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Targeting Huntingtin Expression in Patients with Huntington's Disease.
Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft C, Barker RA, Blair NF, Craufurd D, Priller J, Rickards H, Rosser A, Kordasiewicz HB, Czech C, Swayze EE, Norris DA, Baumann T, Gerlach I, Schobel SA, Paz E, Smith AV, Bennett CF, Lane RM; Phase 1–2a IONIS-HTTRx Study Site Teams. Tabrizi SJ, et al. Among authors: Kordasiewicz HB. N Engl J Med. 2019 Jun 13;380(24):2307-2316. doi: 10.1056/NEJMoa1900907. Epub 2019 May 6. N Engl J Med. 2019. PMID: 31059641 Clinical Trial.
Peripheral huntingtin silencing does not ameliorate central signs of disease in the B6.HttQ111/+ mouse model of Huntington's disease.
Coffey SR, Bragg RM, Minnig S, Ament SA, Cantle JP, Glickenhaus A, Shelnut D, Carrillo JM, Shuttleworth DD, Rodier JA, Noguchi K, Bennett CF, Price ND, Kordasiewicz HB, Carroll JB. Coffey SR, et al. PLoS One. 2017 Apr 28;12(4):e0175968. doi: 10.1371/journal.pone.0175968. eCollection 2017. PLoS One. 2017. PMID: 28453524 Free PMC article.
Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy.
DeVos SL, Miller RL, Schoch KM, Holmes BB, Kebodeaux CS, Wegener AJ, Chen G, Shen T, Tran H, Nichols B, Zanardi TA, Kordasiewicz HB, Swayze EE, Bennett CF, Diamond MI, Miller TM. DeVos SL, et al. Sci Transl Med. 2017 Jan 25;9(374):eaag0481. doi: 10.1126/scitranslmed.aag0481. Sci Transl Med. 2017. PMID: 28123067 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. Mol Ther. 2014 Dec;22(12):2093-106. doi: 10.1038/mt.2014.153. Epub 2014 Aug 7. Mol Ther. 2014. PMID: 25101598 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. 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: Kordasiewicz HB. Sci Transl Med. 2018 Oct 3;10(461):eaar3959. doi: 10.1126/scitranslmed.aar3959. Sci Transl Med. 2018. PMID: 30282695
Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.
McLoughlin HS, Moore LR, Chopra R, Komlo R, McKenzie M, Blumenstein KG, Zhao H, Kordasiewicz HB, Shakkottai VG, Paulson HL. McLoughlin HS, et al. Among authors: Kordasiewicz HB. Ann Neurol. 2018 Jul;84(1):64-77. doi: 10.1002/ana.25264. Epub 2018 Aug 6. Ann Neurol. 2018. PMID: 29908063 Free PMC article.
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