-
1
-
-
84903829672
-
Dynamic RNA modifications in disease
-
25005745
-
Klungland A, Dahl JA. Dynamic RNA modifications in disease. Curr Opin Genet Dev 2014; 26:47-52; PMID:25005745; http://dx.doi.org/10.1016/j.gde.2014.05.006
-
(2014)
Curr Opin Genet Dev
, vol.26
, pp. 47-52
-
-
Klungland, A.1
Dahl, J.A.2
-
2
-
-
84858446718
-
Regulation of cytoplasmic mRNA decay
-
22392217
-
Schoenberg DR, Maquat LE. Regulation of cytoplasmic mRNA decay. Nat Rev Genet 2012; 13:246-59; PMID:22392217; http://dx.doi.org/10.1038/nrg3254
-
(2012)
Nat Rev Genet
, vol.13
, pp. 246-259
-
-
Schoenberg, D.R.1
Maquat, L.E.2
-
3
-
-
84974615755
-
Defective control of pre-messenger RNA splicing in human disease
-
26728853
-
Chabot B, Shkreta L. Defective control of pre-messenger RNA splicing in human disease. J Cell Biol 2016; 212:13-27; PMID:26728853; http://dx.doi.org/10.1083/jcb.201510032
-
(2016)
J Cell Biol
, vol.212
, pp. 13-27
-
-
Chabot, B.1
Shkreta, L.2
-
4
-
-
34548052424
-
Translation matters: protein synthesis defects in inherited disease
-
17680008
-
Scheper GC, van der Knaap MS, Proud CG. Translation matters:protein synthesis defects in inherited disease. Nat Rev Genet 2007; 8:711-23; PMID:17680008; http://dx.doi.org/10.1038/nrg2142
-
(2007)
Nat Rev Genet
, vol.8
, pp. 711-723
-
-
Scheper, G.C.1
van der Knaap, M.S.2
Proud, C.G.3
-
5
-
-
84882589462
-
Mechanisms of RNA-induced toxicity in CAG repeat disorders
-
23907466
-
Nalavade R, Griesche N, Ryan DP, Hildebrand S, Krauss S. Mechanisms of RNA-induced toxicity in CAG repeat disorders. Cell Death Dis 2013; 4:e752; PMID:23907466; http://dx.doi.org/10.1038/cddis.2013.276
-
(2013)
Cell Death Dis
, vol.4
, pp. e752
-
-
Nalavade, R.1
Griesche, N.2
Ryan, D.P.3
Hildebrand, S.4
Krauss, S.5
-
6
-
-
77949512140
-
RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform
-
20055705
-
Bennett CF, Swayze EE. RNA targeting therapeutics:molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol 2010; 50:259-93; PMID:20055705; http://dx.doi.org/10.1146/annurev.pharmtox.010909.105654
-
(2010)
Annu Rev Pharmacol Toxicol
, vol.50
, pp. 259-293
-
-
Bennett, C.F.1
Swayze, E.E.2
-
7
-
-
84856431819
-
RNA therapeutics: beyond RNA interference and antisense oligonucleotides
-
22262036
-
Kole R, Krainer AR, Altman S. RNA therapeutics:beyond RNA interference and antisense oligonucleotides. Nat Rev Drug Discov 2012; 11:125-40; PMID:22262036; http:dx.doi.org/10.1038/nrd3625
-
(2012)
Nat Rev Drug Discov
, vol.11
, pp. 125-140
-
-
Kole, R.1
Krainer, A.R.2
Altman, S.3
-
8
-
-
67649271530
-
Antisense oligonucleotide pharmacokinetics and metabolism
-
19379126
-
Geary RS. Antisense oligonucleotide pharmacokinetics and metabolism. Expert Opin Drug Metab Toxicol 2009; 5:381-91; PMID:19379126; http://dx.doi.org/10.1517/17425250902877680
-
(2009)
Expert Opin Drug Metab Toxicol
, vol.5
, pp. 381-391
-
-
Geary, R.S.1
-
9
-
-
84923106217
-
Therapeutic genome editing: prospects and challenges
-
25654603
-
Cox DB, Platt RJ, Zhang F. Therapeutic genome editing:prospects and challenges. Nat Med 2015; 21:121-31; PMID:25654603; http://dx.doi.org/10.1038/nm.3793
-
(2015)
Nat Med
, vol.21
, pp. 121-131
-
-
Cox, D.B.1
Platt, R.J.2
Zhang, F.3
-
10
-
-
85018359650
-
-
Gene Therapy in Lung Disease CRC Press
-
Myra Stern and Eric WFW Alton. Use of Liposomes in the Treatment of Cystic Fibrosis. Gene Therapy in Lung Disease CRC Press, 2002; 383–396; http://dx.doi.org/10.3109/9780203908822-18
-
(2002)
Use of Liposomes in the Treatment of Cystic Fibrosis
-
-
-
11
-
-
84937764228
-
An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia
-
25849132
-
Sehgal A, Barros S, Ivanciu L, Cooley B, Qin J, Racie T, Hettinger J, Carioto M, Jiang Y, Brodsky J, et al. An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia. Nat Med 2015; 21:492-7; PMID:25849132; http://dx.doi.org/10.1038/nm.3847
-
(2015)
Nat Med
, vol.21
, pp. 492-497
-
-
Sehgal, A.1
Barros, S.2
Ivanciu, L.3
Cooley, B.4
Qin, J.5
Racie, T.6
Hettinger, J.7
Carioto, M.8
Jiang, Y.9
Brodsky, J.10
-
12
-
-
0032545933
-
Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
-
9486653
-
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; PMID:9486653; http://dx.doi.org/10.1038/35888
-
(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
-
13
-
-
2342427578
-
Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs
-
14960586
-
Wu H, Lima WF, Zhang H, Fan A, Sun H, Crooke ST. Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs. J Biol Chem 2004; 279:17181-9; PMID:14960586; http://dx.doi.org/10.1074/jbc.M311683200
-
(2004)
J Biol Chem
, vol.279
, pp. 17181-17189
-
-
Wu, H.1
Lima, W.F.2
Zhang, H.3
Fan, A.4
Sun, H.5
Crooke, S.T.6
-
14
-
-
0036202668
-
A randomized controlled clinical trial of intravitreous fomivirsen for treatment of newly diagnosed peripheral cytomegalovirus retinitis in patients with AIDS
-
11931780
-
Vitravene Study Group. A randomized controlled clinical trial of intravitreous fomivirsen for treatment of newly diagnosed peripheral cytomegalovirus retinitis in patients with AIDS. Am J Ophthalmol 2002; 133:467-74; PMID:11931780; http://dx.doi.org/10.1016/S0002-9394(02)01327-2
-
(2002)
Am J Ophthalmol
, vol.133
, pp. 467-474
-
-
-
15
-
-
0036211366
-
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
-
11931781
-
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; PMID:11931781; http://dx.doi.org/10.1016/S0002-9394(02)01326-0
-
(2002)
Am J Ophthalmol
, vol.133
, pp. 475-483
-
-
-
16
-
-
0036202847
-
Safety of intravitreous fomivirsen for treatment of cytomegalovirus retinitis in patients with AIDS
-
11931782
-
Vitravene Study Group. Safety of intravitreous fomivirsen for treatment of cytomegalovirus retinitis in patients with AIDS. Am J Ophthalmol 2002; 133:484-98; PMID:11931782; http://dx.doi.org/10.1016/S0002-9394(02)01332-6
-
(2002)
Am J Ophthalmol
, vol.133
, pp. 484-498
-
-
-
17
-
-
84979462428
-
Alicaforsen, an antisense inhibitor of ICAM-1, as treatment for chronic refractory pouchitis after proctocolectomy: A case series
-
26966529
-
Greuter T, Biedermann L, Rogler G, Sauter B, Seibold F. Alicaforsen, an antisense inhibitor of ICAM-1, as treatment for chronic refractory pouchitis after proctocolectomy:A case series. United European Gastroenterol J 2016; 4:97-104; PMID:26966529; http://dx.doi.org/10.1177/2050640615593681
-
(2016)
United European Gastroenterol J
, vol.4
, pp. 97-104
-
-
Greuter, T.1
Biedermann, L.2
Rogler, G.3
Sauter, B.4
Seibold, F.5
-
19
-
-
84860625079
-
Antiangiogenic activity of aganirsen in nonhuman primate and rodent models of retinal neovascular disease after topical administration
-
22323484
-
Cloutier F, Lawrence M, Goody R, Lamoureux S, Al-Mahmood S, Colin S, Ferry A, Conduzorgues JP, Hadri A, Cursiefen C, et al. Antiangiogenic activity of aganirsen in nonhuman primate and rodent models of retinal neovascular disease after topical administration. Invest Ophthalmol Vis Sci 2012; 53:1195-203; PMID:22323484; http://dx.doi.org/10.1167/iovs.11-9064
-
(2012)
Invest Ophthalmol Vis Sci
, vol.53
, pp. 1195-1203
-
-
Cloutier, F.1
Lawrence, M.2
Goody, R.3
Lamoureux, S.4
Al-Mahmood, S.5
Colin, S.6
Ferry, A.7
Conduzorgues, J.P.8
Hadri, A.9
Cursiefen, C.10
-
20
-
-
84906936272
-
Aganirsen antisense oligonucleotide eye drops inhibit keratitis-induced corneal neovascularization and reduce need for transplantation: the I-CAN study
-
24811963
-
Cursiefen C, Viaud E, Bock F, Geudelin B, Ferry A, Kadlecová P, Lévy M, Al Mahmood S, Colin S, Thorin E, et al. Aganirsen antisense oligonucleotide eye drops inhibit keratitis-induced corneal neovascularization and reduce need for transplantation:the I-CAN study. Ophthalmology 2014; 121:1683-92; PMID:24811963; http://dx.doi.org/10.1016/j.ophtha.2014.03.038
-
(2014)
Ophthalmology
, vol.121
, pp. 1683-1692
-
-
Cursiefen, C.1
Viaud, E.2
Bock, F.3
Geudelin, B.4
Ferry, A.5
Kadlecová, P.6
Lévy, M.7
Al Mahmood, S.8
Colin, S.9
Thorin, E.10
-
22
-
-
85020607808
-
-
Clinical Trial Phase II/III
-
The STRONG Study. (http://strong-nvg.com/the-study/). Clinical Trial Phase II/III
-
-
-
-
23
-
-
70350435062
-
Antitumor activity of a novel antisense oligonucleotide against Akt1
-
19693774
-
Yoon H, Kim DJ, Ahn EH, Gellert GC, Shay JW, Ahn CH, Lee YB. Antitumor activity of a novel antisense oligonucleotide against Akt1. J Cell Biochem 2009; 108:832-8; PMID:19693774; http://dx.doi.org/10.1002/jcb.22311
-
(2009)
J Cell Biochem
, vol.108
, pp. 832-838
-
-
Yoon, H.1
Kim, D.J.2
Ahn, E.H.3
Gellert, G.C.4
Shay, J.W.5
Ahn, C.H.6
Lee, Y.B.7
-
27
-
-
84903360498
-
Antisense therapies for cancer: bridging the pharmacogenomic divide
-
MacLeod AR. Antisense therapies for cancer:bridging the pharmacogenomic divide. Drug Discovery Today:Therapeutic Strategies 2013; 10:e157-e63
-
(2013)
Drug Discovery Today: Therapeutic Strategies
, vol.10
, pp. e157
-
-
MacLeod, A.R.1
-
28
-
-
0033053332
-
Crystal structure and improved antisense properties of 2′-O-(2-methoxyethyl)-RNA
-
Teplova M, Minasov G, Tereshko V, Inamati GB, Cook PD, Manoharan M, Egli M. Crystal structure and improved antisense properties of 2′-O-(2-methoxyethyl)-RNA. Nat Struct Mol Biol 1999; 6:535-9; PMID:10360355; http://dx.doi.org/10.1038/9304.
