{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T12:52:55Z","timestamp":1792759975633,"version":"4.5.0"},"reference-count":100,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2024,7,25]],"date-time":"2024-07-25T00:00:00Z","timestamp":1721865600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Mol. Neurosci."],"abstract":"<jats:p>Pediatric neurological disorders are frequently devastating and present unmet needs for effective medicine. The successful treatment of spinal muscular atrophy with splice-switching antisense oligonucleotides (SSO) indicates a feasible path to targeting neurological disorders by redirecting pre-mRNA splicing. One direct outcome is the development of SSOs to treat haploinsufficient disorders by targeting naturally occurring non-productive splice isoforms. The development of personalized SSO treatment further inspired the therapeutic exploration of rare diseases. This review will discuss the recent advances that utilize SSOs to treat pediatric neurological disorders.<\/jats:p>","DOI":"10.3389\/fnmol.2024.1412964","type":"journal-article","created":{"date-parts":[[2024,7,25]],"date-time":"2024-07-25T06:02:02Z","timestamp":1721887322000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":9,"title":["Splice-switching antisense oligonucleotides for pediatric neurological disorders"],"prefix":"10.3389","volume":"17","author":[{"given":"Xiaochang","family":"Zhang","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2024,7,25]]},"reference":[{"key":"ref1","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1089\/nat.2022.0060","article-title":"Consensus guidelines for the design and in vitro preclinical efficacy testing N-of-1 exon skipping antisense oligonucleotides","volume":"33","author":"Aartsma-Rus","year":"2023","journal-title":"Nucleic Acid Ther."},{"key":"ref2","doi-asserted-by":"publisher","first-page":"414","DOI":"10.1016\/j.cell.2019.01.013","article-title":"The splicing code Goes deep","volume":"176","author":"Bao","year":"2019","journal-title":"Cell"},{"key":"ref3","doi-asserted-by":"publisher","first-page":"53","DOI":"10.1038\/nature09000","article-title":"Deciphering the splicing code","volume":"465","author":"Barash","year":"2010","journal-title":"Nature"},{"key":"ref4","doi-asserted-by":"publisher","first-page":"1587","DOI":"10.1126\/science.1230612","article-title":"The evolutionary landscape of alternative splicing in vertebrate species","volume":"338","author":"Barbosa-Morais","year":"2012","journal-title":"Science"},{"key":"ref5","doi-asserted-by":"publisher","first-page":"3171","DOI":"10.1073\/pnas.74.8.3171","article-title":"Spliced segments at the 5\u2032 terminus of adenovirus 2 late mRNA","volume":"74","author":"Berget","year":"1977","journal-title":"Proc. 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