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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. Southwell AL, et al. Among authors: villanueva eb. Mol Ther. 2014 Dec;22(12):2093-2106. doi: 10.1038/mt.2014.153. Epub 2014 Aug 7. Mol Ther. 2014. PMID: 25101598 Free PMC article.
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. Southwell AL, et al. Among authors: villanueva eb. Hum Mol Genet. 2013 Jan 1;22(1):18-34. doi: 10.1093/hmg/dds397. Epub 2012 Sep 21. Hum Mol Genet. 2013. PMID: 23001568 Free PMC article.
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. Østergaard ME, et al. Among authors: villanueva eb. Nucleic Acids Res. 2013 Nov;41(21):9634-50. doi: 10.1093/nar/gkt725. Epub 2013 Aug 19. Nucleic Acids Res. 2013. PMID: 23963702 Free PMC article.
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. Southwell AL, et al. Among authors: villanueva eb. Sci Rep. 2015 Jul 15;5:12166. doi: 10.1038/srep12166. Sci Rep. 2015. PMID: 26174131 Free PMC article.
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. Southwell AL, et al. Among authors: villanueva eb. Hum Mol Genet. 2016 Sep 1;25(17):3654-3675. doi: 10.1093/hmg/ddw212. Epub 2016 Jul 4. Hum Mol Genet. 2016. PMID: 27378694 Free PMC article.
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. Southwell AL, et al. Among authors: villanueva eb. Hum Mol Genet. 2017 Mar 15;26(6):1115-1132. doi: 10.1093/hmg/ddx021. Hum Mol Genet. 2017. PMID: 28104789
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. Southwell AL, et al. Among authors: villanueva eb. Sci Transl Med. 2018 Oct 3;10(461):eaar3959. doi: 10.1126/scitranslmed.aar3959. Sci Transl Med. 2018. PMID: 30282695
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. Southwell AL, et al. Among authors: villanueva eb. Neurobiol Dis. 2015 Apr;76:46-56. doi: 10.1016/j.nbd.2015.01.002. Epub 2015 Feb 3. Neurobiol Dis. 2015. PMID: 25662335 Free article.
19 results