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Items: 1 to 20 of 40

1.

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.

2.

Generation of a new transgenic mouse model for assessment of tau gene silencing therapies.

Fromholt S, Reitano C, Brown H, Lewis J, Borchelt DR.

Alzheimers Res Ther. 2016 Sep 5;8:36. doi: 10.1186/s13195-016-0202-1.

3.

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.

4.

Activating frataxin expression by repeat-targeted nucleic acids.

Li L, Matsui M, Corey DR.

Nat Commun. 2016 Feb 4;7:10606. doi: 10.1038/ncomms10606.

5.

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.

6.

Transgenic animal models for study of the pathogenesis of Huntington's disease and therapy.

Chang R, Liu X, Li S, Li XJ.

Drug Des Devel Ther. 2015 Apr 15;9:2179-88. doi: 10.2147/DDDT.S58470. eCollection 2015. Review.

7.

Antisense therapy in neurology.

Lee JJ, Yokota T.

J Pers Med. 2013 Aug 2;3(3):144-76. doi: 10.3390/jpm3030144. Review.

8.

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.

9.

Systemic delivery of P42 peptide: a new weapon to fight Huntington's disease.

Arribat Y, Talmat-Amar Y, Paucard A, Lesport P, Bonneaud N, Bauer C, Bec N, Parmentier ML, Benigno L, Larroque C, Maurel P, Maschat F.

Acta Neuropathol Commun. 2014 Aug 5;2:86. doi: 10.1186/s40478-014-0086-x.

10.

Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity.

Sun X, Marque LO, Cordner Z, Pruitt JL, Bhat M, Li PP, Kannan G, Ladenheim EE, Moran TH, Margolis RL, Rudnicki DD.

Hum Mol Genet. 2014 Dec 1;23(23):6302-17. doi: 10.1093/hmg/ddu349. Epub 2014 Jul 4.

11.

Quantification assays for total and polyglutamine-expanded huntingtin proteins.

Macdonald D, Tessari MA, Boogaard I, Smith M, Pulli K, Szynol A, Albertus F, Lamers MB, Dijkstra S, Kordt D, Reindl W, Herrmann F, McAllister G, Fischer DF, Munoz-Sanjuan I.

PLoS One. 2014 May 9;9(5):e96854. doi: 10.1371/journal.pone.0096854. eCollection 2014.

12.

Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease.

Wang N, Gray M, Lu XH, Cantle JP, Holley SM, Greiner E, Gu X, Shirasaki D, Cepeda C, Li Y, Dong H, Levine MS, Yang XW.

Nat Med. 2014 May;20(5):536-41. doi: 10.1038/nm.3514. Epub 2014 Apr 28.

13.

Exploring the effect of sequence length and composition on allele-selective inhibition of human huntingtin expression by single-stranded silencing RNAs.

Hu J, Liu J, Yu D, Aiba Y, Lee S, Pendergraff H, Boubaker J, Artates JW, Lagier-Tourenne C, Lima WF, Swayze EE, Prakash TP, Corey DR.

Nucleic Acid Ther. 2014 Jun;24(3):199-209. doi: 10.1089/nat.2013.0476. Epub 2014 Apr 2.

14.

Cell-based therapies for Huntington's disease.

Chen Y, Carter RL, Cho IK, Chan AW.

Drug Discov Today. 2014 Jul;19(7):980-4. doi: 10.1016/j.drudis.2014.02.012. Epub 2014 Mar 12. Review.

15.

Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease.

Stanek LM, Sardi SP, Mastis B, Richards AR, Treleaven CM, Taksir T, Misra K, Cheng SH, Shihabuddin LS.

Hum Gene Ther. 2014 May;25(5):461-74. doi: 10.1089/hum.2013.200. Epub 2014 Mar 21.

16.

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.

17.

Small non-coding RNAs add complexity to the RNA pathogenic mechanisms in trinucleotide repeat expansion diseases.

Martí E, Estivill X.

Front Mol Neurosci. 2013 Dec 3;6:45. doi: 10.3389/fnmol.2013.00045. Review.

18.

HDAC4 reduction: a novel therapeutic strategy to target cytoplasmic huntingtin and ameliorate neurodegeneration.

Mielcarek M, Landles C, Weiss A, Bradaia A, Seredenina T, Inuabasi L, Osborne GF, Wadel K, Touller C, Butler R, Robertson J, Franklin SA, Smith DL, Park L, Marks PA, Wanker EE, Olson EN, Luthi-Carter R, van der Putten H, Beaumont V, Bates GP.

PLoS Biol. 2013 Nov;11(11):e1001717. doi: 10.1371/journal.pbio.1001717. Epub 2013 Nov 26.

19.

Allele-selective inhibition of expression of huntingtin and ataxin-3 by RNA duplexes containing unlocked nucleic acid substitutions.

Aiba Y, Hu J, Liu J, Xiang Q, Martinez C, Corey DR.

Biochemistry. 2013 Dec 23;52(51):9329-38. doi: 10.1021/bi4014209. Epub 2013 Nov 27.

20.

Aberrantly spliced HTT, a new player in Huntington's disease pathogenesis.

Gipson TA, Neueder A, Wexler NS, Bates GP, Housman D.

RNA Biol. 2013 Nov;10(11):1647-52. doi: 10.4161/rna.26706. Epub 2013 Oct 11.

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