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A single-cell map of antisense oligonucleotide activity in the brain.
Mortberg MA, Gentile JE, Nadaf NM, Vanderburg C, Simmons S, Dubinsky D, Slamin A, Maldonado S, Petersen CL, Jones N, Kordasiewicz HB, Zhao HT, Vallabh SM, Minikel EV. Mortberg MA, et al. Among authors: zhao ht. Nucleic Acids Res. 2023 Aug 11;51(14):7109-7124. doi: 10.1093/nar/gkad371. Nucleic Acids Res. 2023. PMID: 37188501 Free PMC article.
Prion protein lowering is a disease-modifying therapy across prion disease stages, strains and endpoints.
Minikel EV, Zhao HT, Le J, O'Moore J, Pitstick R, Graffam S, Carlson GA, Kavanaugh MP, Kriz J, Kim JB, Ma J, Wille H, Aiken J, McKenzie D, Doh-Ura K, Beck M, O'Keefe R, Stathopoulos J, Caron T, Schreiber SL, Carroll JB, Kordasiewicz HB, Cabin DE, Vallabh SM. Minikel EV, et al. Among authors: zhao ht. Nucleic Acids Res. 2020 Nov 4;48(19):10615-10631. doi: 10.1093/nar/gkaa616. Nucleic Acids Res. 2020. PMID: 32776089 Free PMC article.
Antisense oligonucleotides extend survival of prion-infected mice.
Raymond GJ, Zhao HT, Race B, Raymond LD, Williams K, Swayze EE, Graffam S, Le J, Caron T, Stathopoulos J, O'Keefe R, Lubke LL, Reidenbach AG, Kraus A, Schreiber SL, Mazur C, Cabin DE, Carroll JB, Minikel EV, Kordasiewicz H, Caughey B, Vallabh SM. Raymond GJ, et al. Among authors: zhao ht. JCI Insight. 2019 Jul 30;5(16):e131175. doi: 10.1172/jci.insight.131175. JCI Insight. 2019. PMID: 31361599 Free PMC article.
The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration.
Jafar-Nejad P, Powers B, Soriano A, Zhao H, Norris DA, Matson J, DeBrosse-Serra B, Watson J, Narayanan P, Chun SJ, Mazur C, Kordasiewicz H, Swayze EE, Rigo F. Jafar-Nejad P, et al. Nucleic Acids Res. 2021 Jan 25;49(2):657-673. doi: 10.1093/nar/gkaa1235. Nucleic Acids Res. 2021. PMID: 33367834 Free PMC article.
Regional variability and genotypic and pharmacodynamic effects on PrP concentration in the CNS.
Mortberg MA, Zhao HT, Reidenbach AG, Gentile JE, Kuhn E, O'Moore J, Dooley PM, Connors TR, Mazur C, Allen SW, Trombetta BA, McManus A, Moore MR, Liu J, Cabin DE, Kordasiewicz HB, Mathews J, Arnold SE, Vallabh SM, Minikel EV. Mortberg MA, et al. Among authors: zhao ht. JCI Insight. 2022 Mar 22;7(6):e156532. doi: 10.1172/jci.insight.156532. JCI Insight. 2022. PMID: 35133987 Free PMC article.
244 results