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Items: 37


Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease.

Caron NS, Southwell AL, Brouwers CC, Cengio LD, Xie Y, Black HF, Anderson LM, Ko S, Zhu X, van Deventer SJ, Evers MM, Konstantinova P, Hayden MR.

Nucleic Acids Res. 2020 Jan 10;48(1):36-54. doi: 10.1093/nar/gkz976.


Huntingtin suppression restores cognitive function in a mouse model of Huntington's disease.

Southwell AL, Kordasiewicz HB, Langbehn D, Skotte NH, Parsons MP, Villanueva EB, Caron NS, Østergaard ME, Anderson LM, Xie Y, Cengio LD, Findlay-Black H, Doty CN, Fitsimmons B, Swayze EE, Seth PP, Raymond LA, Frank Bennett C, Hayden MR.

Sci Transl Med. 2018 Oct 3;10(461). pii: eaar3959. doi: 10.1126/scitranslmed.aar3959.


Preventing mutant huntingtin proteolysis and intermittent fasting promote autophagy in models of Huntington disease.

Ehrnhoefer DE, Martin DDO, Schmidt ME, Qiu X, Ladha S, Caron NS, Skotte NH, Nguyen YTN, Vaid K, Southwell AL, Engemann S, Franciosi S, Hayden MR.

Acta Neuropathol Commun. 2018 Mar 6;6(1):16. doi: 10.1186/s40478-018-0518-0.


HACE1 is essential for astrocyte mitochondrial function and influences Huntington disease phenotypes in vivo.

Ehrnhoefer DE, Southwell AL, Sivasubramanian M, Qiu X, Villanueva EB, Xie Y, Waltl S, Anderson L, Fazeli A, Casal L, Felczak B, Tsang M, Hayden MR.

Hum Mol Genet. 2018 Jan 15;27(2):239-253. doi: 10.1093/hmg/ddx394.


A novel humanized mouse model of Huntington disease for preclinical development of therapeutics targeting mutant huntingtin alleles.

Southwell AL, Skotte NH, Villanueva EB, Østergaard ME, Gu X, Kordasiewicz HB, Kay C, Cheung D, Xie Y, Waltl S, Dal Cengio L, Findlay-Black H, Doty CN, Petoukhov E, Iworima D, Slama R, Ooi J, Pouladi MA, Yang XW, Swayze EE, Seth PP, Hayden MR.

Hum Mol Genet. 2017 Mar 15;26(6):1115-1132. doi: 10.1093/hmg/ddx021.


Sudden death due to paralysis and synaptic and behavioral deficits when Hip14/Zdhhc17 is deleted in adult mice.

Sanders SS, Parsons MP, Mui KK, Southwell AL, Franciosi S, Cheung D, Waltl S, Raymond LA, Hayden MR.

BMC Biol. 2016 Dec 7;14(1):108.


An enhanced Q175 knock-in mouse model of Huntington disease with higher mutant huntingtin levels and accelerated disease phenotypes.

Southwell AL, Smith-Dijak A, Kay C, Sepers M, Villanueva EB, Parsons MP, Xie Y, Anderson L, Felczak B, Waltl S, Ko S, Cheung D, Dal Cengio L, Slama R, Petoukhov E, Raymond LA, Hayden MR.

Hum Mol Genet. 2016 Sep 1;25(17):3654-3675. doi: 10.1093/hmg/ddw212. Epub 2016 Jul 4.


Enhanced immune response to MMP3 stimulation in microglia expressing mutant huntingtin.

Connolly C, Magnusson-Lind A, Lu G, Wagner PK, Southwell AL, Hayden MR, Björkqvist M, Leavitt BR.

Neuroscience. 2016 Jun 14;325:74-88. doi: 10.1016/j.neuroscience.2016.03.031. Epub 2016 Mar 23.


Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease.

Miniarikova J, Zanella I, Huseinovic A, van der Zon T, Hanemaaijer E, Martier R, Koornneef A, Southwell AL, Hayden MR, van Deventer SJ, Petry H, Konstantinova P.

Mol Ther Nucleic Acids. 2016 Mar 22;5:e297. doi: 10.1038/mtna.2016.7.


Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry.

Kay C, Collins JA, Skotte NH, Southwell AL, Warby SC, Caron NS, Doty CN, Nguyen B, Griguoli A, Ross CJ, Squitieri F, Hayden MR.

Mol Ther. 2015 Nov;23(11):1759-1771. doi: 10.1038/mt.2015.128. Epub 2015 Jul 23.


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.


