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Items: 1 to 50 of 93

1.

Lipid Conjugates Enhance Endosomal Release of Antisense Oligonucleotides Into Cells.

Wang S, Allen N, Prakash TP, Liang XH, Crooke ST.

Nucleic Acid Ther. 2019 Jun 3. doi: 10.1089/nat.2019.0794. [Epub ahead of print]

PMID:
31158063
2.

Efficient Electroporation of Neuronal Cells Using Synthetic Oligonucleotides: Identifying Duplex RNA and Antisense Oligonucleotide Activators of Human Frataxin Expression.

Shen X, Beasley S, Putnam JN, Li Y, Prakash TP, Rigo F, Napierala M, Corey DR.

RNA. 2019 May 31. pii: rna.071290.119. doi: 10.1261/rna.071290.119. [Epub ahead of print]

3.

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 May 25. pii: gkz354. doi: 10.1093/nar/gkz354. [Epub ahead of print]

PMID:
31127296
4.

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 May 11. pii: gkz360. doi: 10.1093/nar/gkz360. [Epub ahead of print]

PMID:
31076766
5.

Duplex RNAs and ss-siRNAs Block RNA Foci Associated with Fuchs' Endothelial Corneal Dystrophy.

Hu J, Shen X, Rigo F, Prakash TP, Mootha VV, Corey DR.

Nucleic Acid Ther. 2019 Apr;29(2):73-81. doi: 10.1089/nat.2018.0764. Epub 2019 Jan 24.

PMID:
30676271
6.

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.

7.

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
8.

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.

9.

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
10.

Activating frataxin expression by single-stranded siRNAs targeting the GAA repeat expansion.

Shen X, Kilikevicius A, O'Reilly D, Prakash TP, Damha MJ, Rigo F, Corey DR.

Bioorg Med Chem Lett. 2018 Sep 15;28(17):2850-2855. doi: 10.1016/j.bmcl.2018.07.033. Epub 2018 Jul 21.

11.

Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells.

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

Mol Ther. 2018 May 2;26(5):1228-1240. doi: 10.1016/j.ymthe.2018.02.031. Epub 2018 Mar 6.

12.

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.

13.

Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat.

Li L, Shen X, Liu Z, Norrbom M, Prakash TP, O'Reilly D, Sharma VK, Damha MJ, Watts JK, Rigo F, Corey DR.

Nucleic Acid Ther. 2018 Feb;28(1):23-33. doi: 10.1089/nat.2017.0703. Epub 2018 Jan 17.

14.

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.

15.

Blood Pressure Lowering and Safety Improvements With Liver Angiotensinogen Inhibition in Models of Hypertension and Kidney Injury.

Mullick AE, Yeh ST, Graham MJ, Engelhardt JA, Prakash TP, Crooke RM.

Hypertension. 2017 Sep;70(3):566-576. doi: 10.1161/HYPERTENSIONAHA.117.09755. Epub 2017 Jul 17.

PMID:
28716988
16.

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.

17.

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.

18.

Recognition of c9orf72 Mutant RNA by Single-Stranded Silencing RNAs.

Hu J, Rigo F, Prakash TP, Corey DR.

Nucleic Acid Ther. 2017 Apr;27(2):87-94. doi: 10.1089/nat.2016.0655. Epub 2016 Dec 22.

19.

Limits of RNA 2'-OH Mimicry by Fluorine: Crystal Structure of Bacillus halodurans RNase H Bound to a 2'-FRNA:DNA Hybrid.

Pallan PS, Prakash TP, de Leon AR, Egli M.

Biochemistry. 2016 Sep 27;55(38):5321-5. doi: 10.1021/acs.biochem.6b00849. Epub 2016 Sep 14.

PMID:
27611889
20.

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
21.

Structural Analysis of Human Argonaute-2 Bound to a Modified siRNA Guide.

Schirle NT, Kinberger GA, Murray HF, Lima WF, Prakash TP, MacRae IJ.

J Am Chem Soc. 2016 Jul 20;138(28):8694-7. doi: 10.1021/jacs.6b04454. Epub 2016 Jul 12.

22.

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
23.

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.

24.

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
25.

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.

26.

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
27.

Hsp90 protein interacts with phosphorothioate oligonucleotides containing hydrophobic 2'-modifications and enhances antisense activity.

Liang XH, Shen W, Sun H, Kinberger GA, Prakash TP, Nichols JG, Crooke ST.

Nucleic Acids Res. 2016 May 5;44(8):3892-907. doi: 10.1093/nar/gkw144. Epub 2016 Mar 3.

