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Items: 1 to 50 of 88

1.

Design And Synthesis Of An Azabicyclic Nucleoside Phosphoramidite For Oligonucleotide Antisense Constructs.

Salinas JC, Seth PP, Hanessian S.

Nucleosides Nucleotides Nucleic Acids. 2019 Aug 5:1-23. doi: 10.1080/15257770.2019.1646916. [Epub ahead of print]

PMID:
31380707
2.

Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle.

Prakash TP, Mullick AE, Lee RG, Yu J, Yeh ST, Low A, Chappell AE, Østergaard ME, Murray S, Gaus HJ, Swayze EE, Seth PP.

Nucleic Acids Res. 2019 Jul 9;47(12):6029-6044. doi: 10.1093/nar/gkz354.

3.

Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primates.

Østergaard ME, Jackson M, Low A, E Chappell A, G Lee R, Peralta RQ, Yu J, Kinberger GA, Dan A, Carty R, Tanowitz M, Anderson P, Kim TW, Fradkin L, Mullick AE, Murray S, Rigo F, Prakash TP, Bennett CF, Swayze EE, Gaus HJ, Seth PP.

Nucleic Acids Res. 2019 Jul 9;47(12):6045-6058. doi: 10.1093/nar/gkz360.

4.

Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index.

Shen W, De Hoyos CL, Migawa MT, Vickers TA, Sun H, Low A, Bell TA 3rd, Rahdar M, Mukhopadhyay S, Hart CE, Bell M, Riney S, Murray SF, Greenlee S, Crooke RM, Liang XH, Seth PP, Crooke ST.

Nat Biotechnol. 2019 Jun;37(6):640-650. doi: 10.1038/s41587-019-0106-2. Epub 2019 Apr 29.

PMID:
31036929
5.

Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteins.

Migawa MT, Shen W, Wan WB, Vasquez G, Oestergaard ME, Low A, De Hoyos CL, Gupta R, Murray S, Tanowitz M, Bell M, Nichols JG, Gaus H, Liang XH, Swayze EE, Crooke ST, Seth PP.

Nucleic Acids Res. 2019 Jun 20;47(11):5465-5479. doi: 10.1093/nar/gkz247.

6.

Selective tissue targeting of synthetic nucleic acid drugs.

Seth PP, Tanowitz M, Bennett CF.

J Clin Invest. 2019 Mar 1;129(3):915-925. doi: 10.1172/JCI125228. Epub 2019 Jan 28. Review.

7.

S-Acyl-2-Thioethyl: A Convenient Base-Labile Protecting Group for the Synthesis of siRNAs Containing 5'-Vinylphosphonate.

Nikan M, Li W, Kinberger GA, Seth PP, Swayze EE, Prakash TP.

Molecules. 2019 Jan 9;24(2). pii: E225. doi: 10.3390/molecules24020225.

8.

Characterization of the interactions of chemically-modified therapeutic nucleic acids with plasma proteins using a fluorescence polarization assay.

Gaus HJ, Gupta R, Chappell AE, Østergaard ME, Swayze EE, Seth PP.

Nucleic Acids Res. 2019 Feb 20;47(3):1110-1122. doi: 10.1093/nar/gky1260.

9.

Targeted delivery of antisense oligonucleotides to pancreatic β-cells.

Ämmälä C, Drury WJ 3rd, Knerr L, Ahlstedt I, Stillemark-Billton P, Wennberg-Huldt C, Andersson EM, Valeur E, Jansson-Löfmark R, Janzén D, Sundström L, Meuller J, Claesson J, Andersson P, Johansson C, Lee RG, Prakash TP, Seth PP, Monia BP, Andersson S.

Sci Adv. 2018 Oct 17;4(10):eaat3386. doi: 10.1126/sciadv.aat3386. eCollection 2018 Oct.

10.

Evaluation of the effect of 2'-O-methyl, fluoro hexitol, bicyclo and Morpholino nucleic acid modifications on potency of GalNAc conjugated antisense oligonucleotides in mice.

Prakash TP, Yu J, Kinberger GA, Low A, Jackson M, Rigo F, Swayze EE, Seth PP.

Bioorg Med Chem Lett. 2018 Dec 15;28(23-24):3774-3779. doi: 10.1016/j.bmcl.2018.10.011. Epub 2018 Oct 10.

PMID:
30342955
11.

Huntingtin suppression restores cognitive function in a mouse model of Huntington's disease.