HD iPSC-derived neural progenitors accumulate in culture and are susceptible to BDNF withdrawal due to glutamate toxicity.

Mattis VB, Tom C, Akimov S, Saeedian J, Østergaard ME, Southwell AL, Doty CN, Ornelas L, Sahabian A, Lenaeus L, Mandefro B, Sareen D, Arjomand J, Hayden MR, Ross CA, Svendsen CN.

Hum Mol Genet. 2015 Jun 1;24(11):3257-71. doi: 10.1093/hmg/ddv080. Epub 2015 Mar 3.


Anti-semaphorin 4D immunotherapy ameliorates neuropathology and some cognitive impairment in the YAC128 mouse model of Huntington disease.

Southwell AL, Franciosi S, Villanueva EB, Xie Y, Winter LA, Veeraraghavan J, Jonason A, Felczak B, Zhang W, Kovalik V, Waltl S, Hall G, Pouladi MA, Smith ES, Bowers WJ, Zauderer M, Hayden MR.

Neurobiol Dis. 2015 Apr;76:46-56. doi: 10.1016/j.nbd.2015.01.002. Epub 2015 Feb 3.


Biophysical and biological characterization of hairpin and molecular beacon RNase H active antisense oligonucleotides.

Østergaard ME, Thomas G, Koller E, Southwell AL, Hayden MR, Seth PP.

ACS Chem Biol. 2015 May 15;10(5):1227-33. doi: 10.1021/cb500880f. Epub 2015 Feb 13.


Paul H. Patterson (October 22, 1943 - June 25, 2014).

Southwell AL, Wexler NS.

J Huntingtons Dis. 2014;3(3):221-4. doi: 10.3233/JHD-149006. No abstract available.


Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.

Skotte NH, Southwell AL, Østergaard ME, Carroll JB, Warby SC, Doty CN, Petoukhov E, Vaid K, Kordasiewicz H, Watt AT, Freier SM, Hung G, Seth PP, Bennett CF, Swayze EE, Hayden MR.

PLoS One. 2014 Sep 10;9(9):e107434. doi: 10.1371/journal.pone.0107434. eCollection 2014.


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. Epub 2014 Aug 7.


Striatal synaptic dysfunction and hippocampal plasticity deficits in the Hu97/18 mouse model of Huntington disease.

Kolodziejczyk K, Parsons MP, Southwell AL, Hayden MR, Raymond LA.

PLoS One. 2014 Apr 11;9(4):e94562. doi: 10.1371/journal.pone.0094562. eCollection 2014.


Personalized gene silencing therapeutics for Huntington disease.

Kay C, Skotte NH, Southwell AL, Hayden MR.

Clin Genet. 2014 Jul;86(1):29-36. doi: 10.1111/cge.12385. Epub 2014 Apr 11. Review.


HACE1 reduces oxidative stress and mutant Huntingtin toxicity by promoting the NRF2 response.

Rotblat B, Southwell AL, Ehrnhoefer DE, Skotte NH, Metzler M, Franciosi S, Leprivier G, Somasekharan SP, Barokas A, Deng Y, Tang T, Mathers J, Cetinbas N, Daugaard M, Kwok B, Li L, Carnie CJ, Fink D, Nitsch R, Galpin JD, Ahern CA, Melino G, Penninger JM, Hayden MR, Sorensen PH.

Proc Natl Acad Sci U S A. 2014 Feb 25;111(8):3032-7. doi: 10.1073/pnas.1314421111. Epub 2014 Feb 10.


Bidirectional control of postsynaptic density-95 (PSD-95) clustering by Huntingtin.

Parsons MP, Kang R, Buren C, Dau A, Southwell AL, Doty CN, Sanders SS, Hayden MR, Raymond LA.

J Biol Chem. 2014 Feb 7;289(6):3518-28. doi: 10.1074/jbc.M113.513945. Epub 2013 Dec 17.


Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.

Østergaard ME, Southwell AL, Kordasiewicz H, Watt AT, Skotte NH, Doty CN, Vaid K, Villanueva EB, Swayze EE, Bennett CF, Hayden MR, Seth PP.

Nucleic Acids Res. 2013 Nov;41(21):9634-50. doi: 10.1093/nar/gkt725. Epub 2013 Aug 19.


Hip14l-deficient mice develop neuropathological and behavioural features of Huntington disease.

Sutton LM, Sanders SS, Butland SL, Singaraja RR, Franciosi S, Southwell AL, Doty CN, Schmidt ME, Mui KK, Kovalik V, Young FB, Zhang W, Hayden MR.

