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Items: 1 to 50 of 161

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.

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.

6.

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
7.

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.

8.

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
9.

Hippo Signaling Pathway Dysregulation in Human Huntington's Disease Brain and Neuronal Stem Cells.

Mueller KA, Glajch KE, Huizenga MN, Wilson RA, Granucci EJ, Dios AM, Tousley AR, Iuliano M, Weisman E, LaQuaglia MJ, DiFiglia M, Kegel-Gleason K, Vakili K, Sadri-Vakili G.

Sci Rep. 2018 Jul 27;8(1):11355. doi: 10.1038/s41598-018-29319-4.

10.

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.

11.

The COOH-terminal domain of huntingtin interacts with RhoGEF kalirin and modulates cell survival.

McClory H, Wang X, Sapp E, Gatune LW, Iuliano M, Wu CY, Nathwani G, Kegel-Gleason KB, DiFiglia M, Li X.

Sci Rep. 2018 May 22;8(1):8000. doi: 10.1038/s41598-018-26255-1.

12.

HttQ111/+ Huntington's Disease Knock-in Mice Exhibit Brain Region-Specific Morphological Changes and Synaptic Dysfunction.

Kovalenko M, Milnerwood A, Giordano J, St Claire J, Guide JR, Stromberg M, Gillis T, Sapp E, DiFiglia M, MacDonald ME, Carroll JB, Lee JM, Tappan S, Raymond L, Wheeler VC.

J Huntingtons Dis. 2018;7(1):17-33. doi: 10.3233/JHD-170282.

13.

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
14.

Retracted: Elevated NADPH oxidase activity contributes to oxidative stress and cell death in Huntington's disease.

Valencia A, Sapp E, Kimm JS, McClory H, Reeves PB, Alexander J, Ansong KA, Masso N, Frosch MP, Kegel KB, Li X, DiFiglia M.

Hum Mol Genet. 2017 Nov 1;26(21):4314. doi: 10.1093/hmg/ddx303. No abstract available.

PMID:
28973680
15.

A toxic mutant huntingtin species is resistant to selective autophagy.

Fu Y, Wu P, Pan Y, Sun X, Yang H, Difiglia M, Lu B.

Nat Chem Biol. 2017 Nov;13(11):1152-1154. doi: 10.1038/nchembio.2461. Epub 2017 Sep 4.

PMID:
28869595
16.

KEAP1-modifying small molecule reveals muted NRF2 signaling responses in neural stem cells from Huntington's disease patients.

Quinti L, Dayalan Naidu S, Träger U, Chen X, Kegel-Gleason K, Llères D, Connolly C, Chopra V, Low C, Moniot S, Sapp E, Tousley AR, Vodicka P, Van Kanegan MJ, Kaltenbach LS, Crawford LA, Fuszard M, Higgins M, Miller JRC, Farmer RE, Potluri V, Samajdar S, Meisel L, Zhang N, Snyder A, Stein R, Hersch SM, Ellerby LM, Weerapana E, Schwarzschild MA, Steegborn C, Leavitt BR, Degterev A, Tabrizi SJ, Lo DC, DiFiglia M, Thompson LM, Dinkova-Kostova AT, Kazantsev AG.

Proc Natl Acad Sci U S A. 2017 Jun 6;114(23):E4676-E4685. doi: 10.1073/pnas.1614943114. Epub 2017 May 22.

17.

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.

18.

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.

19.

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
20.

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.

21.

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.

22.

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
23.

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.

24.

Mutated Huntingtin Causes Testicular Pathology in Transgenic Minipig Boars.

Macakova M, Bohuslavova B, Vochozkova P, Pavlok A, Sedlackova M, Vidinska D, Vochyanova K, Liskova I, Valekova I, Baxa M, Ellederova Z, Klima J, Juhas S, Juhasova J, Klouckova J, Haluzik M, Klempir J, Hansikova H, Spacilova J, Collins R, Blumenthal I, Talkowski M, Gusella JF, Howland DS, DiFiglia M, Motlik J.

