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Items: 1 to 50 of 88

1.

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.

Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A.

Nat Biotechnol. 2019 Aug;37(8):884-894. doi: 10.1038/s41587-019-0205-0. Epub 2019 Aug 2.

PMID:
31375812
2.

Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition.

Haraszti RA, Miller R, Dubuke ML, Rockwell HE, Coles AH, Sapp E, Didiot MC, Echeverria D, Stoppato M, Sere YY, Leszyk J, Alterman JF, Godinho BMDC, Hassler MR, McDaniel J, Narain NR, Wollacott R, Wang Y, Shaffer SA, Kiebish MA, DiFiglia M, Aronin N, Khvorova A.

iScience. 2019 Jun 28;16:230-241. doi: 10.1016/j.isci.2019.05.029. Epub 2019 May 27.

3.

Nucleic Acid Therapeutics for Neurological Diseases.

Watts JK, Brown RH, Khvorova A.

Neurotherapeutics. 2019 Apr;16(2):245-247. doi: 10.1007/s13311-019-00736-1. No abstract available.

PMID:
31037650
4.

The valency of fatty acid conjugates impacts siRNA pharmacokinetics, distribution, and efficacy in vivo.

Biscans A, Coles A, Echeverria D, Khvorova A.

J Control Release. 2019 May 28;302:116-125. doi: 10.1016/j.jconrel.2019.03.028. Epub 2019 Mar 30.

PMID:
30940496
5.

Minimal information for studies of extracellular vesicles 2018 (MISEV2018): a position statement of the International Society for Extracellular Vesicles and update of the MISEV2014 guidelines.

