{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T07:48:06Z","timestamp":1790840886063,"version":"4.1.0"},"reference-count":17,"publisher":"EDP Sciences","issue":"2","license":[{"start":{"date-parts":[[2023,2,17]],"date-time":"2023-02-17T00:00:00Z","timestamp":1676592000000},"content-version":"vor","delay-in-days":16,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Med Sci (Paris)"],"published-print":{"date-parts":[[2023,2]]},"abstract":"<jats:p>Les oligonucl\u00e9otides sont des petits acides nucl\u00e9iques de synth\u00e8se capables de moduler l\u2019expression de g\u00e8nes cibles et leurs transcrits. Largement utilis\u00e9s par les chercheurs comme outils de recherche pour moduler l\u2019expression des g\u00e8nes dont ils cherchent \u00e0 d\u00e9crypter les fonctions, les oligonucl\u00e9otides peuvent \u00e9galement servir d\u2019agents th\u00e9rapeutiques pour r\u00e9guler des cibles d\u2019int\u00e9r\u00eat. Apr\u00e8s l\u2019arriv\u00e9e sur le march\u00e9 du premier oligonucl\u00e9otide th\u00e9rapeutique en 1998, le domaine a connu peu de succ\u00e8s cliniques jusqu\u2019en 2016, date \u00e0 laquelle le Spinraza\u00ae devient le premier m\u00e9dicament autoris\u00e9 pour le traitement de l\u2019amyotrophie spinale. Il deviendra dans les ann\u00e9es suivantes le premier \u00ab <jats:italic>blockbuster<\