Format
Sort by
Items per page

Send to

Choose Destination

Search results

Items: 1 to 50 of 77

1.

Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin.

Didiot MC, Ferguson CM, Ly S, Coles AH, Smith AO, Bicknell AA, Hall LM, Sapp E, Echeverria D, Pai AA, DiFiglia M, Moore MJ, Hayward LJ, Aronin N, Khvorova A.

Cell Rep. 2018 Sep 4;24(10):2553-2560.e5. doi: 10.1016/j.celrep.2018.07.106.

2.

Functional features defining the efficacy of cholesterol-conjugated, self-deliverable, chemically modified siRNAs.

Shmushkovich T, Monopoli KR, Homsy D, Leyfer D, Betancur-Boissel M, Khvorova A, Wolfson AD.

Nucleic Acids Res. 2018 Aug 29. doi: 10.1093/nar/gky745. [Epub ahead of print]

PMID:
30169779
3.

Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier.

Godinho BMDC, Henninger N, Bouley J, Alterman JF, Haraszti RA, Gilbert JW, Sapp E, Coles AH, Biscans A, Nikan M, Echeverria D, DiFiglia M, Aronin N, Khvorova A.

Mol Ther. 2018 Aug 8. pii: S1525-0016(18)30376-9. doi: 10.1016/j.ymthe.2018.08.005. [Epub ahead of print]

PMID:
30143435
4.

Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing.

Mir A, Alterman JF, Hassler MR, Debacker AJ, Hudgens E, Echeverria D, Brodsky MH, Khvorova A, Watts JK, Sontheimer EJ.

Nat Commun. 2018 Jul 6;9(1):2641. doi: 10.1038/s41467-018-05073-z.

5.

Optimized Cholesterol-siRNA Chemistry Improves Productive Loading onto Extracellular Vesicles.

Haraszti RA, Miller R, Didiot MC, Biscans A, Alterman JF, Hassler MR, Roux L, Echeverria D, Sapp E, DiFiglia M, Aronin N, Khvorova A.

Mol Ther. 2018 Aug 1;26(8):1973-1982. doi: 10.1016/j.ymthe.2018.05.024. Epub 2018 Jun 21.

PMID:
29937418
6.

Novel Cluster and Monomer-Based GalNAc Structures Induce Effective Uptake of siRNAs in Vitro and in Vivo.

Sharma VK, Osborn MF, Hassler MR, Echeverria D, Ly S, Ulashchik EA, Martynenko-Makaev YV, Shmanai VV, Zatsepin TS, Khvorova A, Watts JK.

Bioconjug Chem. 2018 Jul 18;29(7):2478-2488. doi: 10.1021/acs.bioconjchem.8b00365. Epub 2018 Jul 2.

PMID:
29898368
7.

Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain.

Sanchez-Ramos J, Song S, Kong X, Foroutan P, Martinez G, Dominguez-Viqueria W, Mohapatra S, Mohapatra S, Haraszti RA, Khvorova A, Aronin N, Sava V.

J Drug Deliv Sci Technol. 2018 Feb;43:453-460. doi: 10.1016/j.jddst.2017.11.013. Epub 2017 Nov 21.

8.

Improving siRNA Delivery In Vivo Through Lipid Conjugation.

Osborn MF, Khvorova A.

Nucleic Acid Ther. 2018 Jun;28(3):128-136. doi: 10.1089/nat.2018.0725. Epub 2018 May 10.

PMID:
29746209
9.

Hydrophobicity of Lipid-Conjugated siRNAs Predicts Productive Loading to Small Extracellular Vesicles.

Biscans A, Haraszti RA, Echeverria D, Miller R, Didiot MC, Nikan M, Roux L, Aronin N, Khvorova A.

Mol Ther. 2018 Jun 6;26(6):1520-1528. doi: 10.1016/j.ymthe.2018.03.019. Epub 2018 Apr 4.

PMID:
29699940
10.

Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs.

Osborn MF, Coles AH, Golebiowski D, Echeverria D, Moazami MP, Watts JK, Sena-Esteves M, Khvorova A.

Mol Cancer Ther. 2018 Jun;17(6):1251-1258. doi: 10.1158/1535-7163.MCT-17-1144. Epub 2018 Apr 13.

PMID:
29654062
11.

Editorial: Nucleic Acids Research and Nucleic Acid Therapeutics.

Stoddard BL, Khvorova A, Corey DR, Dynan WS, Fox KR.

Nucleic Acids Res. 2018 Feb 28;46(4):1563-1564. doi: 10.1093/nar/gky059. No abstract available.

12.

Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo.