Théry C, Witwer KW, Aikawa E, Alcaraz MJ, Anderson JD, Andriantsitohaina R, Antoniou A, Arab T, Archer F, Atkin-Smith GK, Ayre DC, Bach JM, Bachurski D, Baharvand H, Balaj L, Baldacchino S, Bauer NN, Baxter AA, Bebawy M, Beckham C, Bedina Zavec A, Benmoussa A, Berardi AC, Bergese P, Bielska E, Blenkiron C, Bobis-Wozowicz S, Boilard E, Boireau W, Bongiovanni A, Borràs FE, Bosch S, Boulanger CM, Breakefield X, Breglio AM, Brennan MÁ, Brigstock DR, Brisson A, Broekman ML, Bromberg JF, Bryl-Górecka P, Buch S, Buck AH, Burger D, Busatto S, Buschmann D, Bussolati B, Buzás EI, Byrd JB, Camussi G, Carter DR, Caruso S, Chamley LW, Chang YT, Chen C, Chen S, Cheng L, Chin AR, Clayton A, Clerici SP, Cocks A, Cocucci E, Coffey RJ, Cordeiro-da-Silva A, Couch Y, Coumans FA, Coyle B, Crescitelli R, Criado MF, D'Souza-Schorey C, Das S, Datta Chaudhuri A, de Candia P, De Santana EF, De Wever O, Del Portillo HA, Demaret T, Deville S, Devitt A, Dhondt B, Di Vizio D, Dieterich LC, Dolo V, Dominguez Rubio AP, Dominici M, Dourado MR, Driedonks TA, Duarte FV, Duncan HM, Eichenberger RM, Ekström K, El Andaloussi S, Elie-Caille C, Erdbrügger U, Falcón-Pérez JM, Fatima F, Fish JE, Flores-Bellver M, Försönits A, Frelet-Barrand A, Fricke F, Fuhrmann G, Gabrielsson S, Gámez-Valero A, Gardiner C, Gärtner K, Gaudin R, Gho YS, Giebel B, Gilbert C, Gimona M, Giusti I, Goberdhan DC, Görgens A, Gorski SM, Greening DW, Gross JC, Gualerzi A, Gupta GN, Gustafson D, Handberg A, Haraszti RA, Harrison P, Hegyesi H, Hendrix A, Hill AF, Hochberg FH, Hoffmann KF, Holder B, Holthofer H, Hosseinkhani B, Hu G, Huang Y, Huber V, Hunt S, Ibrahim AG, Ikezu T, Inal JM, Isin M, Ivanova A, Jackson HK, Jacobsen S, Jay SM, Jayachandran M, Jenster G, Jiang L, Johnson SM, Jones JC, Jong A, Jovanovic-Talisman T, Jung S, Kalluri R, Kano SI, Kaur S, Kawamura Y, Keller ET, Khamari D, Khomyakova E, Khvorova A, Kierulf P, Kim KP, Kislinger T, Klingeborn M, Klinke DJ 2nd, Kornek M, Kosanović MM, Kovács ÁF, Krämer-Albers EM, Krasemann S, Krause M, Kurochkin IV, Kusuma GD, Kuypers S, Laitinen S, Langevin SM, Languino LR, Lannigan J, Lässer C, Laurent LC, Lavieu G, Lázaro-Ibáñez E, Le Lay S, Lee MS, Lee YXF, Lemos DS, Lenassi M, Leszczynska A, Li IT, Liao K, Libregts SF, Ligeti E, Lim R, Lim SK, Linē A, Linnemannstöns K, Llorente A, Lombard CA, Lorenowicz MJ, Lörincz ÁM, Lötvall J, Lovett J, Lowry MC, Loyer X, Lu Q, Lukomska B, Lunavat TR, Maas SL, Malhi H, Marcilla A, Mariani J, Mariscal J, Martens-Uzunova ES, Martin-Jaular L, Martinez MC, Martins VR, Mathieu M, Mathivanan S, Maugeri M, McGinnis LK, McVey MJ, Meckes DG Jr, Meehan KL, Mertens I, Minciacchi VR, Möller A, Møller Jørgensen M, Morales-Kastresana A, Morhayim J, Mullier F, Muraca M, Musante L, Mussack V, Muth DC, Myburgh KH, Najrana T, Nawaz M, Nazarenko I, Nejsum P, Neri C, Neri T, Nieuwland R, Nimrichter L, Nolan JP, Nolte-'t Hoen EN, Noren Hooten N, O'Driscoll L, O'Grady T, O'Loghlen A, Ochiya T, Olivier M, Ortiz A, Ortiz LA, Osteikoetxea X, Østergaard O, Ostrowski M, Park J, Pegtel DM, Peinado H, Perut F, Pfaffl MW, Phinney DG, Pieters BC, Pink RC, Pisetsky DS, Pogge von Strandmann E, Polakovicova I, Poon IK, Powell BH, Prada I, Pulliam L, Quesenberry P, Radeghieri A, Raffai RL, Raimondo S, Rak J, Ramirez MI, Raposo G, Rayyan MS, Regev-Rudzki N, Ricklefs FL, Robbins PD, Roberts DD, Rodrigues SC, Rohde E, Rome S, Rouschop KM, Rughetti A, Russell AE, Saá P, Sahoo S, Salas-Huenuleo E, Sánchez C, Saugstad JA, Saul MJ, Schiffelers RM, Schneider R, Schøyen TH, Scott A, Shahaj E, Sharma S, Shatnyeva O, Shekari F, Shelke GV, Shetty AK, Shiba K, Siljander PR, Silva AM, Skowronek A, Snyder OL 2nd, Soares RP, Sódar BW, Soekmadji C, Sotillo J, Stahl PD, Stoorvogel W, Stott SL, Strasser EF, Swift S, Tahara H, Tewari M, Timms K, Tiwari S, Tixeira R, Tkach M, Toh WS, Tomasini R, Torrecilhas AC, Tosar JP, Toxavidis V, Urbanelli L, Vader P, van Balkom BW, van der Grein SG, Van Deun J, van Herwijnen MJ, Van Keuren-Jensen K, van Niel G, van Royen ME, van Wijnen AJ, Vasconcelos MH, Vechetti IJ Jr, Veit TD, Vella LJ, Velot É, Verweij FJ, Vestad B, Viñas JL, Visnovitz T, Vukman KV, Wahlgren J, Watson DC, Wauben MH, Weaver A, Webber JP, Weber V, Wehman AM, Weiss DJ, Welsh JA, Wendt S, Wheelock AM, Wiener Z, Witte L, Wolfram J, Xagorari A, Xander P, Xu J, Yan X, Yáñez-Mó M, Yin H, Yuana Y, Zappulli V, Zarubova J, Žėkas V, Zhang JY, Zhao Z, Zheng L, Zheutlin AR, Zickler AM, Zimmermann P, Zivkovic AM, Zocco D, Zuba-Surma EK.