Southwell AL, Kordasiewicz HB, Langbehn D, Skotte NH, Parsons MP, Villanueva EB, Caron NS, Østergaard ME, Anderson LM, Xie Y, Cengio LD, Findlay-Black H, Doty CN, Fitsimmons B, Swayze EE, Seth PP, Raymond LA, Frank Bennett C, Hayden MR.

Sci Transl Med. 2018 Oct 3;10(461). pii: eaar3959. doi: 10.1126/scitranslmed.aar3959.

PMID:
30282695
12.

Conception and Synthesis of Oxabicyclic Nucleoside Phosphonates as Internucleotidic Phosphate Surrogates in Antisense Oligonucleotide Constructs.

Salinas JC, Yu J, Østergaard M, Seth PP, Hanessian S.

Org Lett. 2018 Sep 7;20(17):5296-5299. doi: 10.1021/acs.orglett.8b02233. Epub 2018 Aug 27.

PMID:
30146887
13.

Studies directed toward the asialoglycoprotein receptor mediated delivery of 5-fluoro-2'-deoxyuridine for hepatocellular carcinoma.

Rico L, Østergaard ME, Bell M, Seth PP, Hanessian S.

Bioorg Med Chem Lett. 2018 Aug 15;28(15):2652-2654. doi: 10.1016/j.bmcl.2018.06.002. Epub 2018 Jun 1.

PMID:
30042045
14.

Structural Determinants for the Interactions of Chemically Modified Nucleic Acids with the Stabilin-2 Clearance Receptor.

Gaus H, Miller CM, Seth PP, Harris EN.

Biochemistry. 2018 Apr 10;57(14):2061-2064. doi: 10.1021/acs.biochem.8b00126. Epub 2018 Mar 30.

15.

Endosomal Escape of Antisense Oligonucleotides Internalized by Stabilin Receptors Is Regulated by Rab5C and EEA1 During Endosomal Maturation.

Miller CM, Wan WB, Seth PP, Harris EN.

Nucleic Acid Ther. 2018 Apr;28(2):86-96. doi: 10.1089/nat.2017.0694. Epub 2018 Feb 13.

16.

Receptor-Mediated Uptake of Phosphorothioate Antisense Oligonucleotides in Different Cell Types of the Liver.

Miller CM, Tanowitz M, Donner AJ, Prakash TP, Swayze EE, Harris EN, Seth PP.

Nucleic Acid Ther. 2018 Jun;28(3):119-127. doi: 10.1089/nat.2017.0709. Epub 2018 Feb 9. Review.

17.

Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes.

Tanowitz M, Hettrick L, Revenko A, Kinberger GA, Prakash TP, Seth PP.

Nucleic Acids Res. 2017 Dec 1;45(21):12388-12400. doi: 10.1093/nar/gkx960.

18.

Fluorinated Nucleotide Modifications Modulate Allele Selectivity of SNP-Targeting Antisense Oligonucleotides.

Østergaard ME, Nichols J, Dwight TA, Lima W, Jung ME, Swayze EE, Seth PP.

Mol Ther Nucleic Acids. 2017 Jun 16;7:20-30. doi: 10.1016/j.omtn.2017.02.001. Epub 2017 Feb 9.

19.

Identification of metabolically stable 5΄-phosphate analogs that support single-stranded siRNA activity.

Prakash TP, Lima WF, Murray HM, Li W, Kinberger GA, Chappell AE, Gaus H, Seth PP, Bhat B, Crooke ST, Swayze EE.

Nucleic Acids Res. 2017 Jun 20;45(11):6994. doi: 10.1093/nar/gkx381. No abstract available.

20.

Co-Administration of an Excipient Oligonucleotide Helps Delineate Pathways of Productive and Nonproductive Uptake of Phosphorothioate Antisense Oligonucleotides in the Liver.

Donner AJ, Wancewicz EV, Murray HM, Greenlee S, Post N, Bell M, Lima WF, Swayze EE, Seth PP.

Nucleic Acid Ther. 2017 Aug;27(4):209-220. doi: 10.1089/nat.2017.0662. Epub 2017 Apr 27.

PMID:
28448194
21.

Antisense Reduction of Mutant COMP Reduces Growth Plate Chondrocyte Pathology.

Posey KL, Coustry F, Veerisetty AC, Hossain M, Gattis D, Booten S, Alcorn JL, Seth PP, Hecht JT.

Mol Ther. 2017 Mar 1;25(3):705-714. doi: 10.1016/j.ymthe.2016.12.024. Epub 2017 Feb 3.

