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These antisense therapies rely on modified nucleotides sequences (single-strand DNA or RNA, both belonging to the antisense oligonucleotides family, or double-strand interfering RNA) to act specifically on pathogenic target nucleic acids, thanks to complementary base pairing. Depending on the type of molecule, chemical modifications and target, base pairing will lead alternatively to splicing modifications of primary transcript RNA or transient messenger RNA degradation or non-translation. The key to success for neurodegenerative diseases also depends on the ability to reach target cells. The most advanced antisense therapies under development in neurological disorders are presented here, at the clinical stage of development, either at phase 3 or market authorization stage, such as in spinal amyotrophy, Duchenne muscular dystrophy, transthyretin-related hereditary amyloidosis, porphyria and amyotrophic lateral sclerosis; or in earlier clinical phase 1 B, for Huntington's disease, synucleinopathies and tauopathies. We also discuss antisense therapies at the preclinical stage, such as in some tauopathies, spinocerebellar ataxias or other rare neurological disorders. Each subtype of antisense therapy, antisense oligonucleotides or interfering RNA, has proved target engagement or even clinical efficacy in patients; undisputable recent advances for severe and previously untreatable neurological disorders. Antisense therapies show great promise, but many unknowns remain. Expanding the initial successes achieved in orphan or rare diseases to other disorders will be the next challenge, as shown by the recent failure in Huntington disease or due to long-term preclinical toxicity in multiple system atrophy and cystic fibrosis. This will be critical in the perspective of new planned applications to premanifest mutation carriers, or other non-genetic degenerative disorders such as multiple system atrophy or Parkinson disease.<\/jats:p>","DOI":"10.1093\/brain\/awab423","type":"journal-article","created":{"date-parts":[[2021,11,17]],"date-time":"2021-11-17T15:18:54Z","timestamp":1637162334000},"page":"816-831","source":"Crossref","is-referenced-by-count":31,"title":["Antisense therapies in neurological diseases"],"prefix":"10.1093","volume":"145","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9615-0788","authenticated-orcid":false,"given":"Jean-Baptiste","family":"Brunet de Courssou","sequence":"first","affiliation":[{"name":"Assistance Publique H\u00f4pitaux de Paris, Department of Neurology, CIC Neurosciences, Piti\u00e9-Salp\u00eatri\u00e8re Hospital, Sorbonne University, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra","family":"Durr","sequence":"additional","affiliation":[{"name":"Sorbonne University, Paris Brain Institute \u2013 ICM, Inserm, CNRS, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Adams","sequence":"additional","affiliation":[{"name":"Department of Neurology, Bic\u00eatre hospital, Assistance Publique H\u00f4pitaux de Paris, Centre de R\u00e9f\u00e9rence National des Neuropathies P\u00e9riph\u00e9riques Rares, Paris Saclay University, INSERM U 1195, Le Kremlin Bic\u00eatre, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Christophe","family":"Corvol","sequence":"additional","affiliation":[{"name":"Assistance Publique H\u00f4pitaux de Paris, Department of Neurology, CIC Neurosciences, Piti\u00e9-Salp\u00eatri\u00e8re Hospital, Sorbonne University, Paris, France"},{"name":"Sorbonne University, Paris Brain Institute \u2013 ICM, Inserm, CNRS, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2365-1223","authenticated-orcid":false,"given":"Louise-Laure","family":"Mariani","sequence":"additional","affiliation":[{"name":"Assistance Publique H\u00f4pitaux de Paris, Department of Neurology, CIC Neurosciences, Piti\u00e9-Salp\u00eatri\u00e8re Hospital, Sorbonne University, Paris, France"},{"name":"Sorbonne University, Paris Brain Institute \u2013 ICM, Inserm, CNRS, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2021,11,23]]},"reference":[{"issue":"2","key":"2022042902214093800_awab423-B1","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1016\/j.amjmed.2018.07.041","article-title":"Introduction to neurogenetics","volume":"132","author":"Vgontzas","year":"2019","journal-title":"Am J Med."},{"key":"2022042902214093800_awab423-B2","first-page":"e101112","article-title":"RNA toxicity in non-coding repeat expansion disorders","volume":"13","author":"Swinnen","year":"2019","journal-title":"EMBO J."},{"issue":"5","key":"2022042902214093800_awab423-B3","doi-asserted-by":"crossref","first-page":"542","DOI":"10.1038\/s41556-019-0311-8","article-title":"Cellular functions of long noncoding RNAs","volume":"21","author":"Yao","year":"2019","journal-title":"Nat Cell Biol."},{"key":"2022042902214093800_awab423-B4","doi-asserted-by":"crossref","first-page":"101746","DOI":"10.1016\/j.pneurobio.2020.101746","article-title":"Role of circular RNAs in brain development and CNS diseases","volume":"186","author":"Mehta","year":"2020","journal-title":"Prog Neurobiol."},{"key":"2022042902214093800_awab423-B5","doi-asserted-by":"crossref","first-page":"1033","DOI":"10.3389\/fgene.2019.01033","article-title":"The role of non-coding RNAs in neurodevelopmental disorders","volume":"10","author":"Zhang","year":"2019","journal-title":"Front Genet."},{"issue":"1","key":"2022042902214093800_awab423-B6","doi-asserted-by":"crossref","first-page":"1409","DOI":"10.1038\/s41598-018-37872-1","article-title":"Long non-coding RNA repertoire and open chromatin regions constitute midbrain dopaminergic neuron-specific molecular signatures","volume":"9","author":"Gendron","year":"2019","journal-title":"Sci Rep."},{"issue":"4","key":"2022042902214093800_awab423-B7","doi-asserted-by":"crossref","first-page":"445","DOI":"10.1038\/s41431-017-0076-0","article-title":"Human germline gene editing: Recommendations of ESHG and ESHRE","volume":"26","author":"de Wert","year":"2018","journal-title":"Eur J Hum Genet."},{"issue":"12","key":"2022042902214093800_awab423-B8","doi-asserted-by":"crossref","first-page":"1479","DOI":"10.1038\/s41556-019-0424-0","article-title":"Human germline genome editing","volume":"21","author":"A. 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