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Therapeutic antisense oligonucleotides can modulate target gene expression with high specificity through various mechanisms determined by base sequences and chemical modifications; and have shown efficacy in clinical trials for a few rare neurological conditions. Therefore, this review will focus on the applications of antisense oligonucleotides, in particular splice-switching antisense oligomers as promising therapeutics for rare neurological diseases, with key examples of Duchenne muscular dystrophy and spinal muscular atrophy. Challenges and future perspectives in developing antisense therapeutics for rare conditions including target discovery, antisense chemical modifications, animal models for therapeutic validations, and clinical trial designs will also be briefly discussed.<\/jats:p>","DOI":"10.3389\/fnins.2024.1414658","type":"journal-article","created":{"date-parts":[[2024,9,23]],"date-time":"2024-09-23T04:10:16Z","timestamp":1727064616000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":37,"title":["Antisense oligonucleotides and their applications in rare neurological diseases"],"prefix":"10.3389","volume":"18","author":[{"given":"Simon","family":"McDowall","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"May","family":"Aung-Htut","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steve","family":"Wilton","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dunhui","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2024,9,23]]},"reference":[{"key":"B1","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":"","journal-title":"Nucleic Acid Ther."},{"key":"B2","doi-asserted-by":"publisher","first-page":"446","DOI":"10.1261\/rna.079540.122","article-title":"Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: Development, regulation, and implementation considerations.","volume":"29","author":"Aartsma-Rus","year":"","journal-title":"RNA"},{"key":"B3","doi-asserted-by":"publisher","first-page":"145","DOI":"10.1136\/jmedgenet-2015-103387","article-title":"The importance of genetic diagnosis for Duchenne muscular dystrophy.","volume":"53","author":"Aartsma-Rus","year":"2016","journal-title":"J. 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