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Items: 1 to 50 of 71

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.

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.

10.

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.

11.

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.

12.

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
13.

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
14.

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.

15.

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.

16.

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.

17.

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
18.

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.

19.

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.

20.

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
21.

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.

22.

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.

23.

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.

24.

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
25.

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.

26.

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.

27.

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.

28.

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.

29.

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.

30.

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
31.

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.

32.

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.

33.

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
34.

Cysteine oxidation within N-terminal mutant huntingtin promotes oligomerization and delays clearance of soluble protein.

Fox JH, Connor T, Stiles M, Kama J, Lu Z, Dorsey K, Lieberman G, Sapp E, Cherny RA, Banks M, Volitakis I, DiFiglia M, Berezovska O, Bush AI, Hersch SM.

J Biol Chem. 2011 May 20;286(20):18320-30. doi: 10.1074/jbc.M110.199448. Epub 2011 Mar 30. Erratum in: J Biol Chem. 2011 Jul 29;286(30):27068. Liebermann, Gregory [corrected to Lieberman, Gregory].

35.

Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitors.

Kegel KB, Sapp E, Alexander J, Reeves P, Bleckmann D, Sobin L, Masso N, Valencia A, Jeong H, Krainc D, Palacino J, Curtis D, Kuhn R, Betschart C, Sena-Esteves M, Aronin N, Paganetti P, Difiglia M.

Mol Neurodegener. 2010 Dec 14;5:58. doi: 10.1186/1750-1326-5-58.

36.

Aberrant Rab11-dependent trafficking of the neuronal glutamate transporter EAAC1 causes oxidative stress and cell death in Huntington's disease.

Li X, Valencia A, Sapp E, Masso N, Alexander J, Reeves P, Kegel KB, Aronin N, Difiglia M.

J Neurosci. 2010 Mar 31;30(13):4552-61. doi: 10.1523/JNEUROSCI.5865-09.2010.

37.

Mutant huntingtin fragments form oligomers in a polyglutamine length-dependent manner in vitro and in vivo.

Legleiter J, Mitchell E, Lotz GP, Sapp E, Ng C, DiFiglia M, Thompson LM, Muchowski PJ.

J Biol Chem. 2010 May 7;285(19):14777-90. doi: 10.1074/jbc.M109.093708. Epub 2010 Mar 10.

38.

Mutant huntingtin impairs vesicle formation from recycling endosomes by interfering with Rab11 activity.

Li X, Standley C, Sapp E, Valencia A, Qin ZH, Kegel KB, Yoder J, Comer-Tierney LA, Esteves M, Chase K, Alexander J, Masso N, Sobin L, Bellve K, Tuft R, Lifshitz L, Fogarty K, Aronin N, DiFiglia M.

Mol Cell Biol. 2009 Nov;29(22):6106-16. doi: 10.1128/MCB.00420-09. Epub 2009 Sep 14.

39.

Disruption of Rab11 activity in a knock-in mouse model of Huntington's disease.

Li X, Sapp E, Chase K, Comer-Tierney LA, Masso N, Alexander J, Reeves P, Kegel KB, Valencia A, Esteves M, Aronin N, Difiglia M.

Neurobiol Dis. 2009 Nov;36(2):374-83. doi: 10.1016/j.nbd.2009.08.003. Epub 2009 Aug 20.

40.

Mutant huntingtin and glycogen synthase kinase 3-beta accumulate in neuronal lipid rafts of a presymptomatic knock-in mouse model of Huntington's disease.

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

J Neurosci Res. 2010 Jan;88(1):179-90. doi: 10.1002/jnr.22184.

PMID:
19642201
41.

Polyglutamine expansion in huntingtin increases its insertion into lipid bilayers.

Kegel KB, Schewkunow V, Sapp E, Masso N, Wanker EE, DiFiglia M, Goldmann WH.

Biochem Biophys Res Commun. 2009 Sep 25;387(3):472-5. doi: 10.1016/j.bbrc.2009.07.039. Epub 2009 Jul 14.

PMID:
19607813
42.

Polyglutamine expansion in huntingtin alters its interaction with phospholipids.

Kegel KB, Sapp E, Alexander J, Valencia A, Reeves P, Li X, Masso N, Sobin L, Aronin N, DiFiglia M.

J Neurochem. 2009 Sep;110(5):1585-97. doi: 10.1111/j.1471-4159.2009.06255.x. Epub 2009 Jun 29.

43.

A function of huntingtin in guanine nucleotide exchange on Rab11.

Li X, Sapp E, Valencia A, Kegel KB, Qin ZH, Alexander J, Masso N, Reeves P, Ritch JJ, Zeitlin S, Aronin N, Difiglia M.

Neuroreport. 2008 Oct 29;19(16):1643-7. doi: 10.1097/WNR.0b013e328315cd4c.

PMID:
18845944
44.

Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits.

DiFiglia M, Sena-Esteves M, Chase K, Sapp E, Pfister E, Sass M, Yoder J, Reeves P, Pandey RK, Rajeev KG, Manoharan M, Sah DW, Zamore PD, Aronin N.

Proc Natl Acad Sci U S A. 2007 Oct 23;104(43):17204-9. Epub 2007 Oct 16.

45.

Lysosomal proteases are involved in generation of N-terminal huntingtin fragments.

Kim YJ, Sapp E, Cuiffo BG, Sobin L, Yoder J, Kegel KB, Qin ZH, Detloff P, Aronin N, DiFiglia M.

Neurobiol Dis. 2006 May;22(2):346-56. Epub 2006 Jan 19.

PMID:
16423528
46.

Huntingtin associates with acidic phospholipids at the plasma membrane.

Kegel KB, Sapp E, Yoder J, Cuiffo B, Sobin L, Kim YJ, Qin ZH, Hayden MR, Aronin N, Scott DL, Isenberg G, Goldmann WH, DiFiglia M.

J Biol Chem. 2005 Oct 28;280(43):36464-73. Epub 2005 Aug 5.

47.

Huntingtin bodies sequester vesicle-associated proteins by a polyproline-dependent interaction.

Qin ZH, Wang Y, Sapp E, Cuiffo B, Wanker E, Hayden MR, Kegel KB, Aronin N, DiFiglia M.

J Neurosci. 2004 Jan 7;24(1):269-81.

48.

Distribution and ultrastructural localization of torsinA immunoreactivity in the human brain.

Augood SJ, Keller-McGandy CE, Siriani A, Hewett J, Ramesh V, Sapp E, DiFiglia M, Breakefield XO, Standaert DG.

Brain Res. 2003 Oct 3;986(1-2):12-21.

PMID:
12965225
49.

Huntingtin is present in the nucleus, interacts with the transcriptional corepressor C-terminal binding protein, and represses transcription.

Kegel KB, Meloni AR, Yi Y, Kim YJ, Doyle E, Cuiffo BG, Sapp E, Wang Y, Qin ZH, Chen JD, Nevins JR, Aronin N, DiFiglia M.

J Biol Chem. 2002 Mar 1;277(9):7466-76. Epub 2001 Dec 5.

50.

Mice transgenic for exon 1 of the Huntington's disease gene display reduced striatal sensitivity to neurotoxicity induced by dopamine and 6-hydroxydopamine.

Petersén A, Hansson O, Puschban Z, Sapp E, Romero N, Castilho RF, Sulzer D, Rice M, DiFiglia M, Przedborski S, Brundin P.

Eur J Neurosci. 2001 Nov;14(9):1425-35.

PMID:
11722604

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