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Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.
Tran H, Moazami MP, Yang H, McKenna-Yasek D, Douthwright CL, Pinto C, Metterville J, Shin M, Sanil N, Dooley C, Puri A, Weiss A, Wightman N, Gray-Edwards H, Marosfoi M, King RM, Kenderdine T, Fabris D, Bowser R, Watts JK, Brown RH Jr. Tran H, et al. Among authors: watts jk. Nat Med. 2022 Jan;28(1):117-124. doi: 10.1038/s41591-021-01557-6. Epub 2021 Dec 23. Nat Med. 2022. PMID: 34949835 Free PMC article.
Locked Nucleic Acid Gapmers and Conjugates Potently Silence ADAM33, an Asthma-Associated Metalloprotease with Nuclear-Localized mRNA.
Pendergraff HM, Krishnamurthy PM, Debacker AJ, Moazami MP, Sharma VK, Niitsoo L, Yu Y, Tan YN, Haitchi HM, Watts JK. Pendergraff HM, et al. Among authors: watts jk. Mol Ther Nucleic Acids. 2017 Sep 15;8:158-168. doi: 10.1016/j.omtn.2017.06.012. Epub 2017 Jun 21. Mol Ther Nucleic Acids. 2017. PMID: 28918018 Free PMC article.
Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs.
Osborn MF, Coles AH, Golebiowski D, Echeverria D, Moazami MP, Watts JK, Sena-Esteves M, Khvorova A. Osborn MF, et al. Among authors: watts jk. Mol Cancer Ther. 2018 Jun;17(6):1251-1258. doi: 10.1158/1535-7163.MCT-17-1144. Epub 2018 Apr 13. Mol Cancer Ther. 2018. PMID: 29654062 Free PMC article.
Nucleic Acid Therapeutics for Neurological Diseases.
Watts JK, Brown RH, Khvorova A. Watts JK, et al. Neurotherapeutics. 2019 Apr;16(2):245-247. doi: 10.1007/s13311-019-00736-1. Neurotherapeutics. 2019. PMID: 31037650 Free PMC article. No abstract available.
Poly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD.
Krishnan G, Raitcheva D, Bartlett D, Prudencio M, McKenna-Yasek DM, Douthwright C, Oskarsson BE, Ladha S, King OD, Barmada SJ, Miller TM, Bowser R, Watts JK, Petrucelli L, Brown RH, Kankel MW, Gao FB. Krishnan G, et al. Among authors: watts jk. Nat Commun. 2022 May 19;13(1):2799. doi: 10.1038/s41467-022-30387-4. Nat Commun. 2022. PMID: 35589711 Free PMC article.
Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts.
Shin M, Chan IL, Cao Y, Gruntman AM, Lee J, Sousa J, Rodríguez TC, Echeverria D, Devi G, Debacker AJ, Moazami MP, Krishnamurthy PM, Rembetsy-Brown JM, Kelly K, Yukselen O, Donnard E, Parsons TJ, Khvorova A, Sontheimer EJ, Maehr R, Garber M, Watts JK. Shin M, et al. Among authors: watts jk. Nucleic Acids Res. 2022 Aug 26;50(15):8418-8430. doi: 10.1093/nar/gkac630. Nucleic Acids Res. 2022. PMID: 35920332 Free PMC article.
66 results