Format
Sort by
Items per page

Send to

Choose Destination

Search results

Items: 1 to 50 of 123

1.

Antisense oligonucleotides extend survival of prion-infected mice.

Raymond GJ, Zhao HT, Race B, Raymond LD, Williams K, Swayze EE, Graffam S, Le J, Caron T, Stathopoulos J, O'Keefe R, Lubke LL, Reidenbach AG, Kraus A, Schreiber SL, Mazur C, Cabin DE, Carroll JB, Minikel EV, Kordasiewicz H, Caughey B, Vallabh SM.

JCI Insight. 2019 Jul 30;5. pii: 131175. doi: 10.1172/jci.insight.131175.

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.

Targeting Huntingtin Expression in Patients with Huntington's Disease.

Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft C, Barker RA, Blair NF, Craufurd D, Priller J, Rickards H, Rosser A, Kordasiewicz HB, Czech C, Swayze EE, Norris DA, Baumann T, Gerlach I, Schobel SA, Paz E, Smith AV, Bennett CF, Lane RM; Phase 1–2a IONIS-HTTRx Study Site Teams.

N Engl J Med. 2019 Jun 13;380(24):2307-2316. doi: 10.1056/NEJMoa1900907. Epub 2019 May 6.

PMID:
31059641
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.

A modular analysis of microglia gene expression, insights into the aged phenotype.

Cho CE, Damle SS, Wancewicz EV, Mukhopadhyay S, Hart CE, Mazur C, Swayze EE, Kamme F.

BMC Genomics. 2019 Feb 28;20(1):164. doi: 10.1186/s12864-019-5549-9.

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.

Antisense oligonucleotide-mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles.

Friedrich J, Kordasiewicz HB, O'Callaghan B, Handler HP, Wagener C, Duvick L, Swayze EE, Rainwater O, Hofstra B, Benneyworth M, Nichols-Meade T, Yang P, Chen Z, Ortiz JP, Clark HB, Öz G, Larson S, Zoghbi HY, Henzler C, Orr HT.

JCI Insight. 2018 Nov 2;3(21). pii: 123193. doi: 10.1172/jci.insight.123193.

10.

Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7.

Niu C, Prakash TP, Kim A, Quach JL, Huryn LA, Yang Y, Lopez E, Jazayeri A, Hung G, Sopher BL, Brooks BP, Swayze EE, Bennett CF, La Spada AR.

Sci Transl Med. 2018 Oct 31;10(465). pii: eaap8677. doi: 10.1126/scitranslmed.aap8677.

PMID:
30381411
11.

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
12.

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
13.

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models.

McCampbell A, Cole T, Wegener AJ, Tomassy GS, Setnicka A, Farley BJ, Schoch KM, Hoye ML, Shabsovich M, Sun L, Luo Y, Zhang M, Comfort N, Wang B, Amacker J, Thankamony S, Salzman DW, Cudkowicz M, Graham DL, Bennett CF, Kordasiewicz HB, Swayze EE, Miller TM.

J Clin Invest. 2018 Aug 1;128(8):3558-3567. doi: 10.1172/JCI99081. Epub 2018 Jul 16.

14.

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.

15.

PMP22 antisense oligonucleotides reverse Charcot-Marie-Tooth disease type 1A features in rodent models.

Zhao HT, Damle S, Ikeda-Lee K, Kuntz S, Li J, Mohan A, Kim A, Hung G, Scheideler MA, Scherer SS, Svaren J, Swayze EE, Kordasiewicz HB.

J Clin Invest. 2018 Jan 2;128(1):359-368. doi: 10.1172/JCI96499. Epub 2017 Dec 4.

16.

Antisense oligonucleotides selectively suppress target RNA in nociceptive neurons of the pain system and can ameliorate mechanical pain.

Mohan A, Fitzsimmons B, Zhao HT, Jiang Y, Mazur C, Swayze EE, Kordasiewicz HB.

Pain. 2018 Jan;159(1):139-149. doi: 10.1097/j.pain.0000000000001074.

PMID:
28976422
17.

LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson's Disease Mouse Model.

Zhao HT, John N, Delic V, Ikeda-Lee K, Kim A, Weihofen A, Swayze EE, Kordasiewicz HB, West AB, Volpicelli-Daley LA.

Mol Ther Nucleic Acids. 2017 Sep 15;8:508-519. doi: 10.1016/j.omtn.2017.08.002. Epub 2017 Aug 10.