28.

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
29.

Argonaute 2-dependent Regulation of Gene Expression by Single-stranded miRNA Mimics.

Matsui M, Prakash TP, Corey DR.

Mol Ther. 2016 May;24(5):946-55. doi: 10.1038/mt.2016.39. Epub 2016 Feb 23.

30.

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.

31.

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.

32.

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
33.

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
34.

Effect of 2'-O-[2-[2-(N,N-dimethylamino)ethoxy]ethyl] modification on activity of gapmer antisense oligonucleotides containing 2',4'-constrained 2'-O-ethyl nucleic acid.

Pandey SK, Nowak A, Perkins J, Ferng A, Prakash TP.

Bioorg Med Chem Lett. 2015 Apr 15;25(8):1688-1691. doi: 10.1016/j.bmcl.2015.03.004. Epub 2015 Mar 7.

PMID:
25804718
35.

Modulation of Splicing by Single-Stranded Silencing RNAs.

Liu J, Hu J, Hicks JA, Prakash TP, Corey DR.

Nucleic Acid Ther. 2015 Jun;25(3):113-20. doi: 10.1089/nat.2014.0527. Epub 2015 Mar 10.

36.

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.

37.

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.

38.

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.

39.

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
40.

TCP1 complex proteins interact with phosphorothioate oligonucleotides and can co-localize in oligonucleotide-induced nuclear bodies in mammalian cells.

Liang XH, Shen W, Sun H, Prakash TP, Crooke ST.

Nucleic Acids Res. 2014 Jul;42(12):7819-32. doi: 10.1093/nar/gku484. Epub 2014 May 26.

41.

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.

42.

Synthesis and biophysical properties of constrained D-altritol nucleic acids (cANA).

Migawa MT, Prakash TP, Vasquez G, Seth PP, Swayze EE.

Org Lett. 2013 Sep 6;15(17):4316-9. doi: 10.1021/ol401730d. Epub 2013 Aug 13.

PMID:
23937264
43.

ss-siRNAs allele selectively inhibit ataxin-3 expression: multiple mechanisms for an alternative gene silencing strategy.

Liu J, Yu D, Aiba Y, Pendergraff H, Swayze EE, Lima WF, Hu J, Prakash TP, Corey DR.

Nucleic Acids Res. 2013 Nov;41(20):9570-83. doi: 10.1093/nar/gkt693. Epub 2013 Aug 9.

44.

Lipid nanoparticles improve activity of single-stranded siRNA and gapmer antisense oligonucleotides in animals.

Prakash TP, Lima WF, Murray HM, Elbashir S, Cantley W, Foster D, Jayaraman M, Chappell AE, Manoharan M, Swayze EE, Crooke ST.

ACS Chem Biol. 2013 Jul 19;8(7):1402-6. doi: 10.1021/cb4001316. Epub 2013 May 2.

PMID:
23614580
45.

Transcriptional silencing by single-stranded RNAs targeting a noncoding RNA that overlaps a gene promoter.

Matsui M, Prakash TP, Corey DR.

ACS Chem Biol. 2013 Jan 18;8(1):122-6. doi: 10.1021/cb300490j. Epub 2012 Oct 24.

46.

Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.

Yu D, Pendergraff H, Liu J, Kordasiewicz HB, Cleveland DW, Swayze EE, Lima WF, Crooke ST, Prakash TP, Corey DR.

Cell. 2012 Aug 31;150(5):895-908. doi: 10.1016/j.cell.2012.08.002.

47.

Single-stranded siRNAs activate RNAi in animals.

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

Cell. 2012 Aug 31;150(5):883-94. doi: 10.1016/j.cell.2012.08.014.

48.

Structure and nuclease resistance of 2',4'-constrained 2'-O-methoxyethyl (cMOE) and 2'-O-ethyl (cEt) modified DNAs.

Pallan PS, Allerson CR, Berdeja A, Seth PP, Swayze EE, Prakash TP, Egli M.

Chem Commun (Camb). 2012 Aug 25;48(66):8195-7. doi: 10.1039/c2cc32286b. Epub 2012 May 22.

49.

Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing.

Rigo F, Hua Y, Chun SJ, Prakash TP, Krainer AR, Bennett CF.

Nat Chem Biol. 2012 Apr 15;8(6):555-61. doi: 10.1038/nchembio.939.

50.

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 TP, Norrbom M, Swayze EE, Leumann CJ, Seth PP.

Nucleic Acids Res. 2012 Jul;40(13):6135-43. doi: 10.1093/nar/gks273. Epub 2012 Mar 30.

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