{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,8]],"date-time":"2026-10-08T03:04:35Z","timestamp":1791428675171,"version":"4.3.3"},"reference-count":54,"publisher":"Oxford University Press (OUP)","issue":"10","license":[{"start":{"date-parts":[[2026,5,21]],"date-time":"2026-05-21T00:00:00Z","timestamp":1779321600000},"content-version":"vor","delay-in-days":1,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"IONIS"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,5,20]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Aberrant RNA splicing contributes to many human diseases, and splice-switching antisense oligonucleotides are ideally suited as a therapeutic strategy to modulate splicing and restore normal gene expression. Nusinersen (Spinraza\u2122) has revolutionized the treatment of spinal muscular atrophy. It is a splice-switching oligonucleotide (SSO) modified with 2\u2032-O-methoxyethyl (MOE). Here, we evaluate a next-generation ribose modification, 2\u2032-O-[2-(methylamino)-2-oxoethyl] (NMA), which enhances the pharmacological properties of SSOs. We identified a long-lasting NMA-modified human candidate SSO, salanersen, that is three to four-fold more potent than nusinersen in human\u00a0SMN2 transgenic mice. To evaluate the generality of the NMA chemistry, we applied it to modulation of SCN1A exon 20N splicing, a therapeutic strategy for Dravet syndrome. An NMA-modified SSO is 3.5 -fold more potent than STK-001, a MOE-modified SSO currently in clinical trials. Our data establish the NMA chemistry as a broadly applicable ribose modification that markedly improves the pharmacological profile of SSOs, supporting its development as a next-generation platform for splicing modulation therapies.<\/jats:p>","DOI":"10.1093\/nar\/gkag484","type":"journal-article","created":{"date-parts":[[2026,5,2]],"date-time":"2026-05-02T12:27:00Z","timestamp":1777724820000},"source":"Crossref","is-referenced-by-count":5,"title":["Enhanced splicing modulation by NMA-modified antisense oligonucleotides"],"prefix":"10.1093","volume":"54","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5895-6684","authenticated-orcid":false,"given":"Karen","family":"Ling","sequence":"first","affiliation":[{"name":"Ionis Pharmaceuticals Inc. , 2855 Gazelle Ct., Carlsbad, CA 92010 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7466-3312","authenticated-orcid":false,"given":"Thazha P","family":"Prakash","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals Inc. , 2855 Gazelle Ct., Carlsbad, CA 92010 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jinghua","family":"Yu","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals Inc. , 2855 Gazelle Ct., Carlsbad, CA 92010 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michaela","family":"Jackson","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals Inc. , 2855 Gazelle Ct., Carlsbad, CA 92010 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Seung J","family":"Chun","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals Inc. , 2855 Gazelle Ct., Carlsbad, CA 92010 ,","place":["United 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