-
1
-
-
77949512140
-
RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform
-
Bennett, C. F.; Swayze, E. E. RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform Annu. Rev. Pharmacol. Toxicol. 2010, 50, 259-293 10.1146/annurev.pharmtox.010909.105654
-
(2010)
Annu. Rev. Pharmacol. Toxicol.
, vol.50
, pp. 259-293
-
-
Bennett, C.F.1
Swayze, E.E.2
-
2
-
-
0033053332
-
Crystal Strucuture and Improved Antisense Properties of 2′-O-(2-Methoxyethyl)-RNA
-
Teplova, M. M. G.; Tereshko, V.; Inamati, G. B.; Cook, P. D.; Manoharan, M.; Egli, M. Crystal Strucuture and Improved Antisense Properties of 2′-O-(2-Methoxyethyl)-RNA Nat. Struct. Biol. 1999, 6, 535-539 10.1038/9304
-
(1999)
Nat. Struct. Biol.
, vol.6
, pp. 535-539
-
-
Teplova, M.M.G.1
Tereshko, V.2
Inamati, G.B.3
Cook, P.D.4
Manoharan, M.5
Egli, M.6
-
3
-
-
0027168725
-
Evaluation of 2′-Modified Oligonucleotides Containing 2′-Deoxy Gaps as Antisense Inhibitors of Gene Expression
-
Monia, B. P.; Lesnik, E. A.; Gonzalez, C.; Lima, W. F.; McGee, D.; Guinosso, C. J.; Kawasaki, A. M.; Cook, P. D.; Freier, S. M. Evaluation of 2′-Modified Oligonucleotides Containing 2′-Deoxy Gaps as Antisense Inhibitors of Gene Expression J. Biol. Chem. 1993, 268, 14514-14522
-
(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
Guinosso, C.J.6
Kawasaki, A.M.7
Cook, P.D.8
Freier, S.M.9
-
4
-
-
84880668457
-
Clinical Pharmacological Properties of Mipomersen (Kynamro), a Second Generation Antisense Inhibitor of Apolipoprotein B
-
Crooke, S. T.; Geary, R. S. Clinical Pharmacological Properties of Mipomersen (Kynamro), a Second Generation Antisense Inhibitor of Apolipoprotein B Br. J. Clin. Pharmacol. 2013, 76, 269-276 10.1111/j.1365-2125.2012.04469.x
-
(2013)
Br. J. Clin. Pharmacol.
, vol.76
, pp. 269-276
-
-
Crooke, S.T.1
Geary, R.S.2
-
5
-
-
84937252963
-
Pharmacokinetics, Biodistribution and Cell Uptake of Antisense Oligonucleotides
-
Geary, R. S.; Norris, D.; Yu, R.; Bennett, C. F. Pharmacokinetics, Biodistribution and Cell Uptake of Antisense Oligonucleotides Adv. Drug Delivery Rev. 2015, 87, 46-51 10.1016/j.addr.2015.01.008
-
(2015)
Adv. Drug Delivery Rev.
, vol.87
, pp. 46-51
-
-
Geary, R.S.1
Norris, D.2
Yu, R.3
Bennett, C.F.4
-
6
-
-
79953254828
-
Antisense Drug Discovery and Development
-
Yamamoto, T.; Nakatani, M.; Narukawa, K.; Obika, S. Antisense Drug Discovery and Development Future Med. Chem. 2011, 3, 339-365 10.4155/fmc.11.2
-
(2011)
Future Med. Chem.
, vol.3
, pp. 339-365
-
-
Yamamoto, T.1
Nakatani, M.2
Narukawa, K.3
Obika, S.4
-
7
-
-
84888002594
-
Liver as a Target for Oligonucleotide Therapeutics
-
Sehgal, A.; Vaishnaw, A.; Fitzgerald, K. Liver as a Target for Oligonucleotide Therapeutics J. Hepatol. 2013, 59, 1354-1359 10.1016/j.jhep.2013.05.045
-
(2013)
J. Hepatol.
, vol.59
, pp. 1354-1359
-
-
Sehgal, A.1
Vaishnaw, A.2
Fitzgerald, K.3
-
8
-
-
0016304913
-
The Isolation and Properties of a Rabbit Liver Binding Protein Specific for Asialoglycoproteins
-
Hudgin, R. L.; Pricer, W. E.; Ashwell, G.; Stockert, R. J.; Morell, A. G. The Isolation and Properties of a Rabbit Liver Binding Protein Specific for Asialoglycoproteins J. Biol. Chem. 1974, 249, 5536-5543
-
(1974)
J. Biol. Chem.
, vol.249
, pp. 5536-5543
-
-
Hudgin, R.L.1
Pricer, W.E.2
Ashwell, G.3
Stockert, R.J.4
Morell, A.G.5
-
9
-
-
0025043480
-
The Asialoglycoprotein Receptor: A Model for Endocytic Transport Receptors
-
Spiess, M. The Asialoglycoprotein Receptor: A Model for Endocytic Transport Receptors Biochemistry 1990, 29, 10009-10018 10.1021/bi00495a001
-
(1990)
Biochemistry
, vol.29
, pp. 10009-10018
-
-
Spiess, M.1
-
10
-
-
0028998769
-
The Asialoglycoprotein Receptor: Relationships between Structure, Function, and Expression
-
Stockert, R. J. The Asialoglycoprotein Receptor: Relationships Between Structure, Function, and Expression Physiol. Rev. 1995, 75, 591-609
-
(1995)
Physiol. Rev.