-
(1999)
Nat Struct Mol Biol
, vol.6
, pp. 535-539
-
-
Teplova, M.1
Minasov, G.2
Tereshko, V.3
Inamati, G.B.4
Cook, P.D.5
Manoharan, M.6
Egli, M.7
-
29
-
-
77949485460
-
Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial
-
Raal FJ, Santos RD, Blom DJ, Marais AD, Charng M-J, Cromwell WC, Lachmann RH, Gaudet D, Tan JL, Chasan-Taber S, et al. Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia:a randomised, double-blind, placebo-controlled trial. The Lancet 2010; 375:998-1006; http://dx.doi.org/10.1016/S0140-6736(10)60284-X
-
(2010)
The 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
Lachmann, R.H.7
Gaudet, D.8
Tan, J.L.9
Chasan-Taber, S.10
-
30
-
-
84868518061
-
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
-
23060426
-
Stein EA, Dufour R, Gagne C, Gaudet D, East C, Donovan JM, Chin W, Tribble DL, McGowan M. 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; PMID:23060426; http://dx.doi.org/10.1161/CIRCULATIONAHA.112.104125
-
(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
Chin, W.7
Tribble, D.L.8
McGowan, M.9
-
31
-
-
84924415690
-
Long-term efficacy and safety of mipomersen in patients with familial hypercholesterolaemia: 2-year interim results of an open-label extension
-
24366918
-
Santos RD, Duell PB, East C, Guyton JR, Moriarty PM, Chin W, Mittleman RS. Long-term efficacy and safety of mipomersen in patients with familial hypercholesterolaemia:2-year interim results of an open-label extension. Eur Heart J 2015; 36:566-75; PMID:24366918; http://dx.doi.org/10.1093/eurheartj/eht549
-
(2015)
Eur Heart J
, vol.36
, pp. 566-575
-
-
Santos, R.D.1
Duell, P.B.2
East, C.3
Guyton, J.R.4
Moriarty, P.M.5
Chin, W.6
Mittleman, R.S.7
-
32
-
-
84861451481
-
Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy
-
22494066
-
Ackermann EJ, Guo S, Booten S, Alvarado L, Benson M, Hughes S, Monia BP. Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy. Amyloid 2012; 19:43-4; PMID:22494066; http://dx.doi.org/10.3109/13506129.2012.673140
-
(2012)
Amyloid
, vol.19
, pp. 43-44
-
-
Ackermann, E.J.1
Guo, S.2
Booten, S.3
Alvarado, L.4
Benson, M.5
Hughes, S.6
Monia, B.P.7
-
35
-
-
85020580427
-
-
Ionis Pharmaceuticals Press Release. (http://ir.isispharm.com/phoenix.zhtml?c=222170&p=irol-newsArticle&ID2105651). 2015
-
(2015)
-
-
-
38
-
-
85020597027
-
-
OncoGenex Press Release. (http://ir.oncogenex.com/releasedetail.cfm?ReleaseID=933276). 2015
-
(2015)
-
-
-
39
-
-
84857913871
-
Phase I/II trial of custirsen (OGX-011), an inhibitor of clusterin, in combination with a gemcitabine and platinum regimen in patients with previously untreated advanced non-small cell lung cancer
-
22198426
-
Laskin JJ, Nicholas G, Lee C, Gitlitz B, Vincent M, Cormier Y, Stephenson J, Ung Y, Sanborn R, Pressnail B, et al. Phase I/II trial of custirsen (OGX-011), an inhibitor of clusterin, in combination with a gemcitabine and platinum regimen in patients with previously untreated advanced non-small cell lung cancer. J Thorac Oncol 2012; 7:579-86; PMID:22198426; http://dx.doi.org/10.1097/JTO.0b013e31823f459c
-
(2012)
J Thorac Oncol
, vol.7
, pp. 579-586
-
-
Laskin, J.J.1
Nicholas, G.2
Lee, C.3
Gitlitz, B.4
Vincent, M.5
Cormier, Y.6
Stephenson, J.7
Ung, Y.8
Sanborn, R.9
Pressnail, B.10
-
41
-
-
77949297395
-
Synthesis and biophysical evaluation of 2′, 4′-constrained 2′ O-methoxyethyl and 2′, 4′-constrained 2′ O-ethyl nucleic acid analogues
-
20136157
-
Seth PP, Vasquez G, Allerson CA, Berdeja A, Gaus H, Kinberger GA, Prakash TP, Migawa MT, Bhat B, Swayze EE. Synthesis and biophysical evaluation of 2′, 4′-constrained 2′ O-methoxyethyl and 2′, 4′-constrained 2′ O-ethyl nucleic acid analogues. J Org Chem 2010; 75:1569-81; PMID:20136157; http://dx.doi.org/10.1021/jo902560f
-
(2010)
J Org Chem
, vol.75
, pp. 1569-1581
-
-
Seth, P.P.1
Vasquez, G.2
Allerson, C.A.3
Berdeja, A.4
Gaus, H.5
Kinberger, G.A.6
Prakash, T.P.7
Migawa, M.T.8
Bhat, B.9
Swayze, E.E.10
-
42
-
-
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, Nemunaitis J, Fowler N, Zhou T, Schmidt J, Jo M, et al. AZD9150, a next-generation antisense oligonucleotide inhibitor of STAT3 with early evidence of clinical activity in lymphoma and lung cancer. Sci Translat Med 2015; 7:314ra185-314ra185; http://dx.doi.org/10.1126/scitranslmed.aac5272
-
(2015)
Sci Translat Med
, vol.7
, pp. 314ra185
-
-
Hong, D.1
Kurzrock, R.2
Kim, Y.3
Woessner, R.4
Younes, A.5
Nemunaitis, J.6
Fowler, N.7
Zhou, T.8
Schmidt, J.9
Jo, M.10
-
47
-
-
85020588442
-
-
Prakash PT, Seth PP, Swayze EE, Graham MJ. Compositions and methods for modulating apolipoprotein (a) expression (US. Patent No. Nine,181,550). 2015
-
(2015)
Compositions and methods for modulating apolipoprotein (a) expression (US. Patent No. Nine,181,550)
-
-
Prakash, P.T.1
Seth, P.P.2
Swayze, E.E.3
Graham, M.J.4
-
48
-
-
84918578407
-
Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing
-
25434769
-
Nair JK, Willoughby JL, Chan A, Charisse K, Alam MR, Wang Q, Hoekstra M, Kandasamy P, Kel'in AV, Milstein S, et al. Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing. J Am Chem Soc 2014; 136:16958-61; PMID:25434769; http://dx.doi.org/10.1021/ja505986a
-
(2014)
J Am Chem Soc
, vol.136
, pp. 16958-16961
-
-
Nair, J.K.1
Willoughby, J.L.2
Chan, A.3
Charisse, K.4
Alam, M.R.5
Wang, Q.6
Hoekstra, M.7
Kandasamy, P.8
Kel'in, A.V.9
Milstein, S.10
-
49
-
-
84905584822
-
Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice
-
24992960
-
Prakash TP, Graham MJ, Yu J, Carty R, Low A, Chappell A, Schmidt K, Zhao C, Aghajan M, Murray HF, et al. Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice. Nucleic Acids Res 2014; 42:8796-807; PMID:24992960; http://dx.doi.org/10.1093/nar/gku531
-
(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
Chappell, A.6
Schmidt, K.7
Zhao, C.8
Aghajan, M.9
Murray, H.F.10
-
50
-
-
85020600303
-
-
Ionis Pharmaceuticals Press Release. (http://ir.isispharm.com/phoenix.zhtml?c=222170&p=irol-newsArticle&ID=2110180) 2015
-
(2015)
-
-
-
52
-
-
0023614188
-
Dystrophin: the protein product of the Duchenne muscular dystrophy locus
-
3319190
-
Hoffman EP, Brown RH, Kunkel LM. Dystrophin:the protein product of the Duchenne muscular dystrophy locus. Cell 1987; 51:919-28; PMID:3319190; http://dx.doi.org/10.1016/0092-8674(87)90579-4
-
(1987)
Cell
, vol.51
, pp. 919-928
-
-
Hoffman, E.P.1
Brown, R.H.2
Kunkel, L.M.3
-
53
-
-
61649097962
-
Theoretic applicability of antisense‐mediated exon skipping for Duchenne muscular dystrophy mutations
-
19156838
-
Aartsma‐Rus A, Fokkema I, Verschuuren J, Ginjaar I, van Deutekom J, van Ommen GJ, den Dunnen JT. Theoretic applicability of antisense‐mediated exon skipping for Duchenne muscular dystrophy mutations. Hum Mutat 2009; 30:293-9; PMID:19156838; http://dx.doi.org/10.1002/humu.20918
-
(2009)
Hum Mutat
, vol.30
, pp. 293-299
-
-
Aartsma‐Rus, A.1
Fokkema, I.2
Verschuuren, J.3
Ginjaar, I.4
van Deutekom, J.5
van Ommen, G.J.6
den Dunnen, J.T.7
-
54
-
-
0024466501
-
The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion
-
2491009
-
Koenig M, Beggs A, Moyer M, Scherpf S, Heindrich K, Bettecken T, Meng G, Müller CR, Lindlöf M, Kaariainen H, et al. The molecular basis for Duchenne versus Becker muscular dystrophy:correlation of severity with type of deletion. Am J Hum Genet 1989; 45:498; PMID:2491009
-
(1989)
Am J Hum Genet
, vol.45
, pp. 498
-
-
Koenig, M.1
Beggs, A.2
Moyer, M.3
Scherpf, S.4
Heindrich, K.5
Bettecken, T.6
Meng, G.7
Müller, C.R.8
Lindlöf, M.9
Kaariainen, H.10
-
55
-
-
84907991204
-
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study
-
25209738
-
Voit T, Topaloglu H, Straub V, Muntoni F, Deconinck N, Campion G, De Kimpe SJ, Eagle M, Guglieri M, Hood S, et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II):an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol 2014; 13:987-96; PMID:25209738; http://dx.doi.org/10.1016/S1474-4422(14)70195-4
-
(2014)
Lancet Neurol
, vol.13
, pp. 987-996
-
-
Voit, T.1
Topaloglu, H.2
Straub, V.3
Muntoni, F.4
Deconinck, N.5
Campion, G.6
De Kimpe, S.J.7
Eagle, M.8
Guglieri, M.9
Hood, S.10
-
57
-
-
85020609233
-
-
Clinical Trial Phase IIIB
-
Extension Study of Drisapersen in DMD Subjects. (2015). Clinical Trial Phase IIIB
-
(2015)
-
-
-
58
-
-
84890805770
-
Eteplirsen for the treatment of Duchenne muscular dystrophy
-
23907995
-
Mendell JR, Rodino‐Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, et al. Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol 2013; 74:637-47; PMID:23907995; http://dx.doi.org/10.1002/ana.23982
-
(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
Lowes, L.P.6
Alfano, L.7
Gomez, A.M.8
Lewis, S.9
Kota, J.10
-
59
-
-
85020591857
-
-
Clinical Trial Phase III
-
Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI). (2014). Clinical Trial Phase III
-
(2014)
-
-
-
60
-
-
85020577493
-
-
Drisapersen FDA Report. (http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/PeripheralandCentralNervousSystemDrugsAdvisoryCommittee/UCM473737.pdf). 2015
-
(2015)
-
-
-
61
-
-
85002678669
-
GP 251-Exon 53 skipping of the dystrophin gene in patients with Duchenne muscular dystrophy by systemic administration of NS-065/NCNP-01: A phase 1, dose escalation, first-in-human study
-
Komaki H, Nagata T, Saito T, Masuda S, Takeshita E, Tachimori H, et al. GP 251-Exon 53 skipping of the dystrophin gene in patients with Duchenne muscular dystrophy by systemic administration of NS-065/NCNP-01:A phase 1, dose escalation, first-in-human study. Neuromuscular Disorders 2015; 25:S261-S2; http://dx.doi.org/10.1016/j.nmd.2015.06.276
-
(2015)
Neuromuscular Disorders
, vol.25
, pp. S261
-
-
Komaki, H.1
Nagata, T.2
Saito, T.3
Masuda, S.4
Takeshita, E.5
Tachimori, H.6
-
62
-
-
85020597169
-
-
Nippon Shinyaku Press Release. (https://www.nippon-shinyaku.co.jp/english/news/?id=2920). 2016
-
(2016)
-
-
-
63
-
-
16544388608
-
2′-O, 4′-C-Ethylene-bridged nucleic acids (ENA) as next-generation antisense and antigene agents
-
15056846
-
Koizumi M. 2′-O, 4′-C-Ethylene-bridged nucleic acids (ENA) as next-generation antisense and antigene agents. Biol Pharm Bull 2004; 27:453-6; PMID:15056846; http://dx.doi.org/10.1248/bpb.27.453
-
(2004)
Biol Pharm Bull
, vol.27
, pp. 453-456
-
-
Koizumi, M.1
-
64
-
-
85020583534
-
-
Daiichi Sankyo Press Release. (http://www.daiichisankyo.com/media_investors/media_relations/press_releases/detail/006412/160225_635_E.pdf). 2016
-
(2016)
-
-
-
66
-
-
0028797783
-
Identification and characterization of a spinal muscular atrophy-determining gene
-
7813012
-
Lefebvre S, Bürglen L, Reboullet S, Clermont O, Burlet P, Viollet L, Benichou B, Cruaud C, Millasseau P, Zeviani M, et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell 1995; 80:155-65; PMID:7813012; http://dx.doi.org/10.1016/0092-8674(95)90460-3
-
(1995)
Cell
, vol.80
, pp. 155-165
-
-
Lefebvre, S.1
Bürglen, L.2
Reboullet, S.3
Clermont, O.4
Burlet, P.5
Viollet, L.6
Benichou, B.7
Cruaud, C.8
Millasseau, P.9
Zeviani, M.10
-
67
-
-
0033033434
-
A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy
-
Lorson CL, Hahnen E, Androphy EJ, Wirth B. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Proc Natl Acad Sci U S A 1999; 96:6307-11; PMID:103395583; http://dx.doi.org/10.1073/pnas.96.11.6307
-
(1999)
Proc Natl Acad Sci U S A
, vol.96
, pp. 6307-6311
-
-
Lorson, C.L.1
Hahnen, E.2
Androphy, E.J.3
Wirth, B.4
-
68
-
-
41549168514
-
Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice
-
18371932
-
Hua Y, Vickers TA, Okunola HL, Bennett CF, Krainer AR. Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice. Am J Hum Genet 2008; 82:834-48; PMID:18371932; http://dx.doi.org/10.1016/j.ajhg.2008.01.014
-
(2008)
Am J Hum Genet
, vol.82
, pp. 834-848
-
-
Hua, Y.1
Vickers, T.A.2
Okunola, H.L.3
Bennett, C.F.4
Krainer, A.R.5
-
69
-
-
85020609010
-
-
Ionis Pharmaceuticals. (http://ir.ionispharma.com/phoenix.zhtml?c=222170&p=irol-newsArticle&ID=2097778). 2015
-
(2015)
-
-
-
72
-
-
84904412647
-
Development of therapeutic splice-switching oligonucleotides
-
24826963
-
Disterer P, Kryczka A, Liu Y, Badi YE, Wong JJ, Owen JS, Khoo B. Development of therapeutic splice-switching oligonucleotides. Hum Gene Ther 2014; 25:587-98; PMID:24826963; http://dx.doi.org/10.1089/hum.2013.234
-
(2014)
Hum Gene Ther
, vol.25
, pp. 587-598
-
-
Disterer, P.1
Kryczka, A.2
Liu, Y.3
Badi, Y.E.4
Wong, J.J.5
Owen, J.S.6
Khoo, B.7
-
73
-
-
77954220072
-
Aptamers as therapeutics
-
20592747
-