Hassler MR, Turanov AA, Alterman JF, Haraszti RA, Coles AH, Osborn MF, Echeverria D, Nikan M, Salomon WE, Roux L, Godinho BMDC, Davis SM, Morrissey DV, Zamore PD, Karumanchi SA, Moore MJ, Aronin N, Khvorova A.

Nucleic Acids Res. 2018 Mar 16;46(5):2185-2196. doi: 10.1093/nar/gky037.

13.

Loading of Extracellular Vesicles with Hydrophobically Modified siRNAs.

Didiot MC, Haraszti RA, Aronin N, Khvorova A.

Methods Mol Biol. 2018;1740:199-214. doi: 10.1007/978-1-4939-7652-2_16.

PMID:
29388146
14.

Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay.

Godinho BMDC, Gilbert JW, Haraszti RA, Coles AH, Biscans A, Roux L, Nikan M, Echeverria D, Hassler M, Khvorova A.

Nucleic Acid Ther. 2017 Dec;27(6):323-334. doi: 10.1089/nat.2017.0690. Epub 2017 Oct 12.

PMID:
29022758
15.

A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons in vitro with Oligonucleotide Therapeutics.

Alterman JF, Coles AH, Hall LM, Aronin N, Khvorova A, Didiot MC.

Bio Protoc. 2017 Aug 20;7(16). pii: e2501. doi: 10.21769/BioProtoc.2501.

16.

Loading of Extracellular Vesicles with Chemically Stabilized Hydrophobic siRNAs for the Treatment of Disease in the Central Nervous System.

Haraszti RA, Coles A, Aronin N, Khvorova A, Didiot MC.

Bio Protoc. 2017 Jun 20;7(20). pii: e2338. doi: 10.21769/BioProtoc.2338.

17.

Synthesis and biological properties of triazole-linked locked nucleic acid.

Sharma VK, Singh SK, Krishnamurthy PM, Alterman JF, Haraszti RA, Khvorova A, Prasad AK, Watts JK.

Chem Commun (Camb). 2017 Aug 3;53(63):8906-8909. doi: 10.1039/c7cc04092j.

PMID:
28736781
18.

5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo.

Haraszti RA, Roux L, Coles AH, Turanov AA, Alterman JF, Echeverria D, Godinho BMDC, Aronin N, Khvorova A.

Nucleic Acids Res. 2017 Jul 27;45(13):7581-7592. doi: 10.1093/nar/gkx507.

19.

Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable O-Phosphocholine-N-docosahexaenoyl-l-serine siRNA Conjugate in Mouse Brain.

Nikan M, Osborn MF, Coles AH, Biscans A, Godinho BMDC, Haraszti RA, Sapp E, Echeverria D, DiFiglia M, Aronin N, Khvorova A.

Bioconjug Chem. 2017 Jun 21;28(6):1758-1766. doi: 10.1021/acs.bioconjchem.7b00226. Epub 2017 May 10.

20.

The chemical evolution of oligonucleotide therapies of clinical utility.

Khvorova A, Watts JK.

Nat Biotechnol. 2017 Mar;35(3):238-248. doi: 10.1038/nbt.3765. Epub 2017 Feb 27. Review.

21.

Oligonucleotide Therapeutics - A New Class of Cholesterol-Lowering Drugs.

Khvorova A.

N Engl J Med. 2017 Jan 5;376(1):4-7. doi: 10.1056/NEJMp1614154. No abstract available.

PMID:
28052224
22.

Visualization of self-delivering hydrophobically modified siRNA cellular internalization.

Ly S, Navaroli DM, Didiot MC, Cardia J, Pandarinathan L, Alterman JF, Fogarty K, Standley C, Lifshitz LM, Bellve KD, Prot M, Echeverria D, Corvera S, Khvorova A.

Nucleic Acids Res. 2017 Jan 9;45(1):15-25. doi: 10.1093/nar/gkw1005. Epub 2016 Nov 28.

23.

High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources.

Haraszti RA, Didiot MC, Sapp E, Leszyk J, Shaffer SA, Rockwell HE, Gao F, Narain NR, DiFiglia M, Kiebish MA, Aronin N, Khvorova A.

J Extracell Vesicles. 2016 Nov 17;5:32570. doi: 10.3402/jev.v5.32570. eCollection 2016.

24.

Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing.

Didiot MC, Hall LM, Coles AH, Haraszti RA, Godinho BM, Chase K, Sapp E, Ly S, Alterman JF, Hassler MR, Echeverria D, Raj L, Morrissey DV, DiFiglia M, Aronin N, Khvorova A.

