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Items: 1 to 20 of 119

1.

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
2.

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
3.

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.

4.

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.

5.

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.

6.

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
7.

Msh2 acts in medium-spiny striatal neurons as an enhancer of CAG instability and mutant huntingtin phenotypes in Huntington's disease knock-in mice.

Kovalenko M, Dragileva E, St Claire J, Gillis T, Guide JR, New J, Dong H, Kucherlapati R, Kucherlapati MH, Ehrlich ME, Lee JM, Wheeler VC.

PLoS One. 2012;7(9):e44273. doi: 10.1371/journal.pone.0044273. Epub 2012 Sep 7.

8.

Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice.

Dufour BD, Smith CA, Clark RL, Walker TR, McBride JL.

Mol Ther. 2014 Apr;22(4):797-810. doi: 10.1038/mt.2013.289. Epub 2014 Jan 6.

9.

Brain urea increase is an early Huntington's disease pathogenic event observed in a prodromal transgenic sheep model and HD cases.

Handley RR, Reid SJ, Brauning R, Maclean P, Mears ER, Fourie I, Patassini S, Cooper GJS, Rudiger SR, McLaughlan CJ, Verma PJ, Gusella JF, MacDonald ME, Waldvogel HJ, Bawden CS, Faull RLM, Snell RG.

Proc Natl Acad Sci U S A. 2017 Dec 26;114(52):E11293-E11302. doi: 10.1073/pnas.1711243115. Epub 2017 Dec 11.

10.

Characterization of a rat model of Huntington's disease based on targeted expression of mutant huntingtin in the forebrain using adeno-associated viral vectors.

Gabery S, Sajjad MU, Hult S, Soylu R, Kirik D, Petersén Å.

Eur J Neurosci. 2012 Sep;36(6):2789-800. doi: 10.1111/j.1460-9568.2012.08193.x. Epub 2012 Jun 25.

PMID:
22731249
11.

Full length mutant huntingtin is required for altered Ca2+ signaling and apoptosis of striatal neurons in the YAC mouse model of Huntington's disease.

Zhang H, Li Q, Graham RK, Slow E, Hayden MR, Bezprozvanny I.

Neurobiol Dis. 2008 Jul;31(1):80-8. doi: 10.1016/j.nbd.2008.03.010. Epub 2008 Apr 16.

12.

Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic.

Miniarikova J, Evers MM, Konstantinova P.

Mol Ther. 2018 Apr 4;26(4):947-962. doi: 10.1016/j.ymthe.2018.02.002. Epub 2018 Feb 8. Review.

13.

Novel BAC Mouse Model of Huntington's Disease with 225 CAG Repeats Exhibits an Early Widespread and Stable Degenerative Phenotype.

Wegrzynowicz M, Bichell TJ, Soares BD, Loth MK, McGlothan JS, Mori S, Alikhan FS, Hua K, Coughlin JM, Holt HK, Jetter CS, Pomper MG, Osmand AP, Guilarte TR, Bowman AB.

J Huntingtons Dis. 2015;4(1):17-36.

14.

An ovine transgenic Huntington's disease model.

Jacobsen JC, Bawden CS, Rudiger SR, McLaughlan CJ, Reid SJ, Waldvogel HJ, MacDonald ME, Gusella JF, Walker SK, Kelly JM, Webb GC, Faull RL, Rees MI, Snell RG.

Hum Mol Genet. 2010 May 15;19(10):1873-82. doi: 10.1093/hmg/ddq063. Epub 2010 Feb 13.

15.

AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease.

Franich NR, Fitzsimons HL, Fong DM, Klugmann M, During MJ, Young D.

Mol Ther. 2008 May;16(5):947-56. doi: 10.1038/mt.2008.50. Epub 2008 Mar 25.

16.

Frequency of nuclear mutant huntingtin inclusion formation in neurons and glia is cell-type-specific.

Jansen AH, van Hal M, Op den Kelder IC, Meier RT, de Ruiter AA, Schut MH, Smith DL, Grit C, Brouwer N, Kamphuis W, Boddeke HW, den Dunnen WF, van Roon WM, Bates GP, Hol EM, Reits EA.

Glia. 2017 Jan;65(1):50-61. doi: 10.1002/glia.23050. Epub 2016 Sep 12.

17.

CRISPR-Cas9 Mediated Gene-Silencing of the Mutant Huntingtin Gene in an In Vitro Model of Huntington's Disease.

Kolli N, Lu M, Maiti P, Rossignol J, Dunbar GL.

Int J Mol Sci. 2017 Apr 2;18(4). pii: E754. doi: 10.3390/ijms18040754.

18.

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.

19.

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
20.

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.

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