Keefe AD, Pai S, Ellington A. Aptamers as therapeutics. Nat Rev Drug Discov 2010; 9:537-50; PMID:20592747; http://dx.doi.org/10.1038/nrd3141
-
(2010)
Nat Rev Drug Discov
, vol.9
, pp. 537-550
-
-
Keefe, A.D.1
Pai, S.2
Ellington, A.3
-
74
-
-
77649103704
-
Discovery and Development of Therapeutic Aptamers
-
20055704
-
Bouchard PR, Hutabarat RM, Thompson KM. Discovery and Development of Therapeutic Aptamers. Annu Rev Pharmacol Toxicol 2010; 50:237-57; PMID:20055704; http://dx.doi.org/10.1146/annurev.pharmtox.010909.105547
-
(2010)
Annu Rev Pharmacol Toxicol
, vol.50
, pp. 237-257
-
-
Bouchard, P.R.1
Hutabarat, R.M.2
Thompson, K.M.3
-
75
-
-
0032493654
-
2′-Fluoropyrimidine RNA-based aptamers to the 165-amino acid form of vascular endothelial growth factor (VEGF165). Inhibition of receptor binding and VEGF-induced vascular permeability through interactions requiring the exon 7-encoded domain
-
9685413
-
Ruckman J, Green LS, Beeson J, Waugh S, Gillette WL, Henninger DD, Claesson-Welsh L, Janjić N. 2′-Fluoropyrimidine RNA-based aptamers to the 165-amino acid form of vascular endothelial growth factor (VEGF165). Inhibition of receptor binding and VEGF-induced vascular permeability through interactions requiring the exon 7-encoded domain. J Biol Chem 1998; 273:20556-67; PMID:9685413; http://dx.doi.org/10.1074/jbc.273.32.20556
-
(1998)
J Biol Chem
, vol.273
, pp. 20556-20567
-
-
Ruckman, J.1
Green, L.S.2
Beeson, J.3
Waugh, S.4
Gillette, W.L.5
Henninger, D.D.6
Claesson-Welsh, L.7
Janjić, N.8
-
76
-
-
33645095226
-
Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease
-
16518379
-
Ng EWM, Shima DT, Calias P, Cunningham ET, Guyer DR, Adamis AP. Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease. Nat Rev Drug Discov 2006; 5:123-32; PMID:16518379; http://dx.doi.org/10.1038/nrd1955
-
(2006)
Nat Rev Drug Discov
, vol.5
, pp. 123-132
-
-
Ng, E.W.M.1
Shima, D.T.2
Calias, P.3
Cunningham, E.T.4
Guyer, D.R.5
Adamis, A.P.6
-
77
-
-
84959556076
-
Effect of the REG1 anticoagulation system versus bivalirudin on outcomes after percutaneous coronary intervention (REGULATE-PCI): a randomised clinical trial
-
26547100
-
Lincoff AM, Mehran R, Povsic TJ, Zelenkofske SL, Huang Z, Armstrong PW, Steg PG, Bode C, Cohen MG, Buller C, et al. Effect of the REG1 anticoagulation system versus bivalirudin on outcomes after percutaneous coronary intervention (REGULATE-PCI):a randomised clinical trial. Lancet 2016; 387:349-56; PMID:26547100; http://dx.doi.org/10.1016/S0140-6736(15)00515-2
-
(2016)
Lancet
, vol.387
, pp. 349-356
-
-
Lincoff, A.M.1
Mehran, R.2
Povsic, T.J.3
Zelenkofske, S.L.4
Huang, Z.5
Armstrong, P.W.6
Steg, P.G.7
Bode, C.8
Cohen, M.G.9
Buller, C.10
-
79
-
-
84877106579
-
Anti-PDGF combination therapy in neovascular age-related macular degeneration: results of a phase 2b study
-
Dugel PU. Anti-PDGF combination therapy in neovascular age-related macular degeneration:results of a phase 2b study. Retina Today 2013; 8:65-71
-
(2013)
Retina Today
, vol.8
, pp. 65-71
-
-
Dugel, P.U.1
-
82
-
-
82255172093
-
Complement factor 5 inhibition in age-related macular degeneration
-
Monés J. Complement factor 5 inhibition in age-related macular degeneration. Retina Today 2010; 5:52-5
-
(2010)
Retina Today
, vol.5
, pp. 52-55
-
-
Monés, J.1
-
84
-
-
84922431782
-
Turning mirror-image oligonucleotides into drugs: the evolution of Spiegelmer® therapeutics
-
25236655
-
Vater A, Klussmann S. Turning mirror-image oligonucleotides into drugs:the evolution of Spiegelmer® therapeutics. Drug Discovery Today 2015; 20:147-55; PMID:25236655; http://dx.doi.org/10.1016/j.drudis.2014.09.004
-
(2015)
Drug Discovery Today
, vol.20
, pp. 147-155
-
-
Vater, A.1
Klussmann, S.2
-
85
-
-
0027081579
-
Modeling, synthesis, and hybridization properties of (L)-ribonucleic acid
-
Ashley GW. Modeling, synthesis, and hybridization properties of (L)-ribonucleic acid. J Am Chem Soc 1992; 114:9731-6; http://dx.doi.org/10.1021/ja00051a001
-
(1992)
J Am Chem Soc
, vol.114
, pp. 9731-9736
-
-
Ashley, G.W.1
-
86
-
-
84908233244
-
Effect of the antihepcidin Spiegelmer lexaptepid on inflammation-induced decrease in serum iron in humans
-
25163699
-
van Eijk LT, John AS, Schwoebel F, Summo L, Vauléon S, Zöllner S, Laarakkers CM, Kox M, van der Hoeven JG, Swinkels DW, et al. Effect of the antihepcidin Spiegelmer lexaptepid on inflammation-induced decrease in serum iron in humans. Blood 2014; 124:2643-6; PMID:25163699; http://dx.doi.org/10.1182/blood-2014-03-559484
-
(2014)
Blood
, vol.124
, pp. 2643-2646
-
-
van Eijk, L.T.1
John, A.S.2
Schwoebel, F.3
Summo, L.4
Vauléon, S.5
Zöllner, S.6
Laarakkers, C.M.7
Kox, M.8
van der Hoeven, J.G.9
Swinkels, D.W.10
-
87
-
-
84908013351
-
SDF-1 inhibition targets the bone marrow niche for cancer therapy
-
25263552
-
Roccaro AM, Sacco A, Purschke WG, Moschetta M, Buchner K, Maasch C, Zboralski D, Zöllner S, Vonhoff S, Mishima Y, et al. SDF-1 inhibition targets the bone marrow niche for cancer therapy. Cell Rep 2014; 9:118-28; PMID:25263552; http://dx.doi.org/10.1016/j.celrep.2014.08.042
-
(2014)
Cell Rep
, vol.9
, pp. 118-128
-
-
Roccaro, A.M.1
Sacco, A.2
Purschke, W.G.3
Moschetta, M.4
Buchner, K.5
Maasch, C.6
Zboralski, D.7
Zöllner, S.8
Vonhoff, S.9
Mishima, Y.10
-
90
-
-
84928789604
-
Crystal structure of a mirror-image L-RNA aptamer (Spiegelmer) in complex with the natural L-protein target CCL2
-
25901662
-
Oberthür D, Achenbach J, Gabdulkhakov A, Buchner K, Maasch C, Falke S, Rehders D, Klussmann S, Betzel C. Crystal structure of a mirror-image L-RNA aptamer (Spiegelmer) in complex with the natural L-protein target CCL2. Nat Commun 2015; 6:6923; PMID:25901662; http://dx.doi.org/10.1038/ncomms7923
-
(2015)
Nat Commun
, vol.6
, pp. 6923
-
-
Oberthür, D.1
Achenbach, J.2
Gabdulkhakov, A.3
Buchner, K.4
Maasch, C.5
Falke, S.6
Rehders, D.7
Klussmann, S.8
Betzel, C.9
-
93
-
-
38649111931
-
In vitro reconstitution of the human RISC-loading complex
-
18178619
-
MacRae IJ, Ma E, Zhou M, Robinson CV, Doudna JA. In vitro reconstitution of the human RISC-loading complex. Proc Natl Acad Sci U S A 2008; 105:512-7; PMID:18178619; http://dx.doi.org/10.1073/pnas.0710869105
-
(2008)
Proc Natl Acad Sci U S A
, vol.105
, pp. 512-517
-
-
MacRae, I.J.1
Ma, E.2
Zhou, M.3
Robinson, C.V.4
Doudna, J.A.5
-
94
-
-
84907405309
-
Mechanisms of miRNA-Mediated Gene Regulation from Common Downregulation to mRNA-Specific Upregulation
-
25180174
-
Valinezhad Orang A, Safaralizadeh R, Kazemzadeh-Bavili M. Mechanisms of miRNA-Mediated Gene Regulation from Common Downregulation to mRNA-Specific Upregulation. Int J Genomics 2014; 2014:970607; PMID:25180174
-
(2014)
Int J Genomics
, vol.2014
, pp. 970607
-
-
Valinezhad Orang, A.1
Safaralizadeh, R.2
Kazemzadeh-Bavili, M.3
-
95
-
-
0035942736
-
Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
-
11373684
-
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; PMID:11373684; http://dx.doi.org/10.1038/35078107
-
(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
-
96
-
-
0036806941
-
Spatial and temporal ‘knock down’ of gene expression by electroporation of double-stranded RNA and morpholinos into early postimplantation mouse embryos
-
12351170
-
Mellitzer G, Hallonet M, Chen L, Ang SL. Spatial and temporal ‘knock down’ of gene expression by electroporation of double-stranded RNA and morpholinos into early postimplantation mouse embryos. Mech Dev 2002; 118:57-63; PMID:12351170; http://dx.doi.org/10.1016/S0925-4773(02)00191-0
-
(2002)
Mech Dev
, vol.118
, pp. 57-63
-
-
Mellitzer, G.1
Hallonet, M.2
Chen, L.3
Ang, S.L.4
-
97
-
-
0037195064
-
Tissue-specific RNA interference in postimplantation mouse embryos with endoribonuclease-prepared short interfering RNA
-
12391321
-
Calegari F, Haubensak W, Yang D, Huttner WB, Buchholz F. Tissue-specific RNA interference in postimplantation mouse embryos with endoribonuclease-prepared short interfering RNA. Proc Natl Acad Sci U S A 2002; 99:14236-40; PMID:12391321; http://dx.doi.org/10.1073/pnas.192559699
-
(2002)
Proc Natl Acad Sci U S A
, vol.99
, pp. 14236-14240
-
-
Calegari, F.1
Haubensak, W.2
Yang, D.3
Huttner, W.B.4
Buchholz, F.5
-
98
-
-
84897577720
-
Big pharma shows signs of renewed interest in RNAi drugs
-
24504395
-
Zeliadt N. Big pharma shows signs of renewed interest in RNAi drugs. Nat Med 2014; 20:109; PMID:24504395; http://dx.doi.org/10.1038/nm0214-109
-
(2014)
Nat Med
, vol.20
, pp. 109
-
-
Zeliadt, N.1
-
105
-
-
84924625044
-
Are RNAi and miRNA therapeutics truly dead?
-
25595555
-
Conde J, Artzi N. Are RNAi and miRNA therapeutics truly dead? Trends Biotechnol 2015; 33:141-4; PMID:25595555; http://dx.doi.org/10.1016/j.tibtech.2014.12.005
-
(2015)
Trends Biotechnol
, vol.33
, pp. 141-144
-
-
Conde, J.1
Artzi, N.2
-
106
-
-
79957928508
-
Is RNAi dead?
-
21629254
-
Krieg AM. Is RNAi dead? Mol Ther 2011; 19:1001-2; PMID:21629254; http://dx.doi.org/10.1038/mt.2011.94
-
(2011)
Mol Ther
, vol.19
, pp. 1001-1002
-
-
Krieg, A.M.1
-
107
-
-
84883118140
-
Safety and efficacy of RNAi therapy for transthyretin amyloidosis
-
23984729
-
Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med 2013; 369:819-29; PMID:23984729; http://dx.doi.org/10.1056/NEJMoa1208760
-
(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
Mant, T.6
Perez, J.7
Chiesa, J.8
Warrington, S.9
Tranter, E.10
-
108
-
-
84892741671
-
Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial
-
24094767
-
Fitzgerald K, Frank-Kamenetsky M, Shulga-Morskaya S, Liebow A, Bettencourt BR, Sutherland JE, Hutabarat RM, Clausen VA, Karsten V, Cehelsky J, et al. Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers:a randomised, single-blind, placebo-controlled, phase 1 trial. Lancet 2014; 383:60-8; PMID:24094767; http://dx.doi.org/10.1016/S0140-6736(13)61914-5
-
(2014)
Lancet
, vol.383
, pp. 60-68
-
-
Fitzgerald, K.1
Frank-Kamenetsky, M.2
Shulga-Morskaya, S.3
Liebow, A.4
Bettencourt, B.R.5
Sutherland, J.E.6
Hutabarat, R.M.7
Clausen, V.A.8
Karsten, V.9
Cehelsky, J.10
-
109
-
-
33746070441
-
Intravenously administered short interfering RNA accumulates in the kidney and selectively suppresses gene function in renal proximal tubules
-
16714375
-
van de Water FM, Boerman OC, Wouterse AC, Peters JG, Russel FG, Masereeuw R. Intravenously administered short interfering RNA accumulates in the kidney and selectively suppresses gene function in renal proximal tubules. Drug Metab Dispos 2006; 34:1393-7; PMID:16714375; http://dx.doi.org/10.1124/dmd.106.009555
-
(2006)
Drug Metab Dispos
, vol.34
, pp. 1393-1397
-
-
van de Water, F.M.1
Boerman, O.C.2
Wouterse, A.C.3
Peters, J.G.4
Russel, F.G.5
Masereeuw, R.6
-
110
-
-
84959036971
-
-
Wiley interdisciplinary reviews RNA PMID:26840434
-
Alagia A, Eritja R. siRNA and RNAi optimization. Wiley interdisciplinary reviews RNA, 2016 7; 316-329; PMID:26840434; http://dx.doi.org/10.1002/wrna.1337
-
(2016)
siRNA and RNAi optimization
, vol.7
, pp. 316-329
-
-
Alagia, A.1
Eritja, R.2
-
111
-
-
84922724331
-
Going beyond the liver: progress and challenges of targeted delivery of siRNA therapeutics
-
25660205
-
Lorenzer C, Dirin M, Winkler AM, Baumann V, Winkler J. Going beyond the liver:progress and challenges of targeted delivery of siRNA therapeutics. J Control Release 2015; 203:1-15; PMID:25660205; http://dx.doi.org/10.1016/j.jconrel.2015.02.003
-
(2015)
J Control Release
, vol.203
, pp. 1-15
-
-
Lorenzer, C.1
Dirin, M.2
Winkler, A.M.3
Baumann, V.4
Winkler, J.5
-
112
-
-
85015725036
-
Technological development of structural DNA/RNA-based RNAi systems and their applications
-
Jeong EH, Kim H, Jang B, Cho H, Ryu J, Kim B, Park Y, Kim J, Lee JB, Lee H. Technological development of structural DNA/RNA-based RNAi systems and their applications. Adv Drug Deliv Rev 2016; 107:29-43; PMID:26494399; http://dx.doi.org/10.1016/j.addr.2015.10.008
-
(2016)
Adv Drug Deliv Rev
-
-
Jeong, E.H.1
Kim, H.2
Jang, B.3
Cho, H.4
Ryu, J.5
Kim, B.6
Park, Y.7
Kim, J.8
Lee, J.B.9
Lee, H.10
-
113
-
-
84934435856
-
Engineering small interfering RNAs by strategic chemical modification
-
23027047
-
Bramsen JB, Kjems J. Engineering small interfering RNAs by strategic chemical modification. Methods Mol Biol 2013; 942:87-109; PMID:23027047; http://dx.doi.org/10.1007/978-1-62703-119-6_5
-
(2013)
Methods Mol Biol
, vol.942
, pp. 87-109
-
-
Bramsen, J.B.1
Kjems, J.2
-
123
-
-
84954099191
-
RNA Interference (RNAi)-Based Therapeutics: Delivering on the Promise?
