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Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation.
Miller R, Paquette J, Barker A, Sapp E, McHugh N, Bramato B, Yamada N, Alterman J, Echeveria D, Yamada K, Watts J, Anaclet C, DiFiglia M, Khvorova A, Aronin N. Miller R, et al. Among authors: watts j. bioRxiv [Preprint]. 2024 Jun 8:2024.06.06.597639. doi: 10.1101/2024.06.06.597639. bioRxiv. 2024. Update in: Mol Ther Nucleic Acids. 2024 Oct 15;35(4):102359. doi: 10.1016/j.omtn.2024.102359 PMID: 38895198 Free PMC article. Updated. Preprint.
A combinatorial approach for achieving CNS-selective RNAi.
Ferguson CM, Godinho BMDC, Echeverria D, Hassler M, Vangjeli L, Sousa J, McHugh N, Alterman J, Hariharan V, Krishnamurthy PM, Watts J, Rogaev E, Khvorova A. Ferguson CM, et al. Among authors: watts j. Nucleic Acids Res. 2024 May 22;52(9):5273-5284. doi: 10.1093/nar/gkae100. Nucleic Acids Res. 2024. PMID: 38348876 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.
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.
1,759 results