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.

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.

22.

Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy.

DeVos SL, Miller RL, Schoch KM, Holmes BB, Kebodeaux CS, Wegener AJ, Chen G, Shen T, Tran H, Nichols B, Zanardi TA, Kordasiewicz HB, Swayze EE, Bennett CF, Diamond MI, Miller TM.

Sci Transl Med. 2017 Jan 25;9(374). pii: eaag0481. doi: 10.1126/scitranslmed.aag0481.

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.

Tcf4 Regulates Synaptic Plasticity, DNA Methylation, and Memory Function.

Kennedy AJ, Rahn EJ, Paulukaitis BS, Savell KE, Kordasiewicz HB, Wang J, Lewis JW, Posey J, Strange SK, Guzman-Karlsson MC, Phillips SE, Decker K, Motley ST, Swayze EE, Ecker DJ, Michael TP, Day JJ, Sweatt JD.

Cell Rep. 2016 Sep 6;16(10):2666-2685. doi: 10.1016/j.celrep.2016.08.004. Epub 2016 Aug 25.

25.

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
26.

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
27.

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.

28.

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
29.

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.

30.

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
31.

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
32.

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
33.

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
34.

Synthetic CRISPR RNA-Cas9-guided genome editing in human cells.

Rahdar M, McMahon MA, Prakash TP, Swayze EE, Bennett CF, Cleveland DW.

Proc Natl Acad Sci U S A. 2015 Dec 22;112(51):E7110-7. doi: 10.1073/pnas.1520883112. Epub 2015 Nov 16.

35.

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.

36.

Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1.

Pandey SK, Wheeler TM, Justice SL, Kim A, Younis HS, Gattis D, Jauvin D, Puymirat J, Swayze EE, Freier SM, Bennett CF, Thornton CA, MacLeod AR.

J Pharmacol Exp Ther. 2015 Nov;355(2):329-40. doi: 10.1124/jpet.115.226969. Epub 2015 Sep 1.

37.

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
38.

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
39.

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.

40.

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.

41.

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.

42.

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.

43.

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.

44.

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
45.

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.

46.

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice.

Prakash TP, Graham MJ, Yu J, Carty R, Low A, Chappell A, Schmidt K, Zhao C, Aghajan M, Murray HF, Riney S, Booten SL, Murray SF, Gaus H, Crosby J, Lima WF, Guo S, Monia BP, Swayze EE, Seth PP.

Nucleic Acids Res. 2014 Jul;42(13):8796-807. doi: 10.1093/nar/gku531. Epub 2014 Jul 3.

47.

Allele-selective inhibition of mutant atrophin-1 expression by duplex and single-stranded RNAs.

Hu J, Liu J, Narayanannair KJ, Lackey JG, Kuchimanchi S, Rajeev KG, Manoharan M, Swayze EE, Lima WF, Prakash TP, Xiang Q, Martinez C, Corey DR.

Biochemistry. 2014 Jul 22;53(28):4510-8. doi: 10.1021/bi500610r. Epub 2014 Jul 11.

48.

Synthesis and antisense properties of 2'-O-(2S-methoxypropyl)-RNA-modified gapmer antisense oligonucleotides.

Yu J, Pandey SK, Khatri H, Prakash TP, Swayze EE, Seth PP.

ChemMedChem. 2014 Sep;9(9):2040-4. doi: 10.1002/cmdc.201402099. Epub 2014 May 28.

PMID:
24891270
49.

Exploring the effect of sequence length and composition on allele-selective inhibition of human huntingtin expression by single-stranded silencing RNAs.

Hu J, Liu J, Yu D, Aiba Y, Lee S, Pendergraff H, Boubaker J, Artates JW, Lagier-Tourenne C, Lima WF, Swayze EE, Prakash TP, Corey DR.

Nucleic Acid Ther. 2014 Jun;24(3):199-209. doi: 10.1089/nat.2013.0476. Epub 2014 Apr 2.

50.

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.

Østergaard ME, Southwell AL, Kordasiewicz H, Watt AT, Skotte NH, Doty CN, Vaid K, Villanueva EB, Swayze EE, Bennett CF, Hayden MR, Seth PP.

Nucleic Acids Res. 2013 Nov;41(21):9634-50. doi: 10.1093/nar/gkt725. Epub 2013 Aug 19.

Supplemental Content

Loading ...
Support Center