-
26738473
-
Bobbin ML, Rossi JJ. RNA Interference (RNAi)-Based Therapeutics:Delivering on the Promise? Annu Rev Pharmacol Toxicol 2016; 56:103-22; PMID:26738473; http://dx.doi.org/10.1146/annurev-pharmtox-010715-103633
-
(2016)
Annu Rev Pharmacol Toxicol
, vol.56
, pp. 103-122
-
-
Bobbin, M.L.1
Rossi, J.J.2
-
124
-
-
84954441161
-
siRNA Versus miRNA as Therapeutics for Gene Silencing
-
26372022
-
Lam JK, Chow MY, Zhang Y, Leung SW. siRNA Versus miRNA as Therapeutics for Gene Silencing. Mol Ther Nucleic Acids 2015; 4:e252; PMID:26372022; http://dx.doi.org/10.1038/mtna.2015.23
-
(2015)
Mol Ther Nucleic Acids
, vol.4
, pp. e252
-
-
Lam, J.K.1
Chow, M.Y.2
Zhang, Y.3
Leung, S.W.4
-
126
-
-
84901604742
-
The role of microRNAs in hepatitis C virus replication and related liver diseases
-
24871972
-
Lee CH, Kim JH, Lee SW. The role of microRNAs in hepatitis C virus replication and related liver diseases. J Microbiol 2014; 52:445-51; PMID:24871972; http://dx.doi.org/10.1007/s12275-014-4267-x
-
(2014)
J Microbiol
, vol.52
, pp. 445-451
-
-
Lee, C.H.1
Kim, J.H.2
Lee, S.W.3
-
129
-
-
0025231388
-
Direct gene transfer into mouse muscle in vivo
-
1690918
-
Wolff JA, Malone RW, Williams P, Chong W, Acsadi G, Jani A, Felgner PL. Direct gene transfer into mouse muscle in vivo. Science 1990; 247:1465-8; PMID:1690918; http://dx.doi.org/10.1126/science.1690918
-
(1990)
Science
, vol.247
, pp. 1465-1468
-
-
Wolff, J.A.1
Malone, R.W.2
Williams, P.3
Chong, W.4
Acsadi, G.5
Jani, A.6
Felgner, P.L.7
-
130
-
-
0026522713
-
Reversal of diabetes insipidus in Brattleboro rats: intrahypothalamic injection of vasopressin mRNA
-
1546298
-
Jirikowski GF, Sanna PP, Maciejewski-Lenoir D, Bloom FE. Reversal of diabetes insipidus in Brattleboro rats:intrahypothalamic injection of vasopressin mRNA. Science 1992; 255:996-8; PMID:1546298; http://dx.doi.org/10.1126/science.1546298
-
(1992)
Science
, vol.255
, pp. 996-998
-
-
Jirikowski, G.F.1
Sanna, P.P.2
Maciejewski-Lenoir, D.3
Bloom, F.E.4
-
131
-
-
0343185886
-
Foreign nucleic acids as the stimulus to make interferon
-
13956740
-
Isaacs A, Cox RA, Rotem Z. Foreign nucleic acids as the stimulus to make interferon. Lancet 1963; 2:113-6; PMID:13956740; http://dx.doi.org/10.1016/S0140-6736(63)92585-6
-
(1963)
Lancet
, vol.2
, pp. 113-116
-
-
Isaacs, A.1
Cox, R.A.2
Rotem, Z.3
-
132
-
-
84921353914
-
mRNA transcript therapy
-
25359562
-
Weissman D. mRNA transcript therapy. Expert Rev Vaccines 2015; 14:265-81; PMID:25359562; http://dx.doi.org/10.1586/14760584.2015.973859
-
(2015)
Expert Rev Vaccines
, vol.14
, pp. 265-281
-
-
Weissman, D.1
-
133
-
-
0035909372
-
Recognition of double-stranded RNA and activation of NF-kappaB by Toll-like receptor 3
-
11607032
-
Alexopoulou L, Holt AC, Medzhitov R, Flavell RA. Recognition of double-stranded RNA and activation of NF-kappaB by Toll-like receptor 3. Nature 2001; 413:732-8; PMID:11607032; http://dx.doi.org/10.1038/35099560
-
(2001)
Nature
, vol.413
, pp. 732-738
-
-
Alexopoulou, L.1
Holt, A.C.2
Medzhitov, R.3
Flavell, R.A.4
-
134
-
-
77957242130
-
Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation
-
20457754
-
Anderson BR, Muramatsu H, Nallagatla SR, Bevilacqua PC, Sansing LH, Weissman D, Karikó K. Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation. Nucleic Acids Res 2010; 38:5884-92; PMID:20457754; http://dx.doi.org/10.1093/nar/gkq347
-
(2010)
Nucleic Acids Res
, vol.38
, pp. 5884-5892
-
-
Anderson, B.R.1
Muramatsu, H.2
Nallagatla, S.R.3
Bevilacqua, P.C.4
Sansing, L.H.5
Weissman, D.6
Karikó, K.7
-
135
-
-
54949112814
-
Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability
-
18797453
-
Kariko K, Muramatsu H, Welsh FA, Ludwig J, Kato H, Akira S, Weissman D. Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability. Mol Ther 2008; 16:1833-40; PMID:18797453; http://dx.doi.org/10.1038/mt.2008.200
-
(2008)
Mol Ther
, vol.16
, pp. 1833-1840
-
-
Kariko, K.1
Muramatsu, H.2
Welsh, F.A.3
Ludwig, J.4
Kato, H.5
Akira, S.6
Weissman, D.7
-
136
-
-
84878173821
-
Cytosolic sensing of viruses
-
23706667
-
Goubau D, Deddouche S, Reis e Sousa C. Cytosolic sensing of viruses. Immunity 2013; 38:855-69; PMID:23706667; http://dx.doi.org/10.1016/j.immuni.2013.05.007
-
(2013)
Immunity
, vol.38
, pp. 855-869
-
-
Goubau, D.1
Deddouche, S.2
Reis e Sousa, C.3
-
137
-
-
23844464444
-
Suppression of RNA recognition by Toll-like receptors: the impact of nucleoside modification and the evolutionary origin of RNA
-
16111635
-
Kariko K, Buckstein M, Ni H, Weissman D. Suppression of RNA recognition by Toll-like receptors:the impact of nucleoside modification and the evolutionary origin of RNA. Immunity 2005; 23:165-75; PMID:16111635; http://dx.doi.org/10.1016/j.immuni.2005.06.008
-
(2005)
Immunity
, vol.23
, pp. 165-175
-
-
Kariko, K.1
Buckstein, M.2
Ni, H.3
Weissman, D.4
-
138
-
-
79751497093
-
Expression of therapeutic proteins after delivery of chemically modified mRNA in mice
-
21217696
-
Kormann MS, Hasenpusch G, Aneja MK, Nica G, Flemmer AW, Herber-Jonat S, Huppmann M, Mays LE, Illenyi M, Schams A, et al. Expression of therapeutic proteins after delivery of chemically modified mRNA in mice. Nat Biotechnol 2011; 29:154-7; PMID:21217696; http://dx.doi.org/10.1038/nbt.1733
-
(2011)
Nat Biotechnol
, vol.29
, pp. 154-157
-
-
Kormann, M.S.1
Hasenpusch, G.2
Aneja, M.K.3
Nica, G.4
Flemmer, A.W.5
Herber-Jonat, S.6
Huppmann, M.7
Mays, L.E.8
Illenyi, M.9
Schams, A.10
-
139
-
-
79959284255
-
Converting nonsense codons into sense codons by targeted pseudouridylation
-
21677757
-
Karijolich J, Yu YT. Converting nonsense codons into sense codons by targeted pseudouridylation. Nature 2011; 474:395-8; PMID:21677757; http://dx.doi.org/10.1038/nature10165
-
(2011)
Nature
, vol.474
, pp. 395-398
-
-
Karijolich, J.1
Yu, Y.T.2
-
140
-
-
80355137755
-
Denaturing urea polyacrylamide gel electrophoresis (Urea PAGE)
-
19865070
-
Summer H, Gramer R, Droge P. Denaturing urea polyacrylamide gel electrophoresis (Urea PAGE). J Vis Exp 2009; PMID:19865070; http://dx.doi.org/10.3791/1485(2009)
-
(2009)
J Vis Exp
-
-
Summer, H.1
Gramer, R.2
Droge, P.3
-
141
-
-
82255194361
-
Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA
-
21890902
-
Kariko K, Muramatsu H, Ludwig J, Weissman D. Generating the optimal mRNA for therapy:HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA. Nucleic Acids Res 2011; 39:e142; PMID:21890902; http://dx.doi.org/10.1093/nar/gkr695
-
(2011)
Nucleic Acids Res
, vol.39
, pp. e142
-
-
Kariko, K.1
Muramatsu, H.2
Ludwig, J.3
Weissman, D.4
-
142
-
-
0029400164
-
Reverse 5' caps in RNAs made in vitro by phage RNA polymerases
-
8548660
-
Pasquinelli AE, Dahlberg JE, Lund E. Reverse 5' caps in RNAs made in vitro by phage RNA polymerases. RNA 1995; 1:957-67; PMID:8548660
-
(1995)
RNA
, vol.1
, pp. 957-967
-
-
Pasquinelli, A.E.1
Dahlberg, J.E.2
Lund, E.3
-
143
-
-
0034768443
-
Synthesis and properties of mRNAs containing the novel “anti-reverse” cap analogs 7-methyl(3'-O-methyl)GpppG and 7-methyl (3'-deoxy)GpppG
-
11680853
-
Stepinski J, Waddell C, Stolarski R, Darzynkiewicz E, Rhoads RE. Synthesis and properties of mRNAs containing the novel “anti-reverse” cap analogs 7-methyl(3'-O-methyl)GpppG and 7-methyl (3'-deoxy)GpppG. RNA 2001; 7:1486-95; PMID:11680853; http://dx.doi.org/10.1017.S1355838201014078
-
(2001)
RNA
, vol.7
, pp. 1486-1495
-
-
Stepinski, J.1
Waddell, C.2
Stolarski, R.3
Darzynkiewicz, E.4
Rhoads, R.E.5
-
144
-
-
34748856370
-
Phosphorothioate cap analogs stabilize mRNA and increase translational efficiency in mammalian cells
-
17720878
-
Grudzien-Nogalska E, Jemielity J, Kowalska J, Darzynkiewicz E, Rhoads RE. Phosphorothioate cap analogs stabilize mRNA and increase translational efficiency in mammalian cells. RNA 2007; 13:1745-55; PMID:17720878; http://dx.doi.org/10.1261/rna.701307
-
(2007)
RNA
, vol.13
, pp. 1745-1755
-
-
Grudzien-Nogalska, E.1
Jemielity, J.2
Kowalska, J.3
Darzynkiewicz, E.4
Rhoads, R.E.5
-
145
-
-
79952289327
-
Multiparameter RNA and codon optimization: a standardized tool to assess and enhance autologous mammalian gene expression
-
21408612
-
Fath S, Bauer AP, Liss M, Spriestersbach A, Maertens B, Hahn P, Ludwig C, Schäfer F, Graf M, Wagner R. Multiparameter RNA and codon optimization:a standardized tool to assess and enhance autologous mammalian gene expression. PLoS one 2011; 6:e17596; PMID:21408612; http://dx.doi.org/10.1371/journal.pone.0017596
-
(2011)
PLoS one
, vol.6
, pp. e17596
-
-
Fath, S.1
Bauer, A.P.2
Liss, M.3
Spriestersbach, A.4
Maertens, B.5
Hahn, P.6
Ludwig, C.7
Schäfer, F.8
Graf, M.9
Wagner, R.10
-
146
-
-
84860529490
-
UU/UA dinucleotide frequency reduction in coding regions results in increased mRNA stability and protein expression
-
22434136
-
Al-Saif M, Khabar KS. UU/UA dinucleotide frequency reduction in coding regions results in increased mRNA stability and protein expression. Mol Ther 2012; 20:954-9; PMID:22434136; http://dx.doi.org/10.1038/mt.2012.29
-
(2012)
Mol Ther
, vol.20
, pp. 954-959
-
-
Al-Saif, M.1
Khabar, K.S.2
-
147
-
-
84940653144
-
mRNA: Fulfilling the Promise of Gene Therapy
-
26321183
-
Weissman D, Kariko K. mRNA:Fulfilling the Promise of Gene Therapy. Mol Ther 2015; 23:1416-7; PMID:26321183; http://dx.doi.org/10.1038/mt.2015.138
-
(2015)
Mol Ther
, vol.23
, pp. 1416-1417
-
-
Weissman, D.1
Kariko, K.2
-
148
-
-
0028900073
-
The nonamer UUAUUUAUU is the key AU-rich sequence motif that mediates mRNA degradation
-
7891716
-
Zubiaga AM, Belasco JG, Greenberg ME. The nonamer UUAUUUAUU is the key AU-rich sequence motif that mediates mRNA degradation. Mol Cell Biol 1995; 15:2219-30; PMID:7891716; http://dx.doi.org/10.1128/MCB.15.4.2219
-
(1995)
Mol Cell Biol
, vol.15
, pp. 2219-2230
-
-
Zubiaga, A.M.1
Belasco, J.G.2
Greenberg, M.E.3
-
149
-
-
79958192752
-
Control of human β-globin mRNA stability and its impact on β-thalassemia phenotype
-
21357703
-
Peixeiro I, Silva AL, Romao L. Control of human β-globin mRNA stability and its impact on β-thalassemia phenotype. Haematologica 2011; 96:905-13; PMID:21357703; http://dx.doi.org/10.3324/haematol.2010.039206