Hum Mol Genet. 2013 Feb 1;22(3):452-65. doi: 10.1093/hmg/dds441. Epub 2012 Oct 16.


Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases.

Southwell AL, Skotte NH, Bennett CF, Hayden MR.

Trends Mol Med. 2012 Nov;18(11):634-43. doi: 10.1016/j.molmed.2012.09.001. Epub 2012 Sep 28. Review.


A fully humanized transgenic mouse model of Huntington disease.

Southwell AL, Warby SC, Carroll JB, Doty CN, Skotte NH, Zhang W, Villanueva EB, Kovalik V, Xie Y, Pouladi MA, Collins JA, Yang XW, Franciosi S, Hayden MR.

Hum Mol Genet. 2013 Jan 1;22(1):18-34. doi: 10.1093/hmg/dds397. Epub 2012 Sep 21.


Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice.

Pouladi MA, Stanek LM, Xie Y, Franciosi S, Southwell AL, Deng Y, Butland S, Zhang W, Cheng SH, Shihabuddin LS, Hayden MR.

Hum Mol Genet. 2012 May 15;21(10):2219-32. doi: 10.1093/hmg/dds037. Epub 2012 Feb 9.


Caspase-6-Resistant Mutant Huntingtin Does not Rescue the Toxic Effects of Caspase-Cleavable Mutant Huntingtin in vivo.

Graham RK, Deng Y, Pouladi MA, Vaid K, Ehrnhoefer D, Southwell AL, Bissada N, Franciosi S, Hayden MR.

J Huntingtons Dis. 2012;1(2):243-60. doi: 10.3233/JHD-120038.


Age-Dependent Resistance to Excitotoxicity in Htt CAG140 Mice and the Effect of Strain Background.

Strong MK, Southwell AL, Yonan JM, Hayden MR, Macgregor GR, Thompson LM, Steward O.

J Huntingtons Dis. 2012;1(2):221-41. doi: 10.3233/JHD-129005.


Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.

Carroll JB, Warby SC, Southwell AL, Doty CN, Greenlee S, Skotte N, Hung G, Bennett CF, Freier SM, Hayden MR.

Mol Ther. 2011 Dec;19(12):2178-85. doi: 10.1038/mt.2011.201. Epub 2011 Oct 4.


Recombinant Intrabodies as Molecular Tools and Potential Therapeutics for Huntington’s Disease.

Ali K, Southwell AL, Bugg CW, Ko JC, Patterson PH.

In: Lo DC, Hughes RE, editors. Neurobiology of Huntington's Disease: Applications to Drug Discovery. Boca Raton (FL): CRC Press/Taylor & Francis; 2011. Chapter 10.


Mice lacking caspase-2 are protected from behavioral changes, but not pathology, in the YAC128 model of Huntington disease.

Carroll JB, Southwell AL, Graham RK, Lerch JP, Ehrnhoefer DE, Cao LP, Zhang WN, Deng Y, Bissada N, Henkelman RM, Hayden MR.

Mol Neurodegener. 2011 Aug 19;6:59. doi: 10.1186/1750-1326-6-59.


Gene therapy in mouse models of huntington disease.

Southwell AL, Patterson PH.

Neuroscientist. 2011 Apr;17(2):153-62. doi: 10.1177/1073858410386236. Review.


Perturbation with intrabodies reveals that calpain cleavage is required for degradation of huntingtin exon 1.

Southwell AL, Bugg CW, Kaltenbach LS, Dunn D, Butland S, Weiss A, Paganetti P, Lo DC, Patterson PH.

PLoS One. 2011 Jan 31;6(1):e16676. doi: 10.1371/journal.pone.0016676.


Antibody therapy in neurodegenerative disease.

Southwell AL, Patterson PH.

Rev Neurosci. 2010;21(4):273-87.


Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington's disease.

Southwell AL, Ko J, Patterson PH.

J Neurosci. 2009 Oct 28;29(43):13589-602. doi: 10.1523/JNEUROSCI.4286-09.2009.


Intrabodies binding the proline-rich domains of mutant huntingtin increase its turnover and reduce neurotoxicity.

Southwell AL, Khoshnan A, Dunn DE, Bugg CW, Lo DC, Patterson PH.

J Neurosci. 2008 Sep 3;28(36):9013-20. doi: 10.1523/JNEUROSCI.2747-08.2008.


Atypical expansion in mice of the sensory neuron-specific Mrg G protein-coupled receptor family.

Zylka MJ, Dong X, Southwell AL, Anderson DJ.

Proc Natl Acad Sci U S A. 2003 Aug 19;100(17):10043-8. Epub 2003 Aug 8.

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