, vol.75
, pp. 591-609
-
-
Stockert, R.J.1
-
11
-
-
63649163810
-
The Asialoglycoprotein Receptor Regulates Levels of Plasma Glycoproteins Terminating with Sialic Acid A2,6-Galactose
-
Steirer, L. M.; Park, E. I.; Townsend, R. R.; Baenziger, J. U. The Asialoglycoprotein Receptor Regulates Levels of Plasma Glycoproteins Terminating with Sialic Acid A2,6-Galactose J. Biol. Chem. 2009, 284, 3777-3783 10.1074/jbc.M808689200
-
(2009)
J. Biol. Chem.
, vol.284
, pp. 3777-3783
-
-
Steirer, L.M.1
Park, E.I.2
Townsend, R.R.3
Baenziger, J.U.4
-
12
-
-
0019190912
-
Human Hepatic Lectin. Physiochemical Properties and Specificity
-
Baenziger, J. U.; Maynard, Y. Human Hepatic Lectin. Physiochemical Properties and Specificity J. Biol. Chem. 1980, 255, 4607-4613
-
(1980)
J. Biol. Chem.
, vol.255
, pp. 4607-4613
-
-
Baenziger, J.U.1
Maynard, Y.2
-
13
-
-
0034697981
-
Crystal Structure of the Carbohydrate Recognition Domain of the H1 Subunit of the Asialoglycoprotein Receptor
-
Meier, M.; Bider, M. D.; Malashkevich, V. N.; Spiess, M.; Burkhard, P. Crystal Structure of the Carbohydrate Recognition Domain of the H1 Subunit of the Asialoglycoprotein Receptor J. Mol. Biol. 2000, 300, 857-865 10.1006/jmbi.2000.3853
-
(2000)
J. Mol. Biol.
, vol.300
, pp. 857-865
-
-
Meier, M.1
Bider, M.D.2
Malashkevich, V.N.3
Spiess, M.4
Burkhard, P.5
-
14
-
-
0020685762
-
Intracellular Site of Asialoglycoprotein Receptor-Ligand Uncoupling: Double-Label Immunoelectron Microscopy during Receptor-Mediated Endocytosis
-
Geuze, H. J.; Slot, J. W.; Strous, G. J. A. M.; Lodish, H. F.; Schwartz, A. L. Intracellular Site of Asialoglycoprotein Receptor-Ligand Uncoupling: Double-Label Immunoelectron Microscopy During Receptor-Mediated Endocytosis Cell 1983, 32, 277-287 10.1016/0092-8674(83)90518-4
-
(1983)
Cell
, vol.32
, pp. 277-287
-
-
Geuze, H.J.1
Slot, J.W.2
Strous, G.J.A.M.3
Lodish, H.F.4
Schwartz, A.L.5
-
15
-
-
7444229247
-
Design and Synthesis of Novel N-Acetylgalactosamine-Terminated Glycolipids for Targeting of Lipoproteins to the Hepatic Asialoglycoprotein Receptor
-
Rensen, P. C. N.; van Leeuwen, S. H.; Sliedregt, L. A. J. M.; van Berkel, T. J. C.; Biessen, E. A. L. Design and Synthesis of Novel N-Acetylgalactosamine-Terminated Glycolipids for Targeting of Lipoproteins to the Hepatic Asialoglycoprotein Receptor J. Med. Chem. 2004, 47, 5798-5808 10.1021/jm049481d
-
(2004)
J. Med. Chem.
, vol.47
, pp. 5798-5808
-
-
Rensen, P.C.N.1
Van Leeuwen, S.H.2
Sliedregt, L.A.J.M.3
Van Berkel, T.J.C.4
Biessen, E.A.L.5
-
16
-
-
0026952599
-
Gene Transfer into Hepatocytes Using Asialoglycoprotein Receptor Mediated Endocytosis of DNA Complexed with an Artificial Tetra-Antennary Galactose Ligand
-
Plank, C.; Zatloukal, K.; Cotten, M.; Mechtler, K.; Wagner, E. Gene Transfer into Hepatocytes Using Asialoglycoprotein Receptor Mediated Endocytosis of DNA Complexed with an Artificial Tetra-Antennary Galactose Ligand Bioconjugate Chem. 1992, 3, 533-539 10.1021/bc00018a012
-
(1992)
Bioconjugate Chem.
, vol.3
, pp. 533-539
-
-
Plank, C.1
Zatloukal, K.2
Cotten, M.3
Mechtler, K.4
Wagner, E.5
-
17
-
-
0033991046
-
Intrabody Tissue-Specific Delivery of Antisense Conjugates in Animals: Ligand-Linker-Antisense Oligomer Conjugates
-
In, Phillips, M. I. Academic Press: New York
-
Duff, R. J.; Deamond, S. F.; Roby, C.; Zhou, Y.; Tso, P. O. P. Intrabody Tissue-Specific Delivery of Antisense Conjugates in Animals: Ligand-Linker-Antisense Oligomer Conjugates. In Methods in Enzymology, Phillips, M. I., Ed.; Academic Press: New York, 2000; Vol. 313, pp 297-321.
-
(2000)
Methods in Enzymology
, vol.313
, pp. 297-321
-
-
Duff, R.J.1
Deamond, S.F.2
Roby, C.3
Zhou, Y.4
Tso, P.O.P.5
-
18
-
-
0033564268
-
Targeted Delivery of Oligodeoxynucleotides to Parenchymal Liver Cells in Vivo
-
Biessen, E. A.; Vietsch, H.; Rump, E. T.; Fluiter, K.; Kuiper, J.; Bijsterbosch, M. K.; van Berkel, T. J. Targeted Delivery of Oligodeoxynucleotides to Parenchymal Liver Cells In Vivo Biochem. J. 1999, 340, 783-792 10.1042/bj3400783
-
(1999)
Biochem. J.