-
(2011)
Haematologica
, vol.96
, pp. 905-913
-
-
Peixeiro, I.1
Silva, A.L.2
Romao, L.3
-
150
-
-
0038777186
-
Regulation of α-globin mRNA stability
-
12671183
-
Waggoner SA, Liebhaber SA. Regulation of α-globin mRNA stability. Exp Biol Med (Maywood) 2003; 228:387-95; PMID:12671183
-
(2003)
Exp Biol Med (Maywood)
, vol.228
, pp. 387-395
-
-
Waggoner, S.A.1
Liebhaber, S.A.2
-
151
-
-
80055011827
-
The structure and function of the rous sarcoma virus RNA stability element
-
21769913
-
Withers JB, Beemon KL. The structure and function of the rous sarcoma virus RNA stability element. J Cell Biochem 2011; 112:3085-92; PMID:21769913; http://dx.doi.org/10.1002/jcb.23272
-
(2011)
J Cell Biochem
, vol.112
, pp. 3085-3092
-
-
Withers, J.B.1
Beemon, K.L.2
-
152
-
-
0026038913
-
The cap and poly(A) tail function synergistically to regulate mRNA translational efficiency
-
1682219
-
Gallie DR. The cap and poly(A) tail function synergistically to regulate mRNA translational efficiency. Genes Dev 1991; 5:2108-16; PMID:1682219; http://dx.doi.org/10.1101/gad.5.11.2108
-
(1991)
Genes Dev
, vol.5
, pp. 2108-2116
-
-
Gallie, D.R.1
-
153
-
-
42949133831
-
In vivo and in vitro analysis of poly(A) length effects on mRNA translation
-
18369986
-
Peng J, Murray EL, Schoenberg DR. In vivo and in vitro analysis of poly(A) length effects on mRNA translation. Methods Mol Biol 2008; 419:215-30; PMID:18369986; http://dx.doi.org/10.1007/978-1-59745-033-1_15
-
(2008)
Methods Mol Biol
, vol.419
, pp. 215-230
-
-
Peng, J.1
Murray, E.L.2
Schoenberg, D.R.3
-
154
-
-
84958976320
-
Chemically modified RNA induces osteogenesis of stem cells and human tissue explants as well as accelerates bone healing in rats
-
26923361
-
Balmayor ER, Geiger JP, Aneja MK, Berezhanskyy T, Utzinger M, Mykhaylyk O, Rudolph C, Plank C. Chemically modified RNA induces osteogenesis of stem cells and human tissue explants as well as accelerates bone healing in rats. Biomaterials 2016; 87:131-46; PMID:26923361; http://dx.doi.org/10.1016/j.biomaterials.2016.02.018
-
(2016)
Biomaterials
, vol.87
, pp. 131-146
-
-
Balmayor, E.R.1
Geiger, J.P.2
Aneja, M.K.3
Berezhanskyy, T.4
Utzinger, M.5
Mykhaylyk, O.6
Rudolph, C.7
Plank, C.8
-
155
-
-
84943261199
-
Chemically modified RNA activated matrices enhance bone regeneration
-
26415855
-
Elangovan S, Khorsand B, Do AV, Hong L, Dewerth A, Kormann M, Ross RD, Sumner DR, Allamargot C, Salem AK. Chemically modified RNA activated matrices enhance bone regeneration. J Control Release 2015; 218:22-8; PMID:26415855; http://dx.doi.org/10.1016/j.jconrel.2015.09.050
-
(2015)
J Control Release
, vol.218
, pp. 22-28
-
-
Elangovan, S.1
Khorsand, B.2
Do, A.V.3
Hong, L.4
Dewerth, A.5
Kormann, M.6
Ross, R.D.7
Sumner, D.R.8
Allamargot, C.9
Salem, A.K.10
-
156
-
-
78651254192
-
Activation of pluripotency genes in human fibroblast cells by a novel mRNA based approach
-
21209933
-
Plews JR, Li J, Jones M, Moore HD, Mason C, Andrews PW, Na J. Activation of pluripotency genes in human fibroblast cells by a novel mRNA based approach. PloS one 2010; 5:e14397; PMID:21209933; http://dx.doi.org/10.1371/journal.pone.0014397
-
(2010)
PloS one
, vol.5
, pp. e14397
-
-
Plews, J.R.1
Li, J.2
Jones, M.3
Moore, H.D.4
Mason, C.5
Andrews, P.W.6
Na, J.7
-
157
-
-
77958536106
-
Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA
-
20888316
-
Warren L, Manos PD, Ahfeldt T, Loh YH, Li H, Lau F, Ebina W, Mandal PK, Smith ZD, Meissner A, et al. Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 2010; 7:618-30; PMID:20888316; http://dx.doi.org/10.1016/j.stem.2010.08.012
-
(2010)
Cell Stem Cell
, vol.7
, pp. 618-630
-
-
Warren, L.1
Manos, P.D.2
Ahfeldt, T.3
Loh, Y.H.4
Li, H.5
Lau, F.6
Ebina, W.7
Mandal, P.K.8
Smith, Z.D.9
Meissner, A.10
-
158
-
-
84875160871
-
Reprogramming human fibroblasts to pluripotency using modified mRNA
-
23429718
-
Mandal PK, Rossi DJ. Reprogramming human fibroblasts to pluripotency using modified mRNA. Nat Protoc 2013; 8:568-82; PMID:23429718; http://dx.doi.org/10.1038/nprot.2013.019
-
(2013)
Nat Protoc
, vol.8
, pp. 568-582
-
-
Mandal, P.K.1
Rossi, D.J.2
-
159
-
-
84964687203
-
Synthetically modified mRNA for efficient and fast human iPS cell generation and direct transdifferentiation to myoblasts
-
26449459
-
Preskey D, Allison TF, Jones M, Mamchaoui K, Unger C. Synthetically modified mRNA for efficient and fast human iPS cell generation and direct transdifferentiation to myoblasts. Biochem Biophys Res Commun 2015; PMID:26449459; http://dx.doi.org/10.1016/j.bbrc.2015.09.102
-
(2015)
Biochem Biophys Res Commun
-
-
Preskey, D.1
Allison, T.F.2
Jones, M.3
Mamchaoui, K.4
Unger, C.5
-
160
-
-
84903542476
-
Direct reprogramming of human fibroblasts to hepatocyte-like cells by synthetic modified mRNAs
-
24963715
-
Simeonov KP, Uppal H. Direct reprogramming of human fibroblasts to hepatocyte-like cells by synthetic modified mRNAs. PloS One 2014; 9:e100134; PMID:24963715; http://dx.doi.org/10.1371/journal.pone.0100134
-
(2014)
PloS One
, vol.9
, pp. e100134
-
-
Simeonov, K.P.1
Uppal, H.2
-
161
-
-
84860539841
-
Increased erythropoiesis in mice injected with submicrogram quantities of pseudouridine-containing mRNA encoding erythropoietin
-
22334017
-
Kariko K, Muramatsu H, Keller JM, Weissman D. Increased erythropoiesis in mice injected with submicrogram quantities of pseudouridine-containing mRNA encoding erythropoietin. Mol Ther 2012; 20:948-53; PMID:22334017; http://dx.doi.org/10.1038/mt.2012.7
-
(2012)
Mol Ther
, vol.20
, pp. 948-953
-
-
Kariko, K.1
Muramatsu, H.2
Keller, J.M.3
Weissman, D.4
-
162
-
-
84902095353
-
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
-
24681508
-
Yin H, Xue W, Chen S, Bogorad RL, Benedetti E, Grompe M, Koteliansky V, Sharp PA, Jacks T, Anderson DG. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat Biotechnol 2014; 32:551-3; PMID:24681508; http://dx.doi.org/10.1038/nbt.2884
-
(2014)
Nat Biotechnol
, vol.32
, pp. 551-553
-
-
Yin, H.1
Xue, W.2
Chen, S.3
Bogorad, R.L.4
Benedetti, E.5
Grompe, M.6
Koteliansky, V.7
Sharp, P.A.8
Jacks, T.9
Anderson, D.G.10
-
163
-
-
84941206117
-
Generation of gene-modified goats targeting MSTN and FGF5 via zygote injection of CRISPR/Cas9 system
-
26354037
-
Wang X, Yu H, Lei A, Zhou J, Zeng W, Zhu H, Niu Y, Shi B, Cai B, Liu J, et al. Generation of gene-modified goats targeting MSTN and FGF5 via zygote injection of CRISPR/Cas9 system. Sci Rep 2015; 5:13878; PMID:26354037; http://dx.doi.org/10.1038/srep13878
-
(2015)
Sci Rep
, vol.5
, pp. 13878
-
-
Wang, X.1
Yu, H.2
Lei, A.3
Zhou, J.4
Zeng, W.5
Zhu, H.6
Niu, Y.7
Shi, B.8
Cai, B.9
Liu, J.10
-
164
-
-
84953271131
-
Simple Genome Editing of Rodent Intact Embryos by Electroporation
-
26556280
-
Kaneko T, Mashimo T. Simple Genome Editing of Rodent Intact Embryos by Electroporation. PloS one 2015; 10:e0142755; PMID:26556280; http://dx.doi.org/10.1371/journal.pone.0142755
-
(2015)
PloS one
, vol.10
, pp. e0142755
-
-
Kaneko, T.1
Mashimo, T.2
-
165
-
-
84899659140
-
Direct gene disruption by TALENs in medaka embryos
-
24713411
-
Wang T, Hong Y. Direct gene disruption by TALENs in medaka embryos. Gene 2014; 543:28-33; PMID:24713411; http://dx.doi.org/10.1016/j.gene.2014.04.013
-
(2014)
Gene
, vol.543
, pp. 28-33
-
-
Wang, T.1
Hong, Y.2
-
166
-
-
84903083399
-
Production of apolipoprotein C-III knockout rabbits using zinc finger nucleases
-
24301055
-
Yang D, Zhang J, Xu J, Zhu T, Fan Y, Fan J, Chen YE. Production of apolipoprotein C-III knockout rabbits using zinc finger nucleases. J Vis Exp 2013; 81; e50957; PMID:24301055; http://dx.doi.org/10.3791/50957
-
(2013)
J Vis Exp
, pp. 81; e50957
-
-
Yang, D.1
Zhang, J.2
Xu, J.3
Zhu, T.4
Fan, Y.5
Fan, J.6
Chen, Y.E.7
-
167
-
-
79958695683
-
Efficient immunoglobulin gene disruption and targeted replacement in rabbit using zinc finger nucleases
-
21695153
-
Flisikowska T, Thorey IS, Offner S, Ros F, Lifke V, Zeitler B, Rottmann O, Vincent A, Zhang L, Jenkins S, et al. Efficient immunoglobulin gene disruption and targeted replacement in rabbit using zinc finger nucleases. PloS One 2011; 6:e21045; PMID:21695153; http://dx.doi.org/10.1371/journal.pone.0021045
-
(2011)
PloS One
, vol.6
, pp. e21045
-
-
Flisikowska, T.1
Thorey, I.S.2
Offner, S.3
Ros, F.4
Lifke, V.5
Zeitler, B.6
Rottmann, O.7
Vincent, A.8
Zhang, L.9
Jenkins, S.10
-
168
-
-
84899675856
-
Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases
-
24682287
-
Williams DA, Thrasher AJ. Concise review:lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases. Stem Cells Transl Med 2014; 3:636-42; PMID:24682287; http://dx.doi.org/10.5966/sctm.2013-0206
-
(2014)
Stem Cells Transl Med
, vol.3
, pp. 636-642
-
-
Williams, D.A.1
Thrasher, A.J.2
-
169
-
-
50549083102
-
The immunogenicity of adenovirus vectors limits the multispecificity of CD8 T-cell responses to vector-encoded transgenic antigens
-
18612271
-
Schirmbeck R, Reimann J, Kochanek S, Kreppel F. The immunogenicity of adenovirus vectors limits the multispecificity of CD8 T-cell responses to vector-encoded transgenic antigens. Mol Ther 2008; 16:1609-16; PMID:18612271; http://dx.doi.org/10.1038/mt.2008.141
-
(2008)
Mol Ther
, vol.16
, pp. 1609-1616
-
-
Schirmbeck, R.1
Reimann, J.2
Kochanek, S.3
Kreppel, F.4
-
170
-
-
84930947142
-
In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency
-
25985262
-
Mahiny AJ, Dewerth A, Mays LE, Alkhaled M, Mothes B, Malaeksefat E, Loretz B, Rottenberger J, Brosch DM, Reautschnig P, et al. In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency. Nat Biotechnol 2015; 33:584-6; PMID:25985262; http://dx.doi.or/10.1038/nbt.3241
-
(2015)
Nat Biotechnol
, vol.33
, pp. 584-586
-
-
Mahiny, A.J.1
Dewerth, A.2
Mays, L.E.3
Alkhaled, M.4
Mothes, B.5
Malaeksefat, E.6
Loretz, B.7
Rottenberger, J.8
Brosch, D.M.9
Reautschnig, P.10
-
171
-
-
79953117041
-
Evidence for the cure of HIV infection by CCR5Delta32/Delta32 stem cell transplantation
-
21148083
-
Allers K, Hutter G, Hofmann J, Loddenkemper C, Rieger K, Thiel E, Schneider T. Evidence for the cure of HIV infection by CCR5Delta32/Delta32 stem cell transplantation. Blood 2011; 117:2791-9; PMID:21148083; http://dx.doi.org/10.1182/blood-2010-09-309591