Mol Ther. 2016 Oct;24(10):1836-1847. doi: 10.1038/mt.2016.126. Epub 2016 Jun 27.

25.

Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain.

Nikan M, Osborn MF, Coles AH, Godinho BM, Hall LM, Haraszti RA, Hassler MR, Echeverria D, Aronin N, Khvorova A.

Mol Ther Nucleic Acids. 2016 Aug 9;5(8):e344. doi: 10.1038/mtna.2016.50.

26.

Voices of biotech.

Amit I, Baker D, Barker R, Berger B, Bertozzi C, Bhatia S, Biffi A, Demichelis F, Doudna J, Dowdy SF, Endy D, Helmstaedter M, Junca H, June C, Kamb S, Khvorova A, Kim DH, Kim JS, Krishnan Y, Lakadamyali M, Lappalainen T, Lewin S, Liao J, Loman N, Lundberg E, Lynd L, Martin C, Mellman I, Miyawaki A, Mummery C, Nelson K, Paz J, Peralta-Yahya P, Picotti P, Polyak K, Prather K, Qin J, Quake S, Regev A, Rogers JA, Shetty R, Sommer M, Stevens M, Stolovitzky G, Takahashi M, Tang F, Teichmann S, Torres-Padilla ME, Tripathi L, Vemula P, Verdine G, Vollmer F, Wang J, Ying JY, Zhang F, Zhang T.

Nat Biotechnol. 2016 Mar;34(3):270-5. doi: 10.1038/nbt.3502. No abstract available.

PMID:
26963549
27.

Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain.

Alterman JF, Hall LM, Coles AH, Hassler MR, Didiot MC, Chase K, Abraham J, Sottosanti E, Johnson E, Sapp E, Osborn MF, Difiglia M, Aronin N, Khvorova A.

Mol Ther Nucleic Acids. 2015 Dec 1;4:e266. doi: 10.1038/mtna.2015.38.

28.

A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo.

Coles AH, Osborn MF, Alterman JF, Turanov AA, Godinho BM, Kennington L, Chase K, Aronin N, Khvorova A.

Nucleic Acid Ther. 2016 Apr;26(2):86-92. doi: 10.1089/nat.2015.0578. Epub 2015 Nov 23.

29.

Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs.

Osborn MF, Alterman JF, Nikan M, Cao H, Didiot MC, Hassler MR, Coles AH, Khvorova A.

Nucleic Acids Res. 2015 Oct 15;43(18):8664-72. doi: 10.1093/nar/gkv942. Epub 2015 Sep 22.

30.

Corrigendum: Taking charge of siRNA delivery.

Khvorova A, Osborn MF, Hassler MR.

Nat Biotechnol. 2015 May;33(5):563. doi: 10.1038/nbt0515-563c. No abstract available.

PMID:
25965761
31.

Taking charge of siRNA delivery.

Khvorova A, Osborn MF, Hassler MR.

Nat Biotechnol. 2014 Dec;32(12):1197-8. doi: 10.1038/nbt.3091. Erratum in: Nat Biotechnol. 2015 May;33(5):563.

PMID:
25489835
32.

Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye.

Byrne M, Tzekov R, Wang Y, Rodgers A, Cardia J, Ford G, Holton K, Pandarinathan L, Lapierre J, Stanney W, Bulock K, Shaw S, Libertine L, Fettes K, Khvorova A, Kaushal S, Pavco P.

J Ocul Pharmacol Ther. 2013 Dec;29(10):855-64. doi: 10.1089/jop.2013.0148. Epub 2013 Nov 1.

PMID:
24180627
33.

New competition in RNA regulation.

Khvorova A, Wolfson A.

Nat Biotechnol. 2012 Jan 9;30(1):58-9. doi: 10.1038/nbt.2092. No abstract available.

PMID:
22231098
34.

Potent and systematic RNAi mediated silencing with single oligonucleotide compounds.

Lapierre J, Salomon W, Cardia J, Bulock K, Lam JT, Stanney WJ, Ford G, Smith-Anzures B, Woolf T, Kamens J, Khvorova A, Samarsky D.

RNA. 2011 Jun;17(6):1032-7. doi: 10.1261/rna.2399411. Epub 2011 Apr 14.

35.

Specificity and functionality of microRNA inhibitors.

Robertson B, Dalby AB, Karpilow J, Khvorova A, Leake D, Vermeulen A.

Silence. 2010 Apr 1;1(1):10. doi: 10.1186/1758-907X-1-10.

36.

Identification of genes that regulate epithelial cell migration using an siRNA screening approach.

Simpson KJ, Selfors LM, Bui J, Reynolds A, Leake D, Khvorova A, Brugge JS.