22.

Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor.

Schmidt K, Prakash TP, Donner AJ, Kinberger GA, Gaus HJ, Low A, Østergaard ME, Bell M, Swayze EE, Seth PP.

Nucleic Acids Res. 2017 Mar 17;45(5):2294-2306. doi: 10.1093/nar/gkx060.

23.

A novel humanized mouse model of Huntington disease for preclinical development of therapeutics targeting mutant huntingtin alleles.

Southwell AL, Skotte NH, Villanueva EB, Østergaard ME, Gu X, Kordasiewicz HB, Kay C, Cheung D, Xie Y, Waltl S, Dal Cengio L, Findlay-Black H, Doty CN, Petoukhov E, Iworima D, Slama R, Ooi J, Pouladi MA, Yang XW, Swayze EE, Seth PP, Hayden MR.

Hum Mol Genet. 2017 Mar 15;26(6):1115-1132. doi: 10.1093/hmg/ddx021.

PMID:
28104789
24.

Disposition and Pharmacokinetics of a GalNAc3-Conjugated Antisense Oligonucleotide Targeting Human Lipoprotein (a) in Monkeys.

Yu RZ, Gunawan R, Post N, Zanardi T, Hall S, Burkey J, Kim TW, Graham MJ, Prakash TP, Seth PP, Swayze EE, Geary RS, Henry SP, Wang Y.

Nucleic Acid Ther. 2016 Dec;26(6):372-380. Epub 2016 Aug 8.

PMID:
27500733
25.

Novel Targeted Therapy for Precursor B Cell Acute Lymphoblastic Leukemia: anti-CD22 Antibody-MXD3 Antisense Oligonucleotide Conjugate.

Satake N, Duong C, Yoshida S, Oestergaard M, Chen C, Peralta R, Guo S, Seth PP, Li Y, Beckett L, Chung J, Nolta J, Nitin N, Tuscano JM.

Mol Med. 2016 Oct;22:632-642. doi: 10.2119/molmed.2015.00210. Epub 2016 Jul 22.

26.

The Medicinal Chemistry of Therapeutic Oligonucleotides.

Wan WB, Seth PP.

J Med Chem. 2016 Nov 10;59(21):9645-9667. Epub 2016 Jul 29. Review.

PMID:
27434100
27.

Conjugation of mono and di-GalNAc sugars enhances the potency of antisense oligonucleotides via ASGR mediated delivery to hepatocytes.

Kinberger GA, Prakash TP, Yu J, Vasquez G, Low A, Chappell A, Schmidt K, Murray HM, Gaus H, Swayze EE, Seth PP.

Bioorg Med Chem Lett. 2016 Aug 1;26(15):3690-3. doi: 10.1016/j.bmcl.2016.05.084. Epub 2016 May 28.

PMID:
27268307
28.

Elucidation of the Biotransformation Pathways of a Galnac3-conjugated Antisense Oligonucleotide in Rats and Monkeys.

Shemesh CS, Yu RZ, Gaus HJ, Greenlee S, Post N, Schmidt K, Migawa MT, Seth PP, Zanardi TA, Prakash TP, Swayze EE, Henry SP, Wang Y.

Mol Ther Nucleic Acids. 2016 May 10;5:e319. doi: 10.1038/mtna.2016.31.

29.

Synergistic effect of phosphorothioate, 5'-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA.

Prakash TP, Kinberger GA, Murray HM, Chappell A, Riney S, Graham MJ, Lima WF, Swayze EE, Seth PP.

Bioorg Med Chem Lett. 2016 Jun 15;26(12):2817-2820. doi: 10.1016/j.bmcl.2016.04.063. Epub 2016 Apr 27.

PMID:
27161280
30.

Disposition and Pharmacology of a GalNAc3-conjugated ASO Targeting Human Lipoprotein (a) in Mice.

Yu RZ, Graham MJ, Post N, Riney S, Zanardi T, Hall S, Burkey J, Shemesh CS, Prakash TP, Seth PP, Swayze EE, Geary RS, Wang Y, Henry S.

Mol Ther Nucleic Acids. 2016 May 3;5:e317. doi: 10.1038/mtna.2016.26.

31.

Synthesis and biological evaluation of sialyl-oligonucleotide conjugates targeting leukocyte B trans-membranal receptor CD22 as delivery agents for nucleic acid drugs.