-
(2011)
Blood
, vol.117
, pp. 2791-2799
-
-
Allers, K.1
Hutter, G.2
Hofmann, J.3
Loddenkemper, C.4
Rieger, K.5
Thiel, E.6
Schneider, T.7
-
173
-
-
85007564263
-
High-throughput discovery of post-transcriptional cis-regulatory elements
-
26941072
-
Wissink EM, Fogarty EA, Grimson A. High-throughput discovery of post-transcriptional cis-regulatory elements. BMC Genomics 2016; 17:177; PMID:26941072; http://dx.doi.org/10.1186/s12864-016-2479-7
-
(2016)
BMC Genomics
, vol.17
, pp. 177
-
-
Wissink, E.M.1
Fogarty, E.A.2
Grimson, A.3
-
174
-
-
84862855875
-
Nucleic acid sensing at the interface between innate and adaptive immunity in vaccination
-
22728526
-
Desmet CJ, Ishii KJ. Nucleic acid sensing at the interface between innate and adaptive immunity in vaccination. Nat Rev Immunol 2012; 12:479-91; PMID:22728526; http://dx.doi.org/10.1038/nri3247
-
(2012)
Nat Rev Immunol
, vol.12
, pp. 479-491
-
-
Desmet, C.J.1
Ishii, K.J.2
-
175
-
-
84927639135
-
Innate immune pattern recognition: a cell biological perspective
-
25581309
-
Brubaker SW, Bonham KS, Zanoni I, Kagan JC. Innate immune pattern recognition:a cell biological perspective. Annu Rev Immunol 2015; 33:257-90; PMID:25581309; http://dx.doi.org/10.1146/annurev-immunol-032414-112240
-
(2015)
Annu Rev Immunol
, vol.33
, pp. 257-290
-
-
Brubaker, S.W.1
Bonham, K.S.2
Zanoni, I.3
Kagan, J.C.4
-
176
-
-
0034022264
-
The role of dendritic cells in the innate immune system
-
10758402
-
Clark GJ, Angel N, Kato M, Lopez JA, MacDonald K, Vuckovic S, Hart DN. The role of dendritic cells in the innate immune system. Microbes Infect 2000; 2:257-72; PMID:10758402; http://dx.doi.org/10.1016/S1286-4579(00)00302-6
-
(2000)
Microbes Infect
, vol.2
, pp. 257-272
-
-
Clark, G.J.1
Angel, N.2
Kato, M.3
Lopez, J.A.4
MacDonald, K.5
Vuckovic, S.6
Hart, D.N.7
-
177
-
-
84954509568
-
Dendritic Cell Immunotherapy for HIV-1 Infection Using Autologous HIV-1 RNA: A Randomized, Double-Blind, Placebo-Controlled Clinical Trial
-
26751016
-
Jacobson JM, Routy JP, Welles S, DeBenedette M, Tcherepanova I, Angel JB, Asmuth DM, Stein DK, Baril JG, McKellar M, et al. Dendritic Cell Immunotherapy for HIV-1 Infection Using Autologous HIV-1 RNA:A Randomized, Double-Blind, Placebo-Controlled Clinical Trial. J Acquir Immune Defic Syndr 2016; 72:31-8; PMID:26751016; http://dx.doi.org/10.1097/QAI.0000000000000926
-
(2016)
J Acquir Immune Defic Syndr
, vol.72
, pp. 31-38
-
-
Jacobson, J.M.1
Routy, J.P.2
Welles, S.3
DeBenedette, M.4
Tcherepanova, I.5
Angel, J.B.6
Asmuth, D.M.7
Stein, D.K.8
Baril, J.G.9
McKellar, M.10
-
178
-
-
84898490601
-
Allergens are not pathogens: why immunization against allergy differs from vaccination against infectious diseases
-
24280693
-
Weiss R, Scheiblhofer S, Thalhamer J. Allergens are not pathogens:why immunization against allergy differs from vaccination against infectious diseases. Hum Vaccin Immunother 2014; 10:703-7; PMID:24280693; http://dx.doi.org/10.4161/hv.27183
-
(2014)
Hum Vaccin Immunother
, vol.10
, pp. 703-707
-
-
Weiss, R.1
Scheiblhofer, S.2
Thalhamer, J.3
-
179
-
-
78449257794
-
Prophylactic mRNA vaccination against allergy
-
20856111
-
Weiss R, Scheiblhofer S, Roesler E, Ferreira F, Thalhamer J. Prophylactic mRNA vaccination against allergy. Curr Opin Allergy Clin Immunol 2010; 10:567-74; PMID:20856111; http://dx.doi.org/10.1097/ACI.0b013e32833fd5b6
-
(2010)
Curr Opin Allergy Clin Immunol
, vol.10
, pp. 567-574
-
-
Weiss, R.1
Scheiblhofer, S.2
Roesler, E.3
Ferreira, F.4
Thalhamer, J.5
-
180
-
-
78650640612
-
Messenger RNA-based vaccines with dual activity induce balanced TLR-7 dependent adaptive immune responses and provide antitumor activity
-
21150709
-
Fotin-Mleczek M, Duchardt KM, Lorenz C, Pfeiffer R, Ojkic-Zrna S, Probst J, Kallen KJ. Messenger RNA-based vaccines with dual activity induce balanced TLR-7 dependent adaptive immune responses and provide antitumor activity. J Immunother 2011; 34:1-15; PMID:21150709; http://dx.doi.org/10.1097/CJI.0b013e3181f7dbe8
-
(2011)
J Immunother
, vol.34
, pp. 1-15
-
-
Fotin-Mleczek, M.1
Duchardt, K.M.2
Lorenz, C.3
Pfeiffer, R.4
Ojkic-Zrna, S.5
Probst, J.6
Kallen, K.J.7
-
181
-
-
84906841012
-
mRNA-based vaccines synergize with radiation therapy to eradicate established tumors
-
25127546
-
Fotin-Mleczek M, Zanzinger K, Heidenreich R, Lorenz C, Kowalczyk A, Kallen KJ, et al. mRNA-based vaccines synergize with radiation therapy to eradicate established tumors. Radiat Oncol 2014; 9:180; PMID:25127546; http://dx.doi.org/10.1186/1748-717X-9-180
-
(2014)
Radiat Oncol
, vol.9
, pp. 180
-
-
Fotin-Mleczek, M.1
Zanzinger, K.2
Heidenreich, R.3
Lorenz, C.4
Kowalczyk, A.5
Kallen, K.J.6
-
182
-
-
84887989953
-
Personalized medicine in human space flight: using Omics based analyses to develop individualized countermeasures that enhance astronaut safety and performance
-
24273472
-
Schmidt MA, Goodwin TJ. Personalized medicine in human space flight:using Omics based analyses to develop individualized countermeasures that enhance astronaut safety and performance. Metabolomics 2013; 9:1134-56; PMID:24273472; http://dx.doi.org/10.1007/s11306-013-0556-3
-
(2013)
Metabolomics
, vol.9
, pp. 1134-1156
-
-
Schmidt, M.A.1
Goodwin, T.J.2
-
183
-
-
84870869040
-
Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection
-
23159882
-
Petsch B, Schnee M, Vogel AB, Lange E, Hoffmann B, Voss D, Schlake T, Thess A, Kallen KJ, Stitz L, et al. Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection. Nat Biotechnol 2012; 30:1210-6; PMID:23159882; http://dx.doi.org/10.1038/nbt.2436
-
(2012)
Nat Biotechnol
, vol.30
, pp. 1210-1216
-
-
Petsch, B.1
Schnee, M.2
Vogel, A.B.3
Lange, E.4
Hoffmann, B.5
Voss, D.6
Schlake, T.7
Thess, A.8
Kallen, K.J.9
Stitz, L.10
-
184
-
-
33750110852
-
Vaccination with messenger RNA
-
16988444
-
Pascolo S. Vaccination with messenger RNA. Methods Mol Med 2006; 127:23-40; PMID:16988444; htpp://dx.doi.org/10.1385/1-59745-168-1:23
-
(2006)
Methods Mol Med
, vol.127
, pp. 23-40
-
-
Pascolo, S.1
-
185
-
-
0033038501
-
Cationic lipid-mediated transfection of cells in culture requires mitotic activity
-
10435090
-
Mortimer I, Tam P, MacLachlan I, Graham RW, Saravolac EG, Joshi PB. Cationic lipid-mediated transfection of cells in culture requires mitotic activity. Gene Ther 1999; 6:403-11; PMID:10435090; http://dx.doi.org/10.1038/sj.gt.3300837
-
(1999)
Gene Ther
, vol.6
, pp. 403-411
-
-
Mortimer, I.1
Tam, P.2
MacLachlan, I.3
Graham, R.W.4
Saravolac, E.G.5
Joshi, P.B.6
-
186
-
-
84870890171
-
An mRNA vaccine for influenza
-
23222788
-
Wong SS, Webby RJ. An mRNA vaccine for influenza. Nat Biotechnol 2012; 30:1202-4; PMID:23222788; http://dx.doi.org/10.1038/nbt.2439
-
(2012)
Nat Biotechnol
, vol.30
, pp. 1202-1204
-
-
Wong, S.S.1
Webby, R.J.2
-
187
-
-
0033120558
-
Factors associated with the development of neonatal tolerance after the administration of a plasmid DNA vaccine
-
10201898
-
Ichino M, Mor G, Conover J, Weiss WR, Takeno M, Ishii KJ, Klinman DM. Factors associated with the development of neonatal tolerance after the administration of a plasmid DNA vaccine. J Immunol 1999; 162:3814-8; PMID:10201898
-
(1999)
J Immunol
, vol.162
, pp. 3814-3818
-
-
Ichino, M.1
Mor, G.2
Conover, J.3
Weiss, W.R.4
Takeno, M.5
Ishii, K.J.6
Klinman, D.M.7
-
188
-
-
0346751949
-
Cancer immunotherapy using RNA-loaded dendritic cells
-
14632740
-
Ponsaerts P, Van Tendeloo VF, Berneman ZN. Cancer immunotherapy using RNA-loaded dendritic cells. Clin Exp Immunol 2003; 134:378-84; PMID:14632740; http://dx.doi.org/10.1046/j.1365-2249.2003.02286.x
-
(2003)
Clin Exp Immunol
, vol.134
, pp. 378-384
-
-
Ponsaerts, P.1
Van Tendeloo, V.F.2
Berneman, Z.N.3
-
189
-
-
84883458285
-
Therapeutic vaccination against autologous cancer stem cells with mRNA-transfected dendritic cells in patients with glioblastoma
-
23817721
-
Vik-Mo EO, Nyakas M, Mikkelsen BV, Moe MC, Due-Tonnesen P, Suso EM, Sæbøe-Larssen S, Sandberg C, Brinchmann JE, Helseth E, et al. Therapeutic vaccination against autologous cancer stem cells with mRNA-transfected dendritic cells in patients with glioblastoma. Cancer Immunol Immunother 2013; 62:1499-509; PMID:23817721; http://dx.doi.org/10.1007/s00262-013-1453-3
-
(2013)
Cancer Immunol Immunother
, vol.62
, pp. 1499-1509
-
-
Vik-Mo, E.O.1
Nyakas, M.2
Mikkelsen, B.V.3
Moe, M.C.4
Due-Tonnesen, P.5
Suso, E.M.6
Sæbøe-Larssen, S.7
Sandberg, C.8
Brinchmann, J.E.9
Helseth, E.10
-
190
-
-
84857045791
-
mRNA-based dendritic cell vaccination induces potent antiviral T-cell responses in HIV-1-infected patients
-
22156965
-
Van Gulck E, Vlieghe E, Vekemans M, Van Tendeloo VF, Van De Velde A, Smits E, Anguille S, Cools N, Goossens H, Mertens L, et al. mRNA-based dendritic cell vaccination induces potent antiviral T-cell responses in HIV-1-infected patients. AIDS 2012; 26:F1-12; PMID:22156965; http://dx.doi.org/10.1097/QAD.0b013e32834f33e8
-
(2012)
AIDS
, vol.26
, pp. F1-F12
-
-
Van Gulck, E.1
Vlieghe, E.2
Vekemans, M.3
Van Tendeloo, V.F.4
Van De Velde, A.5
Smits, E.6
Anguille, S.7
Cools, N.8
Goossens, H.9
Mertens, L.10
-
191
-
-
84866945151
-
Vaccination with mRNA-electroporated dendritic cells induces robust tumor antigen-specific CD4+ and CD8+ T cells responses in stage III and IV melanoma patients
-
22896657
-
Aarntzen EH, Schreibelt G, Bol K, Lesterhuis WJ, Croockewit AJ, de Wilt JH, van Rossum MM, Blokx WA, Jacobs JF, Duiveman-de Boer T, et al. Vaccination with mRNA-electroporated dendritic cells induces robust tumor antigen-specific CD4+ and CD8+ T cells responses in stage III and IV melanoma patients. Clin Cancer Res 2012; 18:5460-70; PMID:22896657; http://dx.doi.org/10.1158/1078-0432.CCR-11-3368
-
(2012)
Clin Cancer Res
, vol.18
, pp. 5460-5470
-
-
Aarntzen, E.H.1
Schreibelt, G.2
Bol, K.3
Lesterhuis, W.J.4
Croockewit, A.J.5
de Wilt, J.H.6
van Rossum, M.M.7
Blokx, W.A.8
Jacobs, J.F.9
Duiveman-de Boer, T.10
-
192
-
-
79959769072
-
hTERT mRNA dendritic cell vaccination: complete response in a pancreatic cancer patient associated with response against several hTERT epitopes