, vol.340
, pp. 783-792
-
-
Biessen, E.A.1
Vietsch, H.2
Rump, E.T.3
Fluiter, K.4
Kuiper, J.5
Bijsterbosch, M.K.6
Van Berkel, T.J.7
-
19
-
-
84918578407
-
Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
-
Nair, J. K.; Willoughby, J. L.; Chan, A.; Charisse, K.; Alam, M. R.; Wang, Q.; Hoekstra, M.; Kandasamy, P.; Kelin, A. V.; Milstein, S.; Taneja, N.; OShea, J.; Shaikh, S.; Zhang, L.; van der Sluis, R. J.; Jung, M. E.; Akinc, A.; Hutabarat, R.; Kuchimanchi, S.; Fitzgerald, K.; Zimmermann, T.; van Berkel, T. J.; Maier, M. A.; Rajeev, K. G.; Manoharan, M. Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing J. Am. Chem. Soc. 2014, 136, 16958-16961 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
Kelin, A.V.9
Milstein, S.10
Taneja, N.11
O'Shea, J.12
Shaikh, S.13
Zhang, L.14
Van Der Sluis, R.J.15
Jung, M.E.16
Akinc, A.17
Hutabarat, R.18
Kuchimanchi, S.19
Fitzgerald, K.20
Zimmermann, T.21
Van Berkel, T.J.22
Maier, M.A.23
Rajeev, K.G.24
Manoharan, M.25
more..
-
20
-
-
84937764228
-
An Rnai Therapeutic Targeting Antithrombin to Rebalance the Coagulation System and Promote Hemostasis in Hemophilia
-
Sehgal, A.; Barros, S.; Ivanciu, L.; Cooley, B.; Qin, J.; Racie, T.; Hettinger, J.; Carioto, M.; Jiang, Y.; Brodsky, J.; Prabhala, H.; Zhang, X.; Attarwala, H.; Hutabarat, R.; Foster, D.; Milstein, S.; Charisse, K.; Kuchimanchi, S.; Maier, M. A.; Nechev, L.; Kandasamy, P.; Kelin, A. V.; Nair, J. K.; Rajeev, K. G.; Manoharan, M.; Meyers, R.; Sorensen, B.; Simon, A. R.; Dargaud, Y.; Negrier, C.; Camire, R. M.; Akinc, A. An Rnai Therapeutic Targeting Antithrombin to Rebalance the Coagulation System and Promote Hemostasis in Hemophilia Nat. Med. 2015, 21, 492-497 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
Prabhala, H.11
Zhang, X.12
Attarwala, H.13
Hutabarat, R.14
Foster, D.15
Milstein, S.16
Charisse, K.17
Kuchimanchi, S.18
Maier, M.A.19
Nechev, L.20
Kandasamy, P.21
Kelin, A.V.22
Nair, J.K.23
Rajeev, K.G.24
Manoharan, M.25
Meyers, R.26
Sorensen, B.27
Simon, A.R.28
Dargaud, Y.29
Negrier, C.30
Camire, R.M.31
Akinc, A.32
more..
-
21
-
-
84962141105
-
-
In AASLD The Liver Meeting, Washington, DC
-
Bhat, B.; Neben, S.; Tay, J.; Liu, K.; Chau, N.; Hogan, D.; MacKenna, D.; Gibson, N. Rg-101, a GalNAc-Conjugated Anti-Mir Employing a Unique Mechanism of Action by Targeting Host Factor Microrna-122 (Mir-122), Demonstrates Potent Activity and Reduction of HCV in Preclinical Studies. In AASLD The Liver Meeting, Washington, DC, 2013.
-
(2013)
Rg-101, a GalNAc-Conjugated Anti-Mir Employing a Unique Mechanism of Action by Targeting Host Factor Microrna-122 (Mir-122), Demonstrates Potent Activity and Reduction of HCV in Preclinical Studies
-
-
Bhat, B.1
Neben, S.2
Tay, J.3
Liu, K.4
Chau, N.5
Hogan, D.6
MacKenna, D.7
Gibson, N.8
-
22
-
-
84962141123
-
-
246th ACS National Meeting & Exposition, Indianapolis, IN, United States, September 8-12, 2013 (), MEDI-185
-
Manoharan, M. Delivery strategies for RNA interference (RNAi) based therapeutics. From Abstracts of Papers, 246th ACS National Meeting & Exposition, Indianapolis, IN, United States, September 8-12, 2013 (2013), MEDI-185.
-
(2013)
Delivery Strategies for RNA Interference (RNAi) Based Therapeutics. from Abstracts of Papers
-
-
Manoharan, M.1
-
23
-
-
84905584822
-
Targeted Delivery of Antisense Oligonucleotides to Hepatocytes Using Triantennary N-Acetyl Galactosamine Improves Potency 10-Fold in Mice
-
Prakash, T. P.; Graham, M. J.; Yu, J.; Carty, R.; Low, A.; Chappell, A.; Schmidt, K.; Zhao, C.; Aghajan, M.; Murray, H. F.; Riney, S.; Booten, S. L.; Murray, S. F.; Gaus, H.; Crosby, J.; Lima, W. F.; Guo, S.; Monia, B. P.; Swayze, E. E.; Seth, P. P. Targeted Delivery of Antisense Oligonucleotides to Hepatocytes Using Triantennary N-Acetyl Galactosamine Improves Potency 10-Fold in Mice Nucleic Acids Res. 2014, 42, 8796-8807 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
Riney, S.11
Booten, S.L.12
Murray, S.F.13
Gaus, H.14
Crosby, J.15
Lima, W.F.16
Guo, S.17
Monia, B.P.18
Swayze, E.E.19
Seth, P.P.20
more..