St-Pierre G, Pal S, Østergaard ME, Zhou T, Yu J, Tanowitz M, Seth PP, Hanessian S.

Bioorg Med Chem. 2016 Jun 1;24(11):2397-409. doi: 10.1016/j.bmc.2016.03.047. Epub 2016 Mar 29.

PMID:
27117693
32.

Pharmacokinetic and Pharmacodynamic Investigations of ION-353382, a Model Antisense Oligonucleotide: Using Alpha-2-Macroglobulin and Murinoglobulin Double-Knockout Mice.

Shemesh CS, Yu RZ, Gaus HJ, Seth PP, Swayze EE, Bennett FC, Geary RS, Henry SP, Wang Y.

Nucleic Acid Ther. 2016 Aug;26(4):223-35. doi: 10.1089/nat.2016.0607. Epub 2016 Mar 31.

PMID:
27031383
33.

A convenient synthesis of 5'-triantennary N-acetyl-galactosamine clusters based on nitromethanetrispropionic acid.

Migawa MT, Prakash TP, Vasquez G, Wan WB, Yu J, Kinberger GA, Østergaard ME, Swayze EE, Seth PP.

Bioorg Med Chem Lett. 2016 May 1;26(9):2194-7. doi: 10.1016/j.bmcl.2016.03.070. Epub 2016 Mar 17.

PMID:
27025342
34.

Comprehensive Structure-Activity Relationship of Triantennary N-Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes.

Prakash TP, Yu J, Migawa MT, Kinberger GA, Wan WB, Østergaard ME, Carty RL, Vasquez G, Low A, Chappell A, Schmidt K, Aghajan M, Crosby J, Murray HM, Booten SL, Hsiao J, Soriano A, Machemer T, Cauntay P, Burel SA, Murray SF, Gaus H, Graham MJ, Swayze EE, Seth PP.

J Med Chem. 2016 Mar 24;59(6):2718-33. doi: 10.1021/acs.jmedchem.5b01948. Epub 2016 Mar 9.

PMID:
26914862
35.

Stabilin-1 and Stabilin-2 are specific receptors for the cellular internalization of phosphorothioate-modified antisense oligonucleotides (ASOs) in the liver.

Miller CM, Donner AJ, Blank EE, Egger AW, Kellar BM, Østergaard ME, Seth PP, Harris EN.

Nucleic Acids Res. 2016 Apr 7;44(6):2782-94. doi: 10.1093/nar/gkw112. Epub 2016 Feb 22.

36.

Design, synthesis, and duplex-stabilizing properties of conformationally constrained tricyclic analogues of LNA.

Giacometti RD, Salinas JC, Østergaard ME, Swayze EE, Seth PP, Hanessian S.

Org Biomol Chem. 2016 Feb 14;14(6):2034-40. doi: 10.1039/c5ob02576a.

PMID:
26765794
37.

Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts.

Burel SA, Hart CE, Cauntay P, Hsiao J, Machemer T, Katz M, Watt A, Bui HH, Younis H, Sabripour M, Freier SM, Hung G, Dan A, Prakash TP, Seth PP, Swayze EE, Bennett CF, Crooke ST, Henry SP.

Nucleic Acids Res. 2016 Mar 18;44(5):2093-109. doi: 10.1093/nar/gkv1210. Epub 2015 Nov 8.

38.

Solid-phase synthesis of 5'-triantennary N-acetylgalactosamine conjugated antisense oligonucleotides using phosphoramidite chemistry.

Prakash TP, Brad Wan W, Low A, Yu J, Chappell AE, Gaus H, Kinberger GA, Østergaard ME, Migawa MT, Swayze EE, Seth PP.

Bioorg Med Chem Lett. 2015 Oct 1;25(19):4127-30. doi: 10.1016/j.bmcl.2015.08.019. Epub 2015 Aug 8.

PMID:
26299345
39.

DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing.

Nishina K, Piao W, Yoshida-Tanaka K, Sujino Y, Nishina T, Yamamoto T, Nitta K, Yoshioka K, Kuwahara H, Yasuhara H, Baba T, Ono F, Miyata K, Miyake K, Seth PP, Low A, Yoshida M, Bennett CF, Kataoka K, Mizusawa H, Obika S, Yokota T.

Nat Commun. 2015 Aug 10;6:7969. doi: 10.1038/ncomms8969.

40.

Allele-Selective Inhibition of Mutant Huntingtin with 2-Thio- and C5- Triazolylphenyl-Deoxythymidine-Modified Antisense Oligonucleotides.

