartsma-Rus, A.5
-
137
-
-
80052468423
-
Randomized phase II trial of Custirsen (OGX-011) in combination with docetaxel or mitoxantrone as second-line therapy in patients with metastatic castrate-resistant prostate cancer progressing after first-line docetaxel: CUOG trial P-06c
-
Saad F, Hotte S, North S, Eigl B, Chi K, et al. 2011. Randomized phase II trial of Custirsen (OGX-011) in combination with docetaxel or mitoxantrone as second-line therapy in patients with metastatic castrate-resistant prostate cancer progressing after first-line docetaxel: CUOG trial P-06c. Clin. Cancer Res. 17:5765-73
-
(2011)
Clin. Cancer Res
, vol.17
, pp. 5765-5773
-
-
Saad, F.1
Hotte, S.2
North, S.3
Eigl, B.4
Chi, K.5
-
138
-
-
38949153810
-
A phase i study of OGX-011, a 2-methoxyethyl phosphorothioate antisense to clusterin, in combination with docetaxel in patients with advanced cancer
-
Chi KN, Siu LL, Hirte H, Hotte SJ, Knox J, et al. 2008. A phase I study of OGX-011, a 2-methoxyethyl phosphorothioate antisense to clusterin, in combination with docetaxel in patients with advanced cancer. Clin. Cancer Res. 14:833-39
-
(2008)
Clin. Cancer Res
, vol.14
, pp. 833-839
-
-
Chi, K.N.1
Siu, L.L.2
Hirte, H.3
Hotte, S.J.4
Knox, J.5
-
139
-
-
84974845947
-
A phase i dose-escalation study of apatorsen (OGX-427), an antisense inhibitor targeting heat shock protein 27 (Hsp27), in patients with castration-resistant prostate cancer and other advanced cancers
-
Chi KN, Yu EY, Jacobs C, Bazov J, Kollmannsberger C, et al. 2016. A phase I dose-escalation study of apatorsen (OGX-427), an antisense inhibitor targeting heat shock protein 27 (Hsp27), in patients with castration-resistant prostate cancer and other advanced cancers. Ann. Oncol. 27:1116-22
-
(2016)
Ann. Oncol
, vol.27
, pp. 1116-1122
-
-
Chi, K.N.1
Yu, E.Y.2
Jacobs, C.3
Bazov, J.4
Kollmannsberger, C.5
-
140
-
-
84925283858
-
CD49d antisense drug ATL1102 reduces disease activity in patients with relapsing-remitting MS
-
Limmroth V, Barkhof F, Desem N, Diamond MP, Tachas G, ATL1102 Study Group. 2014. CD49d antisense drug ATL1102 reduces disease activity in patients with relapsing-remitting MS. Neurology 83:1780-88
-
(2014)
Neurology
, vol.83
, pp. 1780-1788
-
-
Limmroth, V.1
Barkhof, F.2
Desem, N.3
Diamond, M.P.4
Tachas, G.5
-
141
-
-
84929774279
-
Allergen-Induced asthmatic responses modified by a GATA3-specific DNAzyme
-
Krug N, Hohlfeld JM, Kirsten AM, Kornmann O, Beeh KM, et al. 2015. Allergen-induced asthmatic responses modified by a GATA3-specific DNAzyme. N. Engl. J. Med. 372:1987-95
-
(2015)
N. Engl. J. Med
, vol.372
, pp. 1987-1995
-
-
Krug, N.1
Hohlfeld, J.M.2
Kirsten, A.M.3
Kornmann, O.4
Beeh, K.M.5
-
142
-
-
84936972906
-
Synthetic RNAi triggers and their use in chronic hepatitis B therapies with curative intent
-
Gish RG, Yuen MF, Chan HL, Given BD, Lai CL, et al. 2015. Synthetic RNAi triggers and their use in chronic hepatitis B therapies with curative intent. Antivir. Res. 121:97-108
-
(2015)
Antivir. Res
, vol.121
, pp. 97-108
-
-
Gish, R.G.1
Yuen, M.F.2
Chan, H.L.3
Given, B.D.4
Lai, C.L.5
|