-
24
-
-
84939800605
-
Efficient Synthesis and Biological Evaluation of 5′-GalNAc Conjugated Antisense Oligonucleotides
-
Østergaard, M. E.; Yu, J.; Kinberger, G. A.; Wan, W. B.; Migawa, M. T.; Vasquez, G.; Schmidt, K.; Gaus, H. J.; Murray, H. M.; Low, A.; Swayze, E. E.; Prakash, T. P.; Seth, P. P. Efficient Synthesis and Biological Evaluation of 5′-GalNAc Conjugated Antisense Oligonucleotides Bioconjugate Chem. 2015, 26, 1451-1455 10.1021/acs.bioconjchem.5b00265
-
(2015)
Bioconjugate Chem.
, vol.26
, pp. 1451-1455
-
-
Østergaard, M.E.1
Yu, J.2
Kinberger, G.A.3
Wan, W.B.4
Migawa, M.T.5
Vasquez, G.6
Schmidt, K.7
Gaus, H.J.8
Murray, H.M.9
Low, A.10
Swayze, E.E.11
Prakash, T.P.12
Seth, P.P.13
-
25
-
-
0035813160
-
Determination of the Upper Size Limit for Uptake and Processing of Ligands by the Asialoglycoprotein Receptor on Hepatocytesin in Vitro and in Vivo
-
Rensen, P. C. N.; Sliedregt, L. A. J. M.; Ferns, M.; Kieviet, E.; van Rossenberg, S. M. W.; van Leeuwen, S. H.; van Berkel, T. J. C.; Biessen, E. A. L. Determination of the Upper Size Limit for Uptake and Processing of Ligands by the Asialoglycoprotein Receptor on Hepatocytesin In Vitro and In Vivo J. Biol. Chem. 2001, 276, 37577-37584 10.1074/jbc.M101786200
-
(2001)
J. Biol. Chem.
, vol.276
, pp. 37577-37584
-
-
Rensen, P.C.N.1
Sliedregt, L.A.J.M.2
Ferns, M.3
Kieviet, E.4
Van Rossenberg, S.M.W.5
Van Leeuwen, S.H.6
Van Berkel, T.J.C.7
Biessen, E.A.L.8
-
26
-
-
0028903260
-
Synthesis of Cluster Galactosides with High Affinity for the Hepatic Asialoglycoprotein Receptor
-
Biessen, E. A. L.; Beuting, D. M.; Roelen, H. C. P. F.; van de Marel, G. A.; Van Boom, J. H.; Van Berkel, T. J. C. Synthesis of Cluster Galactosides with High Affinity for the Hepatic Asialoglycoprotein Receptor J. Med. Chem. 1995, 38, 1538-1546 10.1021/jm00009a014
-
(1995)
J. Med. Chem.
, vol.38
, pp. 1538-1546
-
-
Biessen, E.A.L.1
Beuting, D.M.2
Roelen, H.C.P.F.3
Van De Marel, G.A.4
Van Boom, J.H.5
Van Berkel, T.J.C.6
-
27
-
-
7444229247
-
Design and Synthesis of Novel N-Acetylgalactosamine-Terminated Glycolipids for Targeting of Lipoproteins to the Hepatic Asialoglycoprotein Receptor
-
Rensen, P. C.; van Leeuwen, S. H.; Sliedregt, L. A.; van Berkel, T. J.; Biessen, E. A. Design and Synthesis of Novel N-Acetylgalactosamine-Terminated Glycolipids for Targeting of Lipoproteins to the Hepatic Asialoglycoprotein Receptor J. Med. Chem. 2004, 47, 5798-5808 10.1021/jm049481d
-
(2004)
J. Med. Chem.
, vol.47
, pp. 5798-5808
-
-
Rensen, P.C.1
Van Leeuwen, S.H.2
Sliedregt, L.A.3
Van Berkel, T.J.4
Biessen, E.A.5
-
28
-
-
84956507409
-
Interactions of Oligosaccharides and Glycopeptides with Hepatic Carbohydrate Receptors
-
In, Ernst, B. Hart, G. W. Sinaý P. Wiley-VCH Verlag GmbH: Weinheim, Germany
-
Lee, Y. C.; Lee, R. T. Interactions of Oligosaccharides and Glycopeptides with Hepatic Carbohydrate Receptors. In Carbohydrates in Chemistry and Biology, Ernst, B.; Hart, G. W.; Sinaý, P., Eds.; Wiley-VCH Verlag GmbH: Weinheim, Germany, 2008; Vol. 4, pp 549-561.
-
(2008)
Carbohydrates in Chemistry and Biology
, vol.4
, pp. 549-561
-
-
Lee, Y.C.1
Lee, R.T.2
-
29
-
-
0000777421
-
Preparation of Cluster Glycosides of N-Acetylgalactosamine That Have Subnanomolar Binding Constants Towards the Mammalian Hepatic Gal/GalNAc-Specific Receptor
-
Lee, R.; Lee, Y. Preparation of Cluster Glycosides of N-Acetylgalactosamine That Have Subnanomolar Binding Constants Towards the Mammalian Hepatic Gal/GalNAc-Specific Receptor Glycoconjugate J. 1987, 4, 317-328 10.1007/BF01048365
-
(1987)
Glycoconjugate J.