Østergaard ME, Kumar P, Nichols J, Watt A, Sharma PK, Nielsen P, Seth PP.

Nucleic Acid Ther. 2015 Oct;25(5):266-74. doi: 10.1089/nat.2015.0547. Epub 2015 Jul 29.

PMID:
26222265
41.

Efficient Synthesis and Biological Evaluation of 5'-GalNAc Conjugated Antisense Oligonucleotides.

Østergaard ME, Yu J, Kinberger GA, Wan WB, Migawa MT, Vasquez G, Schmidt K, Gaus HJ, Murray HM, Low A, Swayze EE, Prakash TP, Seth PP.

Bioconjug Chem. 2015 Aug 19;26(8):1451-5. doi: 10.1021/acs.bioconjchem.5b00265. Epub 2015 Jun 10.

PMID:
26011654
42.

Identification of metabolically stable 5'-phosphate analogs that support single-stranded siRNA activity.

Prakash TP, Lima WF, Murray HM, Li W, Kinberger GA, Chappell AE, Gaus H, Seth PP, Bhat B, Crooke ST, Swayze EE.

Nucleic Acids Res. 2015 Mar 31;43(6):2993-3011. doi: 10.1093/nar/gkv162. Epub 2015 Mar 9. Erratum in: Nucleic Acids Res. 2017 Jun 20;45(11):6994.

43.

Biophysical and biological characterization of hairpin and molecular beacon RNase H active antisense oligonucleotides.

Østergaard ME, Thomas G, Koller E, Southwell AL, Hayden MR, Seth PP.

ACS Chem Biol. 2015 May 15;10(5):1227-33. doi: 10.1021/cb500880f. Epub 2015 Feb 13.

PMID:
25654188
44.

Synthesis, biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages.

Wan WB, Migawa MT, Vasquez G, Murray HM, Nichols JG, Gaus H, Berdeja A, Lee S, Hart CE, Lima WF, Swayze EE, Seth PP.

Nucleic Acids Res. 2014 Dec 16;42(22):13456-68. doi: 10.1093/nar/gku1115. Epub 2014 Nov 14.

45.

Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.

Skotte NH, Southwell AL, Østergaard ME, Carroll JB, Warby SC, Doty CN, Petoukhov E, Vaid K, Kordasiewicz H, Watt AT, Freier SM, Hung G, Seth PP, Bennett CF, Swayze EE, Hayden MR.

PLoS One. 2014 Sep 10;9(9):e107434. doi: 10.1371/journal.pone.0107434. eCollection 2014.

46.

Antisense oligonucleotide-based therapies for diseases caused by pre-mRNA processing defects.

Rigo F, Seth PP, Bennett CF.

Adv Exp Med Biol. 2014;825:303-52. doi: 10.1007/978-1-4939-1221-6_9. Review.

PMID:
25201110
47.

Comparison of duplex stabilizing properties of 2'-fluorinated nucleic acid analogues with furanose and non-furanose sugar rings.

Østergaard ME, Dwight T, Berdeja A, Swayze EE, Jung ME, Seth PP.

J Org Chem. 2014 Sep 19;79(18):8877-81. doi: 10.1021/jo501381q. Epub 2014 Aug 27.

48.

In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.

Southwell AL, Skotte NH, Kordasiewicz HB, Østergaard ME, Watt AT, Carroll JB, Doty CN, Villanueva EB, Petoukhov E, Vaid K, Xie Y, Freier SM, Swayze EE, Seth PP, Bennett CF, Hayden MR.

Mol Ther. 2014 Dec;22(12):2093-106. doi: 10.1038/mt.2014.153. Epub 2014 Aug 7.

49.

Differential effects on allele selective silencing of mutant huntingtin by two stereoisomers of α,β-constrained nucleic acid.

Østergaard ME, Gerland B, Escudier JM, Swayze EE, Seth PP.

ACS Chem Biol. 2014 Sep 19;9(9):1975-9. doi: 10.1021/cb5003027. Epub 2014 Jul 25.

PMID:
25050989
50.

Synthesis and duplex-stabilizing properties of fluorinated N-methanocarbathymidine analogues locked in the C3'-endo conformation.

Jung ME, Dwight TA, Vigant F, Østergaard ME, Swayze EE, Seth PP.

Angew Chem Int Ed Engl. 2014 Sep 8;53(37):9893-7. doi: 10.1002/anie.201405283. Epub 2014 Jul 15.

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