Nat Cell Biol. 2008 Sep;10(9):1027-38. doi: 10.1038/ncb1762.

PMID:
19160483
37.

Identifying siRNA-induced off-targets by microarray analysis.

Anderson E, Boese Q, Khvorova A, Karpilow J.

Methods Mol Biol. 2008;442:45-63. doi: 10.1007/978-1-59745-191-8_4.

PMID:
18369777
38.

Experimental validation of the importance of seed complement frequency to siRNA specificity.

Anderson EM, Birmingham A, Baskerville S, Reynolds A, Maksimova E, Leake D, Fedorov Y, Karpilow J, Khvorova A.

RNA. 2008 May;14(5):853-61. doi: 10.1261/rna.704708. Epub 2008 Mar 26.

39.

A protocol for designing siRNAs with high functionality and specificity.

Birmingham A, Anderson E, Sullivan K, Reynolds A, Boese Q, Leake D, Karpilow J, Khvorova A.

Nat Protoc. 2007;2(9):2068-78.

PMID:
17853862
40.

Defining the optimal parameters for hairpin-based knockdown constructs.

Li L, Lin X, Khvorova A, Fesik SW, Shen Y.

RNA. 2007 Oct;13(10):1765-74. Epub 2007 Aug 13.

41.

Double-stranded regions are essential design components of potent inhibitors of RISC function.

Vermeulen A, Robertson B, Dalby AB, Marshall WS, Karpilow J, Leake D, Khvorova A, Baskerville S.

RNA. 2007 May;13(5):723-30. Epub 2007 Mar 30.

42.

Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing.

Jackson AL, Burchard J, Leake D, Reynolds A, Schelter J, Guo J, Johnson JM, Lim L, Karpilow J, Nichols K, Marshall W, Khvorova A, Linsley PS.

RNA. 2006 Jul;12(7):1197-205. Epub 2006 May 8.

43.

Off-target effects by siRNA can induce toxic phenotype.

Fedorov Y, Anderson EM, Birmingham A, Reynolds A, Karpilow J, Robinson K, Leake D, Marshall WS, Khvorova A.

RNA. 2006 Jul;12(7):1188-96. Epub 2006 May 8.

44.

Induction of the interferon response by siRNA is cell type- and duplex length-dependent.

Reynolds A, Anderson EM, Vermeulen A, Fedorov Y, Robinson K, Leake D, Karpilow J, Marshall WS, Khvorova A.

RNA. 2006 Jun;12(6):988-93. Epub 2006 Apr 12.

45.

3' UTR seed matches, but not overall identity, are associated with RNAi off-targets.

Birmingham A, Anderson EM, Reynolds A, Ilsley-Tyree D, Leake D, Fedorov Y, Baskerville S, Maksimova E, Robinson K, Karpilow J, Marshall WS, Khvorova A.

Nat Methods. 2006 Mar;3(3):199-204. Erratum in: Nat Methods. 2007 Jun;4(6):533.

PMID:
16489337
46.

Induction of apoptosis by G3139 in melanoma cells.

Benimetskaya L, Lai JC, Khvorova A, Wu S, Miller P, Stein CA.

Ann N Y Acad Sci. 2005 Nov;1058:235-45. Review.

PMID:
16394140
47.

Gene profiling study of G3139- and Bcl-2-targeting siRNAs identifies a unique G3139 molecular signature.

Anderson EM, Miller P, Ilsley D, Marshall W, Khvorova A, Stein CA, Benimetskaya L.

Cancer Gene Ther. 2006 Apr;13(4):406-14.

PMID:
16195754
48.

The contributions of dsRNA structure to Dicer specificity and efficiency.

Vermeulen A, Behlen L, Reynolds A, Wolfson A, Marshall WS, Karpilow J, Khvorova A.

RNA. 2005 May;11(5):674-82. Epub 2005 Apr 5.

49.

Different delivery methods-different expression profiles.

Fedorov Y, King A, Anderson E, Karpilow J, Ilsley D, Marshall W, Khvorova A.

Nat Methods. 2005 Apr;2(4):241. No abstract available. Erratum in: Nat Methods. 2005 Jul;2(7):559.

PMID:
15782213
50.

Phosphorothioate oligodeoxynucleotides and G3139 induce apoptosis in 518A2 melanoma cells.

Lai JC, Benimetskaya L, Khvorova A, Wu S, Hua E, Miller P, Stein CA.

Mol Cancer Ther. 2005 Feb;4(2):305-15. Erratum in: Mol Cancer Ther. 2005 May;4(5):864.

Supplemental Content

Loading ...
Support Center