, vol.4
, pp. 317-328
-
-
Lee, R.1
Lee, Y.2
-
30
-
-
0021772463
-
New Synthetic Cluster Ligands for Galactose/N-Acetylgalactosamine-Specific Lectin of Mammalian Liver
-
Lee, R. T.; Lin, P.; Lee, Y. C. New Synthetic Cluster Ligands for Galactose/N-Acetylgalactosamine-Specific Lectin of Mammalian Liver Biochemistry 1984, 23, 4255-4261 10.1021/bi00313a037
-
(1984)
Biochemistry
, vol.23
, pp. 4255-4261
-
-
Lee, R.T.1
Lin, P.2
Lee, Y.C.3
-
31
-
-
0021099058
-
Binding of Synthetic Oligosaccharides to the Hepatic Gal/GalNAc Lectin Dependence on Fine Structural Features
-
Lee, Y. C.; Townsend, R. R.; Hardy, M. R.; Lönngren, J.; Arnarp, J.; Haraldsson, M.; Lönn, H. Binding of Synthetic Oligosaccharides to the Hepatic Gal/GalNAc Lectin Dependence on Fine Structural Features J. Biol. Chem. 1983, 258, 199-202
-
(1983)
J. Biol. Chem.
, vol.258
, pp. 199-202
-
-
Lee, Y.C.1
Townsend, R.R.2
Hardy, M.R.3
Lönngren, J.4
Arnarp, J.5
Haraldsson, M.6
Lönn, H.7
-
32
-
-
0031021769
-
Solid-Phase Synthesis of Lysine-Based Cluster Galactosides with High Affinity for the Asialoglycoprotein Receptor
-
Valentijn, A. R. P. M.; van der Marel, G. A.; Sliedregt, L. A. J. M.; van Berkel, T. J. C.; Biessen, E. A. L.; van Boom, J. H. Solid-Phase Synthesis of Lysine-Based Cluster Galactosides with High Affinity for the Asialoglycoprotein Receptor Tetrahedron 1997, 53, 759-770 10.1016/S0040-4020(96)01018-6
-
(1997)
Tetrahedron
, vol.53
, pp. 759-770
-
-
Valentijn, A.R.P.M.1
Van Der Marel, G.A.2
Sliedregt, L.A.J.M.3
Van Berkel, T.J.C.4
Biessen, E.A.L.5
Van Boom, J.H.6
-
34
-
-
84926320054
-
Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-Nucleosidic Trivalent N-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo
-
Rajeev, K. G.; Nair, J. K.; Jayaraman, M.; Charisse, K.; Taneja, N.; OShea, J.; Willoughby, J. L. S.; Yucius, K.; Nguyen, T.; Shulga-Morskaya, S.; Milstein, S.; Liebow, A.; Querbes, W.; Borodovsky, A.; Fitzgerald, K.; Maier, M. A.; Manoharan, M. Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-Nucleosidic Trivalent N-Acetylgalactosamine Elicits Robust Gene Silencing In Vivo ChemBioChem 2015, 16, 903-908 10.1002/cbic.201500023
-
(2015)
ChemBioChem
, vol.16
, pp. 903-908
-
-
Rajeev, K.G.1
Nair, J.K.2
Jayaraman, M.3
Charisse, K.4
Taneja, N.5
O'Shea, J.6
Willoughby, J.L.S.7
Yucius, K.8
Nguyen, T.9
Shulga-Morskaya, S.10
Milstein, S.11
Liebow, A.12
Querbes, W.13
Borodovsky, A.14
Fitzgerald, K.15
Maier, M.A.16
Manoharan, M.17
-
35
-
-
0022759438
-
New Procedure for the Preparation of Oligosaccharide Oxazolines
-
Nakabayashi, S.; Warren, C. D.; Jeanloz, R. W. New Procedure for the Preparation of Oligosaccharide Oxazolines Carbohydr. Res. 1986, 150, C7 10.1016/0008-6215(86)80028-3
-
(1986)
Carbohydr. Res.
, vol.150
, pp. C7
-
-
Nakabayashi, S.1
Warren, C.D.2
Jeanloz, R.W.3
-
36
-
-
0037154863
-
An Improved Synthesis of a Trifurcated Newkome-Type Monomer and Orthogonally Protected Two-Generation Dendrons
-
Cardona, C. M.; Gawley, R. E. An Improved Synthesis of a Trifurcated Newkome-Type Monomer and Orthogonally Protected Two-Generation Dendrons J. Org. Chem. 2002, 67, 1411-1413 10.1021/jo0161678
-
(2002)
J. Org. Chem.
, vol.67
, pp. 1411-1413
-
-
Cardona, C.M.1
Gawley, R.E.2
-
37
-
-
0025127646
-
Preparation of Pentafluorophenyl Esters of Fmoc Protected Amino Acids with Pentafluorophenyl Trifluoroacetate
-
Green, M.; Berman, J. Preparation of Pentafluorophenyl Esters of Fmoc Protected Amino Acids with Pentafluorophenyl Trifluoroacetate Tetrahedron Lett. 1990, 31, 5851-5852 10.1016/S0040-4039(00)97976-0
-
(1990)
Tetrahedron Lett.
, vol.31
, pp. 5851-5852
-
-
Green, M.1
Berman, J.2
-
38
-
-
42949121053
-
Comparing in Vitro and in Vivo Activity of 2′-O-[2-(Methylamino)-2-Oxoethyl]- and 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides
-
Prakash, T. P.; Kawasaki, A. M.; Wancewicz, E. V.; Shen, L.; Monia, B. P.; Ross, B. S.; Bhat, B.; Manoharan, M. Comparing In Vitro and In Vivo Activity of 2′-O-[2-(Methylamino)-2-Oxoethyl]- and 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides J. Med. Chem. 2008, 51, 2766-2776 10.1021/jm701537z
-
(2008)
J. Med. Chem.
, vol.51
, pp. 2766-2776
-
-
Prakash, T.P.1
Kawasaki, A.M.2
Wancewicz, E.V.3
Shen, L.4
Monia, B.P.5
Ross, B.S.6
Bhat, B.7
Manoharan, M.8
-
39
-
-
84864062473
-
TricycloDNA-Modified Oligo-2′-Deoxyribonucleotides Reduce Scavenger Receptor B1 mRNA in Hepatic and Extra-Hepatic Tissues a Comparative Study of Oligonucleotide Length, Design and Chemistry
-
Murray, S.; Ittig, D.; Koller, E.; Berdeja, A.; Chappell, A.; Prakash, T. P.; Norrbom, M.; Swayze, E. E.; Leumann, C. J.; Seth, P. P. TricycloDNA-Modified Oligo-2′-Deoxyribonucleotides Reduce Scavenger Receptor B1 mRNA in Hepatic and Extra-Hepatic Tissues a Comparative Study of Oligonucleotide Length, Design and Chemistry Nucleic Acids Res. 2012, 40, 6135-6143 10.1093/nar/gks273
-
(2012)
Nucleic Acids Res.
