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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: doty cn. Hum Mol Genet. 2017 Mar 15;26(6):1115-1132. doi: 10.1093/hmg/ddx021. Hum Mol Genet. 2017. PMID: 28104789
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: doty cn. 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: doty cn. 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.
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: doty cn. 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.
23 results