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This changed recently when antisense oligonucleotides (ASOs) could be translated from in vitro and experimental animal studies into clinical practice. In 2016, two ASOs were approved by the United States US Food and Drug Administration (FDA) and demonstrated remarkable efficacy in Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). ASOs are synthetic single-stranded strings of nucleic acids. They selectively bind to specific premessenger ribonucleic acid (pre-mRNA)\/mRNA sequences and alter protein synthesis by several mechanisms of action. Thus, apart from gene replacement, ASOs may therefore provide the most direct therapeutic strategy for influencing gene expression. In this review, we shall discuss basic mechanisms of ASO action, the role of chemical modifications needed to improve the pharmacodynamic and pharmacokinetic properties of ASOs, and we shall then focus on several ASOs developed for the treatment of neurodegenerative and neuromuscular disorders, including SMA, DMD, myotonic dystrophies, Huntington\u2019s disease, amyotrophic lateral sclerosis and Alzheimer\u2019s disease.<\/jats:p>","DOI":"10.1177\/1756286418776932","type":"journal-article","created":{"date-parts":[[2018,5,24]],"date-time":"2018-05-24T02:02:12Z","timestamp":1527127332000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":118,"title":["Antisense oligonucleotides in neurological disorders"],"prefix":"10.1177","volume":"11","author":[{"given":"Claudia D.","family":"Wurster","sequence":"first","affiliation":[{"name":"Department of Neurology, University of Ulm, Oberer Eselsberg 45, Ulm, 89081, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Albert C.","family":"Ludolph","sequence":"additional","affiliation":[{"name":"Department of Neurology, Ulm University, Ulm, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2018,5,23]]},"reference":[{"key":"bibr1-1756286418776932","doi-asserted-by":"publisher","DOI":"10.1016\/j.addr.2015.03.008"},{"key":"bibr2-1756286418776932","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-pharmtox-010716-104846"},{"key":"bibr3-1756286418776932","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2015.070"},{"key":"bibr4-1756286418776932","first-page":"33","volume-title":"Die histochemischen und physiologischen arbeiten von Friedrich Miescher - aus dem wissenschaftlichen Briefwechsel von F. 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