{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T19:33:57Z","timestamp":1790278437356,"version":"4.1.0"},"reference-count":63,"publisher":"SAGE Publications","issue":"3","license":[{"start":{"date-parts":[[2021,6,1]],"date-time":"2021-06-01T00:00:00Z","timestamp":1622505600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Nucleic Acid Therapeutics"],"published-print":{"date-parts":[[2021,6,1]]},"abstract":"<jats:p>\n                    A series of 2\u2032-deoxy and novel 2\u2032-\n                    <jats:italic toggle=\"yes\">O<\/jats:italic>\n                    -methyl and 2\u2032-\n                    <jats:italic toggle=\"yes\">O<\/jats:italic>\n                    -(2-methoxyethyl) (2\u2032-MOE) oligonucleotides with internucleotide methanesulfonyl (mesyl, \u03bc) or 1-butanesulfonyl (busyl, \u03b2) phosphoramidate groups has been synthesized for evaluation as potential splice-switching oligonucleotides. Evaluation of their splice-switching activity in spinal muscular atrophy patient-derived fibroblasts revealed no significant difference in splice-switching efficacy between 2\u2032-MOE mesyl oligonucleotide and the corresponding phosphorothioate (nusinersen). Yet, a survival study with model neonatal mice has shown the antisense 2\u2032-MOE mesyl oligonucleotide to be inferior to nusinersen at the highest dose of 40\u2009mg\/kg. A reason for their lower activity\n                    <jats:italic toggle=\"yes\">in vivo<\/jats:italic>\n                    as ascertained by cellular uptake study by fluorescent confocal microscopy in HEK293 cell line could possibly be ascribed to compromised endosomal release and\/or nuclear uptake of the 2\u2032-OMe or 2\u2032-MOE \u03bc- and \u03b2-oligonucleotides compared to their phosphorothioate analog.\n                  <\/jats:p>","DOI":"10.1089\/nat.2020.0860","type":"journal-article","created":{"date-parts":[[2021,5,14]],"date-time":"2021-05-14T15:29:04Z","timestamp":1621006144000},"page":"190-200","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":28,"title":["Mesyl Phosphoramidate Oligonucleotides as Potential Splice-Switching Agents: Impact of Backbone Structure on Activity and Intracellular Localization"],"prefix":"10.1177","volume":"31","author":[{"given":"Suzan 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