Neurodegener Dis. 2016;16(3-4):245-59. doi: 10.1159/000443665. Epub 2016 Mar 10.

PMID:
26959244
25.

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.

26.

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.

27.

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.

28.

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
29.

A striatal-enriched intronic GPCR modulates huntingtin levels and toxicity.

Yao Y, Cui X, Al-Ramahi I, Sun X, Li B, Hou J, Difiglia M, Palacino J, Wu ZY, Ma L, Botas J, Lu B.

Elife. 2015 Mar 4;4. doi: 10.7554/eLife.05449.

30.

Glucose transporter 3 is a rab11-dependent trafficking cargo and its transport to the cell surface is reduced in neurons of CAG140 Huntington's disease mice.

McClory H, Williams D, Sapp E, Gatune LW, Wang P, DiFiglia M, Li X.

Acta Neuropathol Commun. 2014 Dec 20;2:179. doi: 10.1186/s40478-014-0178-7.

31.

Behavioral deficits, early gliosis, dysmyelination and synaptic dysfunction in a mouse model of mucolipidosis IV.

Grishchuk Y, Sri S, Rudinskiy N, Ma W, Stember KG, Cottle MW, Sapp E, Difiglia M, Muzikansky A, Betensky RA, Wong AM, Bacskai BJ, Hyman BT, Kelleher RJ 3rd, Cooper JD, Slaugenhaupt SA.

Acta Neuropathol Commun. 2014 Sep 9;2:133. doi: 10.1186/s40478-014-0133-7.

32.

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
33.

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
34.

Preconditioning stimuli induce autophagy via sphingosine kinase 2 in mouse cortical neurons.

Sheng R, Zhang TT, Felice VD, Qin T, Qin ZH, Smith CD, Sapp E, Difiglia M, Waeber C.

J Biol Chem. 2014 Jul 25;289(30):20845-57.

35.

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.

36.

Drugging unconventional targets: insights from Huntington's disease.

Yu S, Liang Y, Palacino J, Difiglia M, Lu B.

Trends Pharmacol Sci. 2014 Feb;35(2):53-62. doi: 10.1016/j.tips.2013.12.001. Epub 2014 Jan 2. Review.

PMID:
24388390
37.

SUMO-2 and PIAS1 modulate insoluble mutant huntingtin protein accumulation.

O'Rourke JG, Gareau JR, Ochaba J, Song W, Raskó T, Reverter D, Lee J, Monteys AM, Pallos J, Mee L, Vashishtha M, Apostol BL, Nicholson TP, Illes K, Zhu YZ, Dasso M, Bates GP, Difiglia M, Davidson B, Wanker EE, Marsh JL, Lima CD, Steffan JS, Thompson LM.

Cell Rep. 2013 Jul 25;4(2):362-75. doi: 10.1016/j.celrep.2013.06.034. Epub 2013 Jul 18.

38.

Identification of NUB1 as a suppressor of mutant Huntington toxicity via enhanced protein clearance.

Lu B, Al-Ramahi I, Valencia A, Wang Q, Berenshteyn F, Yang H, Gallego-Flores T, Ichcho S, Lacoste A, Hild M, Difiglia M, Botas J, Palacino J.

Nat Neurosci. 2013 May;16(5):562-70. doi: 10.1038/nn.3367. Epub 2013 Mar 24.

PMID:
23525043
39.

A transgenic minipig model of Huntington's Disease.

Baxa M, Hruska-Plochan M, Juhas S, Vodicka P, Pavlok A, Juhasova J, Miyanohara A, Nejime T, Klima J, Macakova M, Marsala S, Weiss A, Kubickova S, Musilova P, Vrtel R, Sontag EM, Thompson LM, Schier J, Hansikova H, Howland DS, Cattaneo E, DiFiglia M, Marsala M, Motlik J.