-
21365467
-
Suso EM, Dueland S, Rasmussen AM, Vetrhus T, Aamdal S, Kvalheim G, Gaudernack G. hTERT mRNA dendritic cell vaccination:complete response in a pancreatic cancer patient associated with response against several hTERT epitopes. Cancer Immunol Immunother 2011; 60:809-18; PMID:21365467; http://dx.doi.org/10.1007/s00262-011-0991-9
-
(2011)
Cancer Immunol Immunother
, vol.60
, pp. 809-818
-
-
Suso, E.M.1
Dueland, S.2
Rasmussen, A.M.3
Vetrhus, T.4
Aamdal, S.5
Kvalheim, G.6
Gaudernack, G.7
-
193
-
-
77956384935
-
Induction of complete and molecular remissions in acute myeloid leukemia by Wilms' tumor 1 antigen-targeted dendritic cell vaccination
-
20631300
-
Van Tendeloo VF, Van de Velde A, Van Driessche A, Cools N, Anguille S, Ladell K, Gostick E, Vermeulen K, Pieters K, Nijs G, et al. Induction of complete and molecular remissions in acute myeloid leukemia by Wilms' tumor 1 antigen-targeted dendritic cell vaccination. Proc Natl Acad Sci U S A 2010; 107:13824-9; PMID:20631300; http://dx.doi.org/10.1073/pnas.1008051107
-
(2010)
Proc Natl Acad Sci U S A
, vol.107
, pp. 13824-13829
-
-
Van Tendeloo, V.F.1
Van de Velde, A.2
Van Driessche, A.3
Cools, N.4
Anguille, S.5
Ladell, K.6
Gostick, E.7
Vermeulen, K.8
Pieters, K.9
Nijs, G.10
-
194
-
-
73549125003
-
Immunologic activity and safety of autologous HIV RNA-electroporated dendritic cells in HIV-1 infected patients receiving antiretroviral therapy
-
19889582
-
Routy JP, Boulassel MR, Yassine-Diab B, Nicolette C, Healey D, Jain R, Landry C, Yegorov O, Tcherepanova I, Monesmith T, et al. Immunologic activity and safety of autologous HIV RNA-electroporated dendritic cells in HIV-1 infected patients receiving antiretroviral therapy. Clin Immunol 2010; 134:140-7; PMID:19889582; http://dx.doi.org/10.1016/j.clim.2009.09.009
-
(2010)
Clin Immunol
, vol.134
, pp. 140-147
-
-
Routy, J.P.1
Boulassel, M.R.2
Yassine-Diab, B.3
Nicolette, C.4
Healey, D.5
Jain, R.6
Landry, C.7
Yegorov, O.8
Tcherepanova, I.9
Monesmith, T.10
-
195
-
-
77958527547
-
Immunological response after therapeutic vaccination with WT1 mRNA-loaded dendritic cells in end-stage endometrial carcinoma
-
20944158
-
Coosemans A, Wolfl M, Berneman ZN, Van Tendeloo V, Vergote I, Amant F, Van Gool SW. Immunological response after therapeutic vaccination with WT1 mRNA-loaded dendritic cells in end-stage endometrial carcinoma. Anticancer Res 2010; 30:3709-14; PMID:20944158
-
(2010)
Anticancer Res
, vol.30
, pp. 3709-3714
-
-
Coosemans, A.1
Wolfl, M.2
Berneman, Z.N.3
Van Tendeloo, V.4
Vergote, I.5
Amant, F.6
Van Gool, S.W.7
-
197
-
-
84963629041
-
Phase II Study of Autologous Monocyte-Derived mRNA Electroporated Dendritic Cells (TriMixDC-MEL) Plus Ipilimumab in Patients With Pretreated Advanced Melanoma
-
26926680
-
Wilgenhof S, Corthals J, Heirman C, van Baren N, Lucas S, Kvistborg P, Thielemans K, Neyns B. Phase II Study of Autologous Monocyte-Derived mRNA Electroporated Dendritic Cells (TriMixDC-MEL) Plus Ipilimumab in Patients With Pretreated Advanced Melanoma. J Clin Oncol 2016; 34:1330-8; PMID:26926680; http://dx.doi.org/10.1200/JCO.2015.63.4121
-
(2016)
J Clin Oncol
, vol.34
, pp. 1330-1338
-
-
Wilgenhof, S.1
Corthals, J.2
Heirman, C.3
van Baren, N.4
Lucas, S.5
Kvistborg, P.6
Thielemans, K.7
Neyns, B.8
-
198
-
-
84940486922
-
Intranodal vaccination with mRNA-optimized dendritic cells in metastatic melanoma patients
-
26405571
-
Bol KF, Figdor CG, Aarntzen EH, Welzen ME, van Rossum MM, Blokx WA, van de Rakt MW, Scharenborg NM, de Boer AJ, Pots JM, et al. Intranodal vaccination with mRNA-optimized dendritic cells in metastatic melanoma patients. Oncoimmunology 2015; 4:e1019197; PMID:26405571; http://dx.doi.org/10.1080/2162402X.2015.1019197
-
(2015)
Oncoimmunology
, vol.4
, pp. e1019197
-
-
Bol, K.F.1
Figdor, C.G.2
Aarntzen, E.H.3
Welzen, M.E.4
van Rossum, M.M.5
Blokx, W.A.6
van de Rakt, M.W.7
Scharenborg, N.M.8
de Boer, A.J.9
Pots, J.M.10
-
203
-
-
67651152679
-
Direct injection of protamine-protected mRNA: results of a phase 1/2 vaccination trial in metastatic melanoma patients
-
19609242
-
Weide B, Pascolo S, Scheel B, Derhovanessian E, Pflugfelder A, Eigentler TK, Pawelec G, Hoerr I, Rammensee HG, Garbe C. Direct injection of protamine-protected mRNA:results of a phase 1/2 vaccination trial in metastatic melanoma patients. J Immunother 2009; 32:498-507; PMID:19609242; http://dx.doi.org/10.1097/CJI.0b013e3181a00068
-
(2009)
J Immunother
, vol.32
, pp. 498-507
-
-
Weide, B.1
Pascolo, S.2
Scheel, B.3
Derhovanessian, E.4
Pflugfelder, A.5
Eigentler, T.K.6
Pawelec, G.7
Hoerr, I.8
Rammensee, H.G.9
Garbe, C.10
-
205
-
-
84883230210
-
Rapidly produced SAM((R)) vaccine against H7N9 influenza is immunogenic in mice
-
26038486
-
Hekele A, Bertholet S, Archer J, Gibson DG, Palladino G, Brito LA, Otten GR, Brazzoli M, Buccato S, Bonci A, et al. Rapidly produced SAM((R)) vaccine against H7N9 influenza is immunogenic in mice. Emerg Microbes Infect 2013; 2:e52; PMID:26038486; http://dx.doi.org/10.1038/emi.2013.54
-
(2013)
Emerg Microbes Infect
, vol.2
, pp. e52
-
-
Hekele, A.1
Bertholet, S.2
Archer, J.3
Gibson, D.G.4
Palladino, G.5
Brito, L.A.6
Otten, G.R.7
Brazzoli, M.8
Buccato, S.9
Bonci, A.10
-
210
-
-
85020604600
-
-
Clinical Trial Phase I
-
IVAC MUTANOME Phase I Clinical Trial. (2014). Clinical Trial Phase I
-
(2014)
-
-
-
211
-
-
84929288176
-
A novel RNA-based adjuvant combines strong immunostimulatory capacities with a favorable safety profile
-
25530186
-
Heidenreich R, Jasny E, Kowalczyk A, Lutz J, Probst J, Baumhof P, Scheel B, Voss S, Kallen KJ, Fotin-Mleczek M. A novel RNA-based adjuvant combines strong immunostimulatory capacities with a favorable safety profile. Int J Cancer 2015; 137:372-84; PMID:25530186; http://dx.doi.org/10.1002/ijc.29402
-
(2015)
Int J Cancer
, vol.137
, pp. 372-384
-
-
Heidenreich, R.1
Jasny, E.2
Kowalczyk, A.3
Lutz, J.4
Probst, J.5
Baumhof, P.6
Scheel, B.7
Voss, S.8
Kallen, K.J.9
Fotin-Mleczek, M.10
-
212
-
-
85020589447
-
-
CureVac Press Release. (http://www.curevac.com/fileadmin/curevac.de/media/Content/Newsroom/20151221_CureVac_Press_release_JP_Morgan.pdf). 2015
-
(2015)
-
-
-
213
-
-
84947087821
-
Biomaterials for mRNA delivery
-
26280625
-
Islam MA, Reesor EK, Xu Y, Zope HR, Zetter BR, Shi J. Biomaterials for mRNA delivery. Biomater Sci 2015; 3:1519-33; PMID:26280625; http://dx.doi.org/10.1039/C5BM00198F
-
(2015)
Biomater Sci
, vol.3
, pp. 1519-1533
-
-
Islam, M.A.1
Reesor, E.K.2
Xu, Y.3
Zope, H.R.4
Zetter, B.R.5
Shi, J.6
-
214
-
-
84865994357
-
Nonviral delivery of self-amplifying RNA vaccines
-
22908294
-
Geall AJ, Verma A, Otten GR, Shaw CA, Hekele A, Banerjee K, Cu Y, Beard CW, Brito LA, Krucker T, et al. Nonviral delivery of self-amplifying RNA vaccines. Proc Natl Acad Sci U S A 2012; 109:14604-9; PMID:22908294; http://dx.doi.org/10.1073/pnas.1209367109
-
(2012)
Proc Natl Acad Sci U S A
, vol.109
, pp. 14604-14609
-
-
Geall, A.J.1
Verma, A.2
Otten, G.R.3
Shaw, C.A.4
Hekele, A.5
Banerjee, K.6
Cu, Y.7
Beard, C.W.8
Brito, L.A.9
Krucker, T.10
-
215
-
-
84898604804
-
Development of nucleic acid vaccines: use of self-amplifying RNA in lipid nanoparticles
-
24748793
-
Rodriguez-Gascon A, del Pozo-Rodriguez A, Solinis MA. Development of nucleic acid vaccines:use of self-amplifying RNA in lipid nanoparticles. Int J Nanomedicine 2014; 9:1833-43; PMID:24748793; http://dx.doi.org/10.2147/IJN.S39810
-
(2014)
Int J Nanomedicine
, vol.9
, pp. 1833-1843
-
-
Rodriguez-Gascon, A.1
del Pozo-Rodriguez, A.2
Solinis, M.A.3
-
216
-
-
84883573474
-
RNA replicon delivery via lipid-complexed PRINT protein particles
-
23924216
-
Xu J, Luft JC, Yi X, Tian S, Owens G, Wang J, Johnson A, Berglund P, Smith J, Napier ME, et al. RNA replicon delivery via lipid-complexed PRINT protein particles. Mol Pharm 2013; 10:3366-74; PMID:23924216; http://dx.doi.org/10.1021/mp400190z
-
(2013)
Mol Pharm
, vol.10
, pp. 3366-3374
-
-
Xu, J.1
Luft, J.C.2
Yi, X.3
Tian, S.4
Owens, G.5
Wang, J.6
Johnson, A.7
Berglund, P.8
Smith, J.9
Napier, M.E.10
-
217
-
-
84921384147
-
The messenger's great message for vaccination
-
25586101
-
Pascolo S. The messenger's great message for vaccination. Expert Rev Vaccines 2015; 14:153-6; PMID:25586101; http://dx.doi.org/10.1586/14760584.2015.1000871
-
(2015)
Expert Rev Vaccines
, vol.14
, pp. 153-156
-
-
Pascolo, S.1
-
218
-
-
84947087001
-
Prophylactic mRNA Vaccination against Allergy Confers Long-Term Memory Responses and Persistent Protection in Mice
-
26557723
-
Hattinger E, Scheiblhofer S, Roesler E, Thalhamer T, Thalhamer J, Weiss R. Prophylactic mRNA Vaccination against Allergy Confers Long-Term Memory Responses and Persistent Protection in Mice. J Immunol Res 2015; 2015:797421; PMID:26557723; http://dx.doi.org/10.1155/2015/797421
-
(2015)
J Immunol Res
, vol.2015
, pp. 797421
-
-
Hattinger, E.1
Scheiblhofer, S.2
Roesler, E.3
Thalhamer, T.4
Thalhamer, J.5
Weiss, R.6
-
219
-
-
0027312787
-
A double-blind, placebo-controlled evaluation of sublingual immunotherapy with standardized cat extract
-
8349933
-
Nelson HS, Oppenheimer J, Vatsia GA, Buchmeier A. A double-blind, placebo-controlled evaluation of sublingual immunotherapy with standardized cat extract. J Allergy Clin Immunol 1993; 92:229-36; PMID:8349933; http://dx.doi.org/10.1016/0091-6749(93)90166-D
-
(1993)
J Allergy Clin Immunol
, vol.92
, pp. 229-236
-
-
Nelson, H.S.1
Oppenheimer, J.2
Vatsia, G.A.3
Buchmeier, A.4
-
220
-
-
71749093911
-
Immunize and disappear-safety-optimized mRNA vaccination with a panel of 29 allergens
-
19665781
-
Roesler E, Weiss R, Weinberger EE, Fruehwirth A, Stoecklinger A, Mostbock S, Ferreira F, Thalhamer J, Scheiblhofer S. Immunize and disappear-safety-optimized mRNA vaccination with a panel of 29 allergens. J Allergy Clin Immunol 2009; 124:1070-7 e1-11; PMID:19665781; http://dx.doi.org/10.1016/j.jaci.2009.06.036
-
(2009)
J Allergy Clin Immunol
, vol.124
, pp. 1070-1077
-
-
Roesler, E.1
Weiss, R.2
Weinberger, E.E.3
Fruehwirth, A.4
Stoecklinger, A.5
Mostbock, S.6
Ferreira, F.7
Thalhamer, J.8
Scheiblhofer, S.9