, vol.40
, pp. 6135-6143
-
-
Murray, S.1
Ittig, D.2
Koller, E.3
Berdeja, A.4
Chappell, A.5
Prakash, T.P.6
Norrbom, M.7
Swayze, E.E.8
Leumann, C.J.9
Seth, P.P.10
-
40
-
-
0019159338
-
Galactose and N-Acetylgalactosamine-Specific Endocytosis of Glycopeptides by Isolated Rat Hepatocytes
-
Baenziger, J. U.; Fiete, D. Galactose and N-Acetylgalactosamine-Specific Endocytosis of Glycopeptides by Isolated Rat Hepatocytes Cell 1980, 22, 611-620 10.1016/0092-8674(80)90371-2
-
(1980)
Cell
, vol.22
, pp. 611-620
-
-
Baenziger, J.U.1
Fiete, D.2
-
41
-
-
84858657146
-
A Rapid Two-Step Method for Isolation of Functional Primary Mouse Hepatocytes: Cell Characterization and Asialoglycoprotein Receptor Based Assay Development
-
Severgnini, M.; Sherman, J.; Sehgal, A.; Jayaprakash, N. K.; Aubin, J.; Wang, G.; Zhang, L.; Peng, C. G.; Yucius, K.; Butler, J.; Fitzgerald, K. A Rapid Two-Step Method for Isolation of Functional Primary Mouse Hepatocytes: Cell Characterization and Asialoglycoprotein Receptor Based Assay Development Cytotechnology 2012, 64, 187-195 10.1007/s10616-011-9407-0
-
(2012)
Cytotechnology
, vol.64
, pp. 187-195
-
-
Severgnini, M.1
Sherman, J.2
Sehgal, A.3
Jayaprakash, N.K.4
Aubin, J.5
Wang, G.6
Zhang, L.7
Peng, C.G.8
Yucius, K.9
Butler, J.10
Fitzgerald, K.11
-
42
-
-
79959479529
-
Mechanisms of Single-Stranded Phosphorothioate Modified Antisense Oligonucleotide Accumulation in Hepatocytes
-
Koller, E.; Vincent, T. M.; Chappell, A.; De, S.; Manoharan, M.; Bennett, C. F. Mechanisms of Single-Stranded Phosphorothioate Modified Antisense Oligonucleotide Accumulation in Hepatocytes Nucleic Acids Res. 2011, 39, 4795-4807 10.1093/nar/gkr089
-
(2011)
Nucleic Acids Res.
, vol.39
, pp. 4795-4807
-
-
Koller, E.1
Vincent, T.M.2
Chappell, A.3
De, S.4
Manoharan, M.5
Bennett, C.F.6
-
43
-
-
79954417405
-
New and More Efficient Multivalent Glyco-Ligands for Asialoglycoprotein Receptor of Mammalian Hepatocytes
-
Lee, R. T.; Wang, M.-H.; Lin, W.-J.; Lee, Y. C. New and More Efficient Multivalent Glyco-Ligands for Asialoglycoprotein Receptor of Mammalian Hepatocytes Bioorg. Med. Chem. 2011, 19, 2494-2500 10.1016/j.bmc.2011.03.027
-
(2011)
Bioorg. Med. Chem.
, vol.19
, pp. 2494-2500
-
-
Lee, R.T.1
Wang, M.-H.2
Lin, W.-J.3
Lee, Y.C.4
-
44
-
-
84944930132
-
Solid-Phase Synthesis of 5′-Triantennary N-Acetylgalactosamine Conjugated Antisense Oligonucleotides Using Phosphoramidite Chemistry
-
Prakash, T. P.; Brad Wan, W.; Low, A.; Yu, J.; Chappell, A. E.; Gaus, H.; Kinberger, G. A.; Østergaard, M. E.; Migawa, M. T.; Swayze, E. E.; Seth, P. P. Solid-Phase Synthesis of 5′-Triantennary N-Acetylgalactosamine Conjugated Antisense Oligonucleotides Using Phosphoramidite Chemistry Bioorg. Med. Chem. Lett. 2015, 25, 4127-4130 10.1016/j.bmcl.2015.08.019
-
(2015)
Bioorg. Med. Chem. Lett.
, vol.25
, pp. 4127-4130
-
-
Prakash, T.P.1
Brad Wan, W.2
Low, A.3
Yu, J.4
Chappell, A.E.5
Gaus, H.6
Kinberger, G.A.7
Østergaard, M.E.8
Migawa, M.T.9
Swayze, E.E.10
Seth, P.P.11
-
45
-
-
84949782714
-
Serial Incorporation of a Monovalent GalNAc Phosphoramidite Unit into Hepatocyte-Targeting Antisense Oligonucleotides
-
Yamamoto, T.; Sawamura, M.; Wada, F.; Harada-Shiba, M.; Obika, S. Serial Incorporation of a Monovalent GalNAc Phosphoramidite Unit into Hepatocyte-Targeting Antisense Oligonucleotides Bioorg. Med. Chem. 2015, 24, 26 10.1016/j.bmc.2015.11.036
-
(2015)
Bioorg. Med. Chem.
