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Items: 1 to 20 of 31


Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease.

Hadaczek P, Stanek L, Ciesielska A, Sudhakar V, Samaranch L, Pivirotto P, Bringas J, O'Riordan C, Mastis B, San Sebastian W, Forsayeth J, Cheng SH, Bankiewicz KS, Shihabuddin LS.

Mol Ther Methods Clin Dev. 2016 Jun 29;3:16037. doi: 10.1038/mtm.2016.37.


Intestinal brush-border Na+/H+ exchanger-3 drives H+-coupled iron absorption in the mouse.

Shawki A, Engevik MA, Kim RS, Knight PB, Baik RA, Anthony SR, Worrell RT, Shull GE, Mackenzie B.

Am J Physiol Gastrointest Liver Physiol. 2016 Sep 1;311(3):G423-30. doi: 10.1152/ajpgi.00167.2016.


Recent Trends in Detection of Huntingtin and Preclinical Models of Huntington's Disease.

Mantha N, Das NG, Das SK.

ISRN Mol Biol. 2014 May 14;2014:190976. doi: 10.1155/2014/190976. Review.


Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.

Hocquemiller M, Giersch L, Audrain M, Parker S, Cartier N.

Hum Gene Ther. 2016 Jul;27(7):478-96. doi: 10.1089/hum.2016.087.


Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.

Keiser MS, Kordasiewicz HB, McBride JL.

Hum Mol Genet. 2016 Apr 15;25(R1):R53-64. doi: 10.1093/hmg/ddv442. Review.


Intestinal DMT1 is critical for iron absorption in the mouse but is not required for the absorption of copper or manganese.

Shawki A, Anthony SR, Nose Y, Engevik MA, Niespodzany EJ, Barrientos T, Öhrvik H, Worrell RT, Thiele DJ, Mackenzie B.

Am J Physiol Gastrointest Liver Physiol. 2015 Oct 15;309(8):G635-47. doi: 10.1152/ajpgi.00160.2015.


AAV9 delivering a modified human Mullerian inhibiting substance as a gene therapy in patient-derived xenografts of ovarian cancer.

Pépin D, Sosulski A, Zhang L, Wang D, Vathipadiekal V, Hendren K, Coletti CM, Yu A, Castro CM, Birrer MJ, Gao G, Donahoe PK.

Proc Natl Acad Sci U S A. 2015 Aug 11;112(32):E4418-27. doi: 10.1073/pnas.1510604112.


Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression.

Southwell AL, Smith SE, Davis TR, Caron NS, Villanueva EB, Xie Y, Collins JA, Ye ML, Sturrock A, Leavitt BR, Schrum AG, Hayden MR.

Sci Rep. 2015 Jul 15;5:12166. doi: 10.1038/srep12166.


Viral vector mediated expression of mutant huntingtin in the dorsal raphe produces disease-related neuropathology but not depressive-like behaviors in wildtype mice.

Pitzer M, Lueras J, Warden A, Weber S, McBride J.

Brain Res. 2015 May 22;1608:177-90. doi: 10.1016/j.brainres.2015.02.027.


In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.

Southwell AL, Skotte NH, Kordasiewicz HB, Østergaard ME, Watt AT, Carroll JB, Doty CN, Villanueva EB, Petoukhov E, Vaid K, Xie Y, Freier SM, Swayze EE, Seth PP, Bennett CF, Hayden MR.

Mol Ther. 2014 Dec;22(12):2093-106. doi: 10.1038/mt.2014.153.


Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts.

Scholefield J, Watson L, Smith D, Greenberg J, Wood MJ.

Eur J Hum Genet. 2014 Dec;22(12):1369-75. doi: 10.1038/ejhg.2014.39.


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.


KIBRA (KIdney/BRAin protein) regulates learning and memory and stabilizes Protein kinase Mζ.

Vogt-Eisele A, Krüger C, Duning K, Weber D, Spoelgen R, Pitzer C, Plaas C, Eisenhardt G, Meyer A, Vogt G, Krieger M, Handwerker E, Wennmann DO, Weide T, Skryabin BV, Klugmann M, Pavenstädt H, Huentelmann MJ, Kremerskothen J, Schneider A.

J Neurochem. 2014 Mar;128(5):686-700. doi: 10.1111/jnc.12480.


Epigenetic mechanisms of neurodegeneration in Huntington's disease.

Lee J, Hwang YJ, Kim KY, Kowall NW, Ryu H.

Neurotherapeutics. 2013 Oct;10(4):664-76. doi: 10.1007/s13311-013-0206-5. Review.


Glial promoter selectivity following AAV-delivery to the immature brain.

von Jonquieres G, Mersmann N, Klugmann CB, Harasta AE, Lutz B, Teahan O, Housley GD, Fröhlich D, Krämer-Albers EM, Klugmann M.

PLoS One. 2013 Jun 14;8(6):e65646. doi: 10.1371/journal.pone.0065646.


The hematopoietic cytokine granulocyte-macrophage colony stimulating factor is important for cognitive functions.

Krieger M, Both M, Kranig SA, Pitzer C, Klugmann M, Vogt G, Draguhn A, Schneider A.

Sci Rep. 2012;2:697.


Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.

Kordasiewicz HB, Stanek LM, Wancewicz EV, Mazur C, McAlonis MM, Pytel KA, Artates JW, Weiss A, Cheng SH, Shihabuddin LS, Hung G, Bennett CF, Cleveland DW.

Neuron. 2012 Jun 21;74(6):1031-44. doi: 10.1016/j.neuron.2012.05.009.


Nucleic Acid-Based Therapy Approaches for Huntington's Disease.

Vagner T, Young D, Mouravlev A.

Neurol Res Int. 2012;2012:358370. doi: 10.1155/2012/358370.


Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease.

McBride JL, Pitzer MR, Boudreau RL, Dufour B, Hobbs T, Ojeda SR, Davidson BL.

Mol Ther. 2011 Dec;19(12):2152-62. doi: 10.1038/mt.2011.219.

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