-
221
-
-
0033065121
-
Induction of antibody responses to new B cell epitopes indicates vaccination character of allergen immunotherapy
-
10382766
-
Ball T, Sperr WR, Valent P, Lidholm J, Spitzauer S, Ebner C, Kraft D, Valenta R. Induction of antibody responses to new B cell epitopes indicates vaccination character of allergen immunotherapy. Eur J Immunol 1999; 29:2026-36; PMID:10382766; http://dx.doi.org/10.1002/(SICI)1521-4141(199906)29:06%3c2026::AID-IMMU2026%3e3.0.CO;2-2
-
(1999)
Eur J Immunol
, vol.29
, pp. 2026-2036
-
-
Ball, T.1
Sperr, W.R.2
Valent, P.3
Lidholm, J.4
Spitzauer, S.5
Ebner, C.6
Kraft, D.7
Valenta, R.8
-
222
-
-
8044234361
-
Measurement of IgE antibodies against purified grass pollen allergens (Lol p 1, 2, 3 and 5) during immunotherapy
-
9117883
-
Van Ree R, Van Leeuwen WA, Dieges PH, Van Wijk RG, De Jong N, Brewczyski PZ, Kroon AM, Schilte PP, Tan KY, Simon-Licht IF, et al. Measurement of IgE antibodies against purified grass pollen allergens (Lol p 1, 2, 3 and 5) during immunotherapy. Clin Exp Allergy 1997; 27:68-74; PMID:9117883; http://dx.doi.org/10.1046/j.1365-2222.1997.d01-416.x
-
(1997)
Clin Exp Allergy
, vol.27
, pp. 68-74
-
-
Van Ree, R.1
Van Leeuwen, W.A.2
Dieges, P.H.3
Van Wijk, R.G.4
De Jong, N.5
Brewczyski, P.Z.6
Kroon, A.M.7
Schilte, P.P.8
Tan, K.Y.9
Simon-Licht, I.F.10
-
223
-
-
84867835332
-
An RNA-deaminase conjugate selectively repairs point mutations
-
23038402
-
Stafforst T, Schneider MF. An RNA-deaminase conjugate selectively repairs point mutations. Angewandte Chemie 2012; 51:11166-9; PMID:23038402; http://dx.doi.org/10.1002/anie.201206489
-
(2012)
Angewandte Chemie
, vol.51
, pp. 11166-11169
-
-
Stafforst, T.1
Schneider, M.F.2
-
224
-
-
84903209128
-
Optimal guideRNAs for re-directing deaminase activity of hADAR1 and hADAR2 in trans
-
24744243
-
Schneider MF, Wettengel J, Hoffmann PC, Stafforst T. Optimal guideRNAs for re-directing deaminase activity of hADAR1 and hADAR2 in trans. Nucleic Acids Res 2014; 42:e87; PMID:24744243; http://dx.doi.org/10.1093/nar/gku272
-
(2014)
Nucleic Acids Res
, vol.42
, pp. e87
-
-
Schneider, M.F.1
Wettengel, J.2
Hoffmann, P.C.3
Stafforst, T.4
-
225
-
-
84887300101
-
Correction of mutations within the cystic fibrosis transmembrane conductance regulator by site-directed RNA editing
-
24108353
-
Montiel-Gonzalez MF, Vallecillo-Viejo I, Yudowski GA, Rosenthal JJ. Correction of mutations within the cystic fibrosis transmembrane conductance regulator by site-directed RNA editing. Proc Natl Acad Sci U S A 2013; 110:18285-90; PMID:24108353; http://dx.doi.org/10.1073/pnas.1306243110
-
(2013)
Proc Natl Acad Sci U S A
, vol.110
, pp. 18285-18290
-
-
Montiel-Gonzalez, M.F.1
Vallecillo-Viejo, I.2
Yudowski, G.A.3
Rosenthal, J.J.4
-
226
-
-
0035997389
-
RNA editing by adenosine deaminases that act on RNA
-
12045112
-
Bass BL. RNA editing by adenosine deaminases that act on RNA. Annu Rev Biochem 2002; 71:817-46; PMID:12045112; http://dx.doi.org/10.1146/annurev.biochem.71.110601.135501
-
(2002)
Annu Rev Biochem
, vol.71
, pp. 817-846
-
-
Bass, B.L.1
-
227
-
-
77952293063
-
Functions and regulation of RNA editing by ADAR deaminases
-
20192758
-
Nishikura K. Functions and regulation of RNA editing by ADAR deaminases. Annu Rev Biochem 2010; 79:321-49; PMID:20192758; http://dx.doi.org/10.1146/annurev-biochem-060208-105251
-
(2010)
Annu Rev Biochem
, vol.79
, pp. 321-349
-
-
Nishikura, K.1
-
228
-
-
85027937587
-
Site-directed RNA editing with antagomir deaminases–a tool to study protein and RNA function
-
24954543
-
Vogel P, Stafforst T. Site-directed RNA editing with antagomir deaminases–a tool to study protein and RNA function. ChemMedChem 2014; 9:2021-5; PMID:24954543; http://dx.doi.org/10.1002/cmdc.201402139
-
(2014)
ChemMedChem
, vol.9
, pp. 2021-2025
-
-
Vogel, P.1
Stafforst, T.2
-
229
-
-
84902201236
-
Improving site-directed RNA editing in vitro and in cell culture by chemical modification of the guideRNA
-
24890431
-
Vogel P, Schneider MF, Wettengel J, Stafforst T. Improving site-directed RNA editing in vitro and in cell culture by chemical modification of the guideRNA. Angewandte Chemie 2014; 53:6267-71; PMID:24890431; http://dx.doi.org/10.1002/anie.201402634
-
(2014)
Angewandte Chemie
, vol.53
, pp. 6267-6271
-
-
Vogel, P.1
Schneider, M.F.2
Wettengel, J.3
Stafforst, T.4
-
230
-
-
84952837160
-
Site-Directed RNA Editing in Vivo Can Be Triggered by the Light-Driven Assembly of an Artificial Riboprotein
-
26594902
-
Hanswillemenke A, Kuzdere T, Vogel P, Jékely G, Stafforst T. Site-Directed RNA Editing in Vivo Can Be Triggered by the Light-Driven Assembly of an Artificial Riboprotein. J Am Chem Soc 2015; 137:15875-81; PMID:26594902; http://dx.doi.org/10.1021/jacs.5b10216
-
(2015)
J Am Chem Soc
, vol.137
, pp. 15875-15881
-
-
Hanswillemenke, A.1
Kuzdere, T.2
Vogel, P.3
Jékely, G.4
Stafforst, T.5
-
231
-
-
37749034810
-
Targeted pre-mRNA modification for gene silencing and regulation
-
18066073
-
Zhao X, Yu Y-T. Targeted pre-mRNA modification for gene silencing and regulation. Nat Methods 2008; 5:95-100; PMID:18066073; http://dx.doi.org/10.1038/nmeth1142
-
(2008)
Nat Methods
, vol.5
, pp. 95-100
-
-
Zhao, X.1
Yu, Y.-T.2
-
232
-
-
84856431819
-
RNA therapeutics: beyond RNA interference and antisense oligonucleotides
-
22262036
-
Kole R, Krainer AR, Altman S. RNA therapeutics:beyond RNA interference and antisense oligonucleotides. Nat Rev Drug Discov 2012; 11:125-40; PMID:22262036; http://dx.doi.org/10.1038/nrd3625
-
(2012)
Nat Rev Drug Discov
, vol.11
, pp. 125-140
-
-
Kole, R.1
Krainer, A.R.2
Altman, S.3
-
233
-
-
0037449753
-
C-to-U RNA editing: mechanisms leading to genetic diversity
-
12446660
-
Blanc V, Davidson NO. C-to-U RNA editing:mechanisms leading to genetic diversity. J Biol Chem 2003; 278:1395-8; PMID:12446660; http://dx.doi.org/10.1074/jbc.R200024200
-
(2003)
J Biol Chem
, vol.278
, pp. 1395-1398
-
-
Blanc, V.1
Davidson, N.O.2
-
234
-
-
84956890608
-
N6-methyladenosine-encoded epitranscriptomics
-
26840897
-
Liu N, Pan T. N6-methyladenosine-encoded epitranscriptomics. Nat Struct Mol Biol 2016; 23:98-102; PMID:26840897; http://dx.doi.org/10.1038/nsmb.3162
-
(2016)
Nat Struct Mol Biol
, vol.23
, pp. 98-102
-
-
Liu, N.1
Pan, T.2
-
235
-
-
84875692043
-
MODOMICS: a database of RNA modification pathways—2013 update
-
23118484
-
Machnicka MA, Milanowska K, Oglou OO, Purta E, Kurkowska M, Olchowik A, Januszewski W, Kalinowski S, Dunin-Horkawicz S, Rother KM, et al. MODOMICS:a database of RNA modification pathways—2013 update. Nucleic Acids Res 2013; 41:D262-D7; PMID:23118484; http://dx.doi.org/10.1093/nar/gks1007
-
(2013)
Nucleic Acids Res
, vol.41
, pp. D262
-
-
Machnicka, M.A.1
Milanowska, K.2
Oglou, O.O.3
Purta, E.4
Kurkowska, M.5
Olchowik, A.6
Januszewski, W.7
Kalinowski, S.8
Dunin-Horkawicz, S.9
Rother, K.M.10
-
236
-
-
84955751699
-
A Biocatalytic Cascade for Versatile One‐Pot Modification of mRNA Starting from Methionine Analogues
-
Muttach F, Rentmeister A. A Biocatalytic Cascade for Versatile One‐Pot Modification of mRNA Starting from Methionine Analogues. Angewandte Chemie International Edition 2016; 55:1917-20; http://dx.doi.org/10.1002/anie.201507577
-
(2016)
Angewandte Chemie International Edition
, vol.55
, pp. 1917-1920
-
-
Muttach, F.1
Rentmeister, A.2
-
237
-
-
0035869154
-
Fully modified 2′ MOE oligonucleotides redirect polyadenylation
-
11238995
-
Vickers TA, Wyatt JR, Burckin T, Bennett CF, Freier SM. Fully modified 2′ MOE oligonucleotides redirect polyadenylation. Nucleic Acids Res 2001; 29:1293-9; PMID:11238995; http://dx.doi.org/10.1093/nar/29.6.1293
-
(2001)
Nucleic Acids Res
, vol.29
, pp. 1293-1299
-
-
Vickers, T.A.1
Wyatt, J.R.2
Burckin, T.3
Bennett, C.F.4
Freier, S.M.5
-
238
-
-
3943089138
-
Mechanism of transfer RNA maturation by CCA-adding enzyme without using an oligonucleotide template
-
15295590
-
Xiong Y, Steitz TA. Mechanism of transfer RNA maturation by CCA-adding enzyme without using an oligonucleotide template. Nature 2004; 430:640-5; PMID:15295590; http://dx.doi.org/10.1038/nature02711
-
(2004)
Nature
, vol.430
, pp. 640-645
-
-
Xiong, Y.1
Steitz, T.A.2
-
239
-
-
33846062140
-
Reengineering CCA-adding enzymes to function as (U, G)-or dCdCdA-adding enzymes or poly (C, A) and poly (U, G) polymerases
-
Cho HD, Verlinde CL, Weiner AM. Reengineering CCA-adding enzymes to function as (U, G)-or dCdCdA-adding enzymes or poly (C, A) and poly (U, G) polymerases. Proc Natl Acad Sci U S A 2007; 104:54-9; PMID:17179213; http://dx.doi.org/10.1073/pnas.0606961104
-
(2007)
Proc Natl Acad Sci U S A
, vol.104
, pp. 54-59
-
-
Cho, H.D.1
Verlinde, C.L.2
Weiner, A.M.3
-
240
-
-
33746489955
-
Identification, cloning, and functional analysis of the human U6 snRNA-specific terminal uridylyl transferase
-
16790842
-
Trippe R, Guschina E, Hossbach M, Urlaub H, Lührmann R, Benecke B-J. Identification, cloning, and functional analysis of the human U6 snRNA-specific terminal uridylyl transferase. RNA 2006; 12:1494-504; PMID:16790842; http://dx.doi.org/10.1261/rna.87706
-
(2006)
RNA
, vol.12
, pp. 1494-1504
-
-
Trippe, R.1
Guschina, E.2
Hossbach, M.3
Urlaub, H.4
Lührmann, R.5
Benecke, B.-J.6
-
241
-
-
35548959608
-
RNA-specific ribonucleotidyl transferases
-
17872511
-
Martin G, Keller W. RNA-specific ribonucleotidyl transferases. RNA 2007; 13:1834-49; PMID:17872511; http://dx.doi.org/10.1261/rna.652807
-
(2007)
RNA
, vol.13
, pp. 1834-1849
-
-
Martin, G.1
Keller, W.2
-
242
-
-
2542626706
-
Reversal of cystic fibrosis phenotype in a cultured Δ508 cystic fibrosis transmembrane conductance regulator cell line by oligonucleotide insertion
-
15148387
-
Zamecnik PC, Raychowdhury MK, Tabatadze DR, Cantiello HF. Reversal of cystic fibrosis phenotype in a cultured Δ508 cystic fibrosis transmembrane conductance regulator cell line by oligonucleotide insertion. Proc Natl Acad Sci U S A 2004; 101:8150-5; PMID:15148387; http://dx.doi.org/10.1073/pnas.0401933101
-
(2004)
Proc Natl Acad Sci U S A
, vol.101
, pp. 8150-8155
-
-
Zamecnik, P.C.1
Raychowdhury, M.K.2
Tabatadze, D.R.3
Cantiello, H.F.4
|