J Huntingtons Dis. 2013;2(1):47-68. doi: 10.3233/JHD-130001.

PMID:
25063429
40.

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
41.

Elevated NADPH oxidase activity contributes to oxidative stress and cell death in Huntington's disease.

Valencia A, Sapp E, Kimm JS, McClory H, Reeves PB, Alexander J, Ansong KA, Masso N, Frosch MP, Kegel KB, Li X, DiFiglia M.

Hum Mol Genet. 2013 Mar 15;22(6):1112-31. doi: 10.1093/hmg/dds516. Epub 2012 Dec 7. Retraction in: Hum Mol Genet. 2017 Nov 1;26(21):4314.

42.

The role of chaperone-mediated autophagy in huntingtin degradation.

Qi L, Zhang XD, Wu JC, Lin F, Wang J, DiFiglia M, Qin ZH.

PLoS One. 2012;7(10):e46834. doi: 10.1371/journal.pone.0046834. Epub 2012 Oct 11.

43.

The regulation of N-terminal Huntingtin (Htt552) accumulation by Beclin1.

Wu JC, Qi L, Wang Y, Kegel KB, Yoder J, Difiglia M, Qin ZH, Lin F.

Acta Pharmacol Sin. 2012 Jun;33(6):743-51. doi: 10.1038/aps.2012.14. Epub 2012 Apr 30.

44.

Deficient Rab11 activity underlies glucose hypometabolism in primary neurons of Huntington's disease mice.

Li X, Valencia A, McClory H, Sapp E, Kegel KB, Difiglia M.

Biochem Biophys Res Commun. 2012 May 18;421(4):727-30. doi: 10.1016/j.bbrc.2012.04.070. Epub 2012 Apr 20.

PMID:
22542623
45.

Multiple phenotypes in Huntington disease mouse neural stem cells.

Ritch JJ, Valencia A, Alexander J, Sapp E, Gatune L, Sangrey GR, Sinha S, Scherber CM, Zeitlin S, Sadri-Vakili G, Irimia D, Difiglia M, Kegel KB.

Mol Cell Neurosci. 2012 May;50(1):70-81. doi: 10.1016/j.mcn.2012.03.011. Epub 2012 Apr 6.

46.

Expression of mutant N-terminal huntingtin fragment (htt552-100Q) in astrocytes suppresses the secretion of BDNF.

Wang L, Lin F, Wang J, Wu J, Han R, Zhu L, Difiglia M, Qin Z.

Brain Res. 2012 Apr 17;1449:69-82. doi: 10.1016/j.brainres.2012.01.077. Epub 2012 Feb 23.

PMID:
22410294
47.

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.

48.

Rapamycin prevents the mutant huntingtin-suppressed GLT-1 expression in cultured astrocytes.

Chen LL, Wu JC, Wang LH, Wang J, Qin ZH, Difiglia M, Lin F.

Acta Pharmacol Sin. 2012 Mar;33(3):385-92. doi: 10.1038/aps.2011.162. Epub 2012 Jan 23.

49.

Truncated N-terminal huntingtin fragment with expanded-polyglutamine (htt552-100Q) suppresses brain-derived neurotrophic factor transcription in astrocytes.

Wang L, Lin F, Wang J, Wu J, Han R, Zhu L, Zhang G, DiFiglia M, Qin Z.

Acta Biochim Biophys Sin (Shanghai). 2012 Mar;44(3):249-58. doi: 10.1093/abbs/gmr125. Epub 2012 Jan 9.

PMID:
22234237
50.

Reagents that block neuronal death from Huntington's disease also curb oxidative stress.

Valencia A, Sapp E, Reeves PB, Alexander J, Masso N, Li X, Kegel KB, DiFiglia M.

Neuroreport. 2012 Jan 4;23(1):10-5. doi: 10.1097/WNR.0b013e32834d92e6.

PMID:
22045254

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