, vol.24
, pp. 26
-
-
Yamamoto, T.1
Sawamura, M.2
Wada, F.3
Harada-Shiba, M.4
Obika, S.5
-
46
-
-
0028114548
-
Effect of Phosphorothioate Modification of Oligodeoxynucleotides on Specific Protein Binding
-
Brown, D. A.; Kang, S. H.; Gryaznov, S. M.; DeDionisio, L.; Heidenreich, O.; Sullivan, S.; Xu, X.; Nerenberg, M. I. Effect of Phosphorothioate Modification of Oligodeoxynucleotides on Specific Protein Binding J. Biol. Chem. 1994, 269, 26801-26805
-
(1994)
J. Biol. Chem.
, vol.269
, pp. 26801-26805
-
-
Brown, D.A.1
Kang, S.H.2
Gryaznov, S.M.3
DeDionisio, L.4
Heidenreich, O.5
Sullivan, S.6
Xu, X.7
Nerenberg, M.I.8
-
47
-
-
0035119936
-
Pharmacokinetic Properties of 2′-O-(2-Methoxyethyl)-Modified Oligonucleotide Analogs in Rats
-
Geary, R. S.; Watanabe, T. A.; Truong, L.; Freier, S.; Lesnik, E. A.; Sioufi, N. B.; Sasmor, H.; Manoharan, M.; Levin, A. A. Pharmacokinetic Properties of 2′-O-(2-Methoxyethyl)-Modified Oligonucleotide Analogs in Rats J. Pharmacol. Exp. Ther. 2001, 296, 890-897
-
(2001)
J. Pharmacol. Exp. Ther.
, vol.296
, pp. 890-897
-
-
Geary, R.S.1
Watanabe, T.A.2
Truong, L.3
Freier, S.4
Lesnik, E.A.5
Sioufi, N.B.6
Sasmor, H.7
Manoharan, M.8
Levin, A.A.9
-
48
-
-
0032697942
-
A Review of the Issues in the Pharmacokinetics and Toxicology of Phosphorothioate Antisense Oligonucleotides
-
Levin, A. A. A Review of the Issues in the Pharmacokinetics and Toxicology of Phosphorothioate Antisense Oligonucleotides Biochim. Biophys. Acta, Gene Struct. Expression 1999, 1489, 69-84 10.1016/S0167-4781(99)00140-2
-
(1999)
Biochim. Biophys. Acta, Gene Struct. Expression
, vol.1489
, pp. 69-84
-
-
Levin, A.A.1
-
49
-
-
84867346804
-
Assessing Unintended Hybridization-Induced Biological Effects of Oligonucleotides
-
Lindow, M.; Vornlocher, H.-P.; Riley, D.; Kornbrust, D. J.; Burchard, J.; Whiteley, L. O.; Kamens, J.; Thompson, J. D.; Nochur, S.; Younis, H.; Bartz, S.; Parry, J.; Ferrari, N.; Henry, S. P.; Levin, A. A. Assessing Unintended Hybridization-Induced Biological Effects of Oligonucleotides Nat. Biotechnol. 2012, 30, 920-923 10.1038/nbt.2376
-
(2012)
Nat. Biotechnol.
, vol.30
, pp. 920-923
-
-
Lindow, M.1
Vornlocher, H.-P.2
Riley, D.3
Kornbrust, D.J.4
Burchard, J.5
Whiteley, L.O.6
Kamens, J.7
Thompson, J.D.8
Nochur, S.9
Younis, H.10
Bartz, S.11
Parry, J.12
Ferrari, N.13
Henry, S.P.14
Levin, A.A.15
-
50
-
-
84892936309
-
Antisense Oligonucleotide Treatment Ameliorates Alpha-1 Antitrypsin-Related Liver Disease in Mice
-
Guo, S.; Booten, S. L.; Aghajan, M.; Hung, G.; Zhao, C.; Blomenkamp, K.; Gattis, D.; Watt, A.; Freier, S. M.; Teckman, J. H.; McCaleb, M. L.; Monia, B. P. Antisense Oligonucleotide Treatment Ameliorates Alpha-1 Antitrypsin-Related Liver Disease in Mice J. Clin. Invest. 2014, 124, 251-261 10.1172/JCI67968
-
(2014)
J. Clin. Invest.
, vol.124
, pp. 251-261
-
-
Guo, S.1
Booten, S.L.2
Aghajan, M.3
Hung, G.4
Zhao, C.5
Blomenkamp, K.6
Gattis, D.7
Watt, A.8
Freier, S.M.9
Teckman, J.H.10
McCaleb, M.L.11
Monia, B.P.12
-
51
-
-
84879076198
-
Antithrombotic Effect of Antisense Factor XI Oligonucleotide Treatment in Primates
-
Crosby, J. R.; Marzec, U.; Revenko, A. S.; Zhao, C.; Gao, D.; Matafonov, A.; Gailani, D.; MacLeod, A. R.; Tucker, E. I.; Gruber, A.; Hanson, S. R.; Monia, B. P. Antithrombotic Effect of Antisense Factor XI Oligonucleotide Treatment in Primates Arterioscler., Thromb., Vasc. Biol. 2013, 33, 1670-1678 10.1161/ATVBAHA.113.301282
-
(2013)
Arterioscler., Thromb., Vasc. Biol.