J Extracell Vesicles. 2018 Nov 23;7(1):1535750. doi: 10.1080/20013078.2018.1535750. eCollection 2018.

6.

Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington's Disease.

Tousley A, Iuliano M, Weisman E, Sapp E, Zhang N, Vodicka P, Alexander J, Aviolat H, Gatune L, Reeves P, Li X, Khvorova A, Ellerby LM, Aronin N, DiFiglia M, Kegel-Gleason KB.

J Huntingtons Dis. 2019;8(1):53-69. doi: 10.3233/JHD-180311.

7.

Gene Silencing With siRNA (RNA Interference): A New Therapeutic Option During Ex Vivo Machine Liver Perfusion Preservation

Thijssen MF, Brüggenwirth IMA, Gillooly A, Khvorova A, Kowalik TF, Martins PN.

Liver Transpl. 2019 Jan;25(1):140-151. doi: 10.1002/lt.25383. Review.

PMID:
30561891
8.

Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo.

Biscans A, Coles A, Haraszti R, Echeverria D, Hassler M, Osborn M, Khvorova A.

Nucleic Acids Res. 2019 Feb 20;47(3):1082-1096. doi: 10.1093/nar/gky1239.

9.

Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways.

Osborn MF, Coles AH, Biscans A, Haraszti RA, Roux L, Davis S, Ly S, Echeverria D, Hassler MR, Godinho BMDC, Nikan M, Khvorova A.

Nucleic Acids Res. 2019 Feb 20;47(3):1070-1081. doi: 10.1093/nar/gky1232.

10.

RNAi modulation of placental sFLT1 for the treatment of preeclampsia.

Turanov AA, Lo A, Hassler MR, Makris A, Ashar-Patel A, Alterman JF, Coles AH, Haraszti RA, Roux L, Godinho BMDC, Echeverria D, Pears S, Iliopoulos J, Shanmugalingam R, Ogle R, Zsengeller ZK, Hennessy A, Karumanchi SA, Moore MJ, Khvorova A.

Nat Biotechnol. 2018 Nov 19. doi: 10.1038/nbt.4297. [Epub ahead of print]

11.

Exosomes Produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved Activity.

Haraszti RA, Miller R, Stoppato M, Sere YY, Coles A, Didiot MC, Wollacott R, Sapp E, Dubuke ML, Li X, Shaffer SA, DiFiglia M, Wang Y, Aronin N, Khvorova A.

Mol Ther. 2018 Dec 5;26(12):2838-2847. doi: 10.1016/j.ymthe.2018.09.015. Epub 2018 Sep 22.

12.

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.

13.

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 Nov 16;46(20):10905-10916. doi: 10.1093/nar/gky745.

14.

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 Nov 7;26(11):2580-2591. doi: 10.1016/j.ymthe.2018.08.005. Epub 2018 Aug 8.

PMID:
30143435
15.

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.

16.

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.

17.

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
18.

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.

19.

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. Review.

20.

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.

21.

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.

22.

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.

23.

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.

24.

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
25.

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.

26.

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.

27.

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.

28.

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
29.

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.

30.

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.

31.

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.

32.

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
33.

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.

34.

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.

35.

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.

36.

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.

37.

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
38.

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.

39.

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.

40.

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.

41.

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
42.

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
43.

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
44.

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
45.

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.

46.

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.

47.

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
48.

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
49.

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.

50.

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

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