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Items: 1 to 50 of 153

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.

Huntingtin associates with the actin cytoskeleton and α-actinin isoforms to influence stimulus dependent morphology changes.

Tousley A, Iuliano M, Weisman E, Sapp E, Richardson H, Vodicka P, Alexander J, Aronin N, DiFiglia M, Kegel-Gleason KB.

PLoS One. 2019 Feb 15;14(2):e0212337. doi: 10.1371/journal.pone.0212337. eCollection 2019.

4.

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.

5.

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer.

Wang D, Zhong L, Li M, Li J, Tran K, Ren L, He R, Xie J, Moser RP, Fraser C, Kuchel T, Sena-Esteves M, Flotte TR, Aronin N, Gao G.

Mol Ther Methods Clin Dev. 2018 Oct 4;11:65-72. doi: 10.1016/j.omtm.2018.09.003. eCollection 2018 Dec 14.

6.

IMPROVING AFTER-HOURS CALLS TO ENDOCRINOLOGY FELLOWS.

Siddiqui UM, Ayyoub S, Raja S, Yeasmin S, Harlan DM, Aronin N, Torres MS.

Endocr Pract. 2019 Jan;25(1):51-54. doi: 10.4158/EP-2018-0339. Epub 2018 Nov 1.

PMID:
30383491
7.

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.

8.

Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration.

Mondo E, Moser R, Gao G, Mueller C, Sena-Esteves M, Sapp E, Pfister E, O'Connell D, Takle K, Erger KE, Liu W, Conlon TJ, DiFiglia M, Gounis MJ, Aronin N.

J Huntingtons Dis. 2018;7(4):309-319. doi: 10.3233/JHD-180302.

PMID:
30320596
9.

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.

10.

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
11.

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.

12.

A Fresh Look at Huntingtin mRNA Processing in Huntington's Disease.

Romo L, Mohn ES, Aronin N.

J Huntingtons Dis. 2018;7(2):101-108. doi: 10.3233/JHD-180292.

13.

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.

14.

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.

15.

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.

16.

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
17.

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.

Pfister EL, DiNardo N, Mondo E, Borel F, Conroy F, Fraser C, Gernoux G, Han X, Hu D, Johnson E, Kennington L, Liu P, Reid SJ, Sapp E, Vodicka P, Kuchel T, Morton AJ, Howland D, Moser R, Sena-Esteves M, Gao G, Mueller C, DiFiglia M, Aronin N.

Hum Gene Ther. 2018 Jun;29(6):663-673. doi: 10.1089/hum.2017.199. Epub 2018 Feb 23.

PMID:
29207890
18.

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.

19.

Alterations in mRNA 3' UTR Isoform Abundance Accompany Gene Expression Changes in Human Huntington's Disease Brains.

Romo L, Ashar-Patel A, Pfister E, Aronin N.

Cell Rep. 2017 Sep 26;20(13):3057-3070. doi: 10.1016/j.celrep.2017.09.009.

20.

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.

21.

Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin.

Pfister EL, Chase KO, Sun H, Kennington LA, Conroy F, Johnson E, Miller R, Borel F, Aronin N, Mueller C.

Mol Ther Nucleic Acids. 2017 Jun 16;7:324-334. doi: 10.1016/j.omtn.2017.04.011. Epub 2017 Apr 14.

22.

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.

23.

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.

24.

Allele-Selective Suppression of Mutant Huntingtin in Primary Human Blood Cells.

Miller JRC, Pfister EL, Liu W, Andre R, Träger U, Kennington LA, Lo K, Dijkstra S, Macdonald D, Ostroff G, Aronin N, Tabrizi SJ.

Sci Rep. 2017 Apr 24;7:46740. doi: 10.1038/srep46740.

25.

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.

26.

Cellular Analysis of Silencing the Huntington's Disease Gene Using AAV9 Mediated Delivery of Artificial Micro RNA into the Striatum of Q140/Q140 Mice.

Keeler AM, Sapp E, Chase K, Sottosanti E, Danielson E, Pfister E, Stoica L, DiFiglia M, Aronin N, Sena-Esteves M.

J Huntingtons Dis. 2016 Oct 1;5(3):239-248.

PMID:
27689620
27.

Autophagy Activation by Transcription Factor EB (TFEB) in Striatum of HDQ175/Q7 Mice.

Vodicka P, Chase K, Iuliano M, Tousley A, Valentine DT, Sapp E, Kegel-Gleason KB, Sena-Esteves M, Aronin N, DiFiglia M.

J Huntingtons Dis. 2016 Oct 1;5(3):249-260.

28.

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.

29.

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.

30.

Effects of Exogenous NUB1 Expression in the Striatum of HDQ175/Q7 Mice.

Vodicka P, Chase K, Iuliano M, Valentine DT, Sapp E, Lu B, Kegel-Gleason KB, Sena-Esteves M, Aronin N, DiFiglia M.

J Huntingtons Dis. 2016 Jun 13;5(2):163-74. doi: 10.3233/JHD-160195.

PMID:
27314618
31.

Does the Mutant CAG Expansion in Huntingtin mRNA Interfere with Exonucleolytic Cleavage of its First Exon?

Liu W, Pfister EL, Kennington LA, Chase KO, Mueller C, DiFiglia M, Aronin N.

J Huntingtons Dis. 2016;5(1):33-8. doi: 10.3233/JHD-150183.

32.

Excitatory transmission onto AgRP neurons is regulated by cJun NH2-terminal kinase 3 in response to metabolic stress.

Vernia S, Morel C, Madara JC, Cavanagh-Kyros J, Barrett T, Chase K, Kennedy NJ, Jung DY, Kim JK, Aronin N, Flavell RA, Lowell BB, Davis RJ.

Elife. 2016 Feb 24;5:e10031. doi: 10.7554/eLife.10031.

33.

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.

Choudhury SR, Harris AF, Cabral DJ, Keeler AM, Sapp E, Ferreira JS, Gray-Edwards HL, Johnson JA, Johnson AK, Su Q, Stoica L, DiFiglia M, Aronin N, Martin DR, Gao G, Sena-Esteves M.

Mol Ther. 2016 Apr;24(4):726-35. doi: 10.1038/mt.2015.231. Epub 2015 Dec 28.

34.

Recurrent Mutations of Chromatin-Remodeling Genes and Kinase Receptors in Pheochromocytomas and Paragangliomas.

Toledo RA, Qin Y, Cheng ZM, Gao Q, Iwata S, Silva GM, Prasad ML, Ocal IT, Rao S, Aronin N, Barontini M, Bruder J, Reddick RL, Chen Y, Aguiar RC, Dahia PL.

Clin Cancer Res. 2016 May 1;22(9):2301-10. doi: 10.1158/1078-0432.CCR-15-1841. Epub 2015 Dec 23.

35.

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.

36.

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.

37.

Safety of Striatal Infusion of siRNA in a Transgenic Huntington's Disease Mouse Model.

Johnson E, Chase K, McGowan S, Mondo E, Pfister E, Mick E, Friedline RH, Kim JK, Sapp E, DiFiglia M, Aronin N.

J Huntingtons Dis. 2015;4(3):219-229. doi: 10.3233/JHD-150163.

38.

Mass Spectrometry Analysis of Wild-Type and Knock-in Q140/Q140 Huntington's Disease Mouse Brains Reveals Changes in Glycerophospholipids Including Alterations in Phosphatidic Acid and Lyso-Phosphatidic Acid.

Vodicka P, Mo S, Tousley A, Green KM, Sapp E, Iuliano M, Sadri-Vakili G, Shaffer SA, Aronin N, DiFiglia M, Kegel-Gleason KB.

J Huntingtons Dis. 2015;4(2):187-201. doi: 10.3233/JHD-150149.

PMID:
26397899
39.

CRISPRseek: a bioconductor package to identify target-specific guide RNAs for CRISPR-Cas9 genome-editing systems.

Zhu LJ, Holmes BR, Aronin N, Brodsky MH.

PLoS One. 2014 Sep 23;9(9):e108424. doi: 10.1371/journal.pone.0108424. eCollection 2014.

40.

Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing.

Aronin N, DiFiglia M.

Mov Disord. 2014 Sep 15;29(11):1455-61. doi: 10.1002/mds.26020. Epub 2014 Aug 27. Review.

PMID:
25164989
41.

Assessment of chloroquine treatment for modulating autophagy flux in brain of WT and HD mice.

Vodicka P, Lim J, Williams DT, Kegel KB, Chase K, Park H, Marchionini D, Wilkinson S, Mead T, Birch H, Yates D, Lyons K, Dominguez C, Beconi M, Yue Z, Aronin N, DiFiglia M.

J Huntingtons Dis. 2014;3(2):159-74. doi: 10.3233/JHD-130081.

PMID:
25062859
42.

Striatal synaptosomes from Hdh140Q/140Q knock-in mice have altered protein levels, novel sites of methionine oxidation, and excess glutamate release after stimulation.

Valencia A, Sapp E, Kimm JS, McClory H, Ansong KA, Yohrling G, Kwak S, Kegel KB, Green KM, Shaffer SA, Aronin N, DiFiglia M.

J Huntingtons Dis. 2013;2(4):459-75.

43.

A synthetic luciferin improves bioluminescence imaging in live mice.

Evans MS, Chaurette JP, Adams ST Jr, Reddy GR, Paley MA, Aronin N, Prescher JA, Miller SC.

Nat Methods. 2014 Apr;11(4):393-5. doi: 10.1038/nmeth.2839. Epub 2014 Feb 9. Erratum in: Nat Methods. 2014 Apr;11(4):395.

44.

HTT-lowering reverses Huntington's disease immune dysfunction caused by NFκB pathway dysregulation.

Träger U, Andre R, Lahiri N, Magnusson-Lind A, Weiss A, Grueninger S, McKinnon C, Sirinathsinghji E, Kahlon S, Pfister EL, Moser R, Hummerich H, Antoniou M, Bates GP, Luthi-Carter R, Lowdell MW, Björkqvist M, Ostroff GR, Aronin N, Tabrizi SJ.

Brain. 2014 Mar;137(Pt 3):819-33. doi: 10.1093/brain/awt355. Epub 2014 Jan 22.

45.

Finding the striatum in sheep: use of a multi-modal guided approach for convection enhanced delivery.

van der Bom IM, Moser RP, Gao G, Mondo E, O'Connell D, Gounis MJ, McGowan S, Chaurette J, Bishop N, Sena-Esteves MS, Mueller C, Aronin N.

J Huntingtons Dis. 2013;2(1):41-5. doi: 10.3233/JHD-130053.

PMID:
25063428
46.

Increased Steady-State Mutant Huntingtin mRNA in Huntington's Disease Brain.

Liu W, Chaurette J, Pfister EL, Kennington LA, Chase KO, Bullock J, Vonsattel JP, Faull RL, Macdonald D, DiFiglia M, Zamore PD, Aronin N.

J Huntingtons Dis. 2013;2(4):491-500. doi: 10.3233/JHD-130079.

PMID:
25062733
47.

Hunting down huntingtin.

Aronin N, Moore M.

N Engl J Med. 2012 Nov 1;367(18):1753-4. doi: 10.1056/NEJMcibr1209595. No abstract available.

PMID:
23113488
48.

Native mutant huntingtin in human brain: evidence for prevalence of full-length monomer.

Sapp E, Valencia A, Li X, Aronin N, Kegel KB, Vonsattel JP, Young AB, Wexler N, DiFiglia M.

J Biol Chem. 2012 Apr 13;287(16):13487-99. doi: 10.1074/jbc.M111.286609. Epub 2012 Feb 27.

49.

Frameless multimodal image guidance of localized convection-enhanced delivery of therapeutics in the brain.

van der Bom IM, Moser RP, Gao G, Sena-Esteves M, Aronin N, Gounis MJ.

J Neurointerv Surg. 2013 Jan 1;5(1):69-72. doi: 10.1136/neurintsurg-2011-010170. Epub 2011 Dec 22.

50.

Argonaute protein identity and pairing geometry determine cooperativity in mammalian RNA silencing.

Broderick JA, Salomon WE, Ryder SP, Aronin N, Zamore PD.

RNA. 2011 Oct;17(10):1858-69. doi: 10.1261/rna.2778911. Epub 2011 Aug 30.

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