, vol.33
, pp. 1670-1678
-
-
Crosby, J.R.1
Marzec, U.2
Revenko, A.S.3
Zhao, C.4
Gao, D.5
Matafonov, A.6
Gailani, D.7
MacLeod, A.R.8
Tucker, E.I.9
Gruber, A.10
Hanson, S.R.11
Monia, B.P.12
-
52
-
-
78649471947
-
Inhibition of the Intrinsic Coagulation Pathway Factor XI by Antisense Oligonucleotides: A Novel Antithrombotic Strategy with Lowered Bleeding Risk
-
Zhang, H.; Lowenberg, E. C.; Crosby, J. R.; MacLeod, A. R.; Zhao, C.; Gao, D.; Black, C.; Revenko, A. S.; Meijers, J. C.; Stroes, E. S.; Levi, M.; Monia, B. P. Inhibition of the Intrinsic Coagulation Pathway Factor XI by Antisense Oligonucleotides: A Novel Antithrombotic Strategy with Lowered Bleeding Risk Blood 2010, 116, 4684-4692 10.1182/blood-2010-04-277798
-
(2010)
Blood
, vol.116
, pp. 4684-4692
-
-
Zhang, H.1
Lowenberg, E.C.2
Crosby, J.R.3
MacLeod, A.R.4
Zhao, C.5
Gao, D.6
Black, C.7
Revenko, A.S.8
Meijers, J.C.9
Stroes, E.S.10
Levi, M.11
Monia, B.P.12
-
53
-
-
84920984860
-
Factor XI Antisense Oligonucleotide for Prevention of Venous Thrombosis
-
Bueller, H. R.; Bethune, C.; Bhanot, S.; Gailani, D.; Monia, B. P.; Raskob, G. E.; Segers, A.; Verhamme, P.; Weitz, J. I. Factor XI Antisense Oligonucleotide for Prevention of Venous Thrombosis N. Engl. J. Med. 2015, 372, 232-240 10.1056/NEJMoa1405760
-
(2015)
N. Engl. J. Med.
, vol.372
, pp. 232-240
-
-
Bueller, H.R.1
Bethune, C.2
Bhanot, S.3
Gailani, D.4
Monia, B.P.5
Raskob, G.E.6
Segers, A.7
Verhamme, P.8
Weitz, J.I.9
-
54
-
-
84861451481
-
Clinical Development of an Antisense Therapy for the Treatment of Transthyretin-Associated Polyneuropathy
-
Ackermann, E. J.; Guo, S.; Booten, S.; Alvarado, L.; Benson, M.; Hughes, S.; Monia, B. P. Clinical Development of an Antisense Therapy for the Treatment of Transthyretin-Associated Polyneuropathy Amyloid 2012, 19, 43-44 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
-
55
-
-
0031821074
-
In Vivo Distribution and Metabolism of a Phosphorothioate Oligonucleotide within Rat Liver after Intravenous Administration
-
Graham, M. J.; Crooke, S. T.; Monteith, D. K.; Cooper, S. R.; Lemonidis, K. M.; Stecker, K. K.; Martin, M. J.; Crooke, R. M. In Vivo Distribution and Metabolism of a Phosphorothioate Oligonucleotide within Rat Liver after Intravenous Administration J. Pharmacol. Exp. Ther. 1998, 286, 447-458
-
(1998)
J. Pharmacol. Exp. Ther.
, vol.286
, pp. 447-458
-
-
Graham, M.J.1
Crooke, S.T.2
Monteith, D.K.3
Cooper, S.R.4
Lemonidis, K.M.5
Stecker, K.K.6
Martin, M.J.7
Crooke, R.M.8
-
56
-
-
0035427644
-
Hepatic Distribution of a Phosphorothioate Oligodeoxynucleotide within Rodents Following Intravenous Administration
-
Graham, M. J.; Crooke, S. T.; Lemonidis, K. M.; Gaus, H. J.; Templin, M. V.; Crooke, R. M. Hepatic Distribution of a Phosphorothioate Oligodeoxynucleotide within Rodents Following Intravenous Administration Biochem. Pharmacol. 2001, 62, 297-306 10.1016/S0006-2952(01)00669-4
-
(2001)
Biochem. Pharmacol.
, vol.62
, pp. 297-306
-
-
Graham, M.J.1
Crooke, S.T.2
Lemonidis, K.M.3
Gaus, H.J.4
Templin, M.V.5
Crooke, R.M.6
-
57
-
-
84962181299
-
-
American Heart Association Scientific Sessions, November 7-11, Orlando, Florida, USA
-
Witztum, J. Compelling Results from Isis-Apo(a)-Lrx Phase 1/2a Study. American Heart Association Scientific Sessions 2015, November 7-11, Orlando, Florida, USA.
-
(2015)
Compelling Results from Isis-Apo(a)-Lrx Phase 1/2a Study
-
-
Witztum, J.1
-
58
-
-
0026018794
-
Partial Characterization of Low Density Lipoprotein Preparations Isolated from Fresh and Frozen Plasma after Radiolabeling by Seven Different Methods
-
Atsma, D. E.; Kempen, H. J.; Nieuwenhuizen, W.; van Hooft, F. M.; Pauwels, E. K. Partial Characterization of Low Density Lipoprotein Preparations Isolated from Fresh and Frozen Plasma after Radiolabeling by Seven Different Methods J. Lipid Res. 1991, 32, 173-181
-
(1991)
J. Lipid Res.
, vol.32
, pp. 173-181
-
-
Atsma, D.E.1
Kempen, H.J.2
Nieuwenhuizen, W.3
Van Hooft, F.M.4
Pauwels, E.K.5
-
59
-
-
33646244247
-
Targeted Suppression of an Amyloidogenic Transthyretin with Antisense Oligonucleotides
-
Benson, M. D.; Kluve-Beckerman, B.; Zeldenrust, S. R.; Siesky, A. M.; Bodenmiller, D. M.; Showalter, A. D.; Sloop, K. W. Targeted Suppression of an Amyloidogenic Transthyretin with Antisense Oligonucleotides Muscle Nerve 2006, 33, 609-618 10.1002/mus.20503
-
(2006)
Muscle Nerve
, vol.33
, pp. 609-618
-
-
Benson, M.D.1
Kluve-Beckerman, B.2
Zeldenrust, S.R.3
Siesky, A.M.4
Bodenmiller, D.M.5
Showalter, A.D.6
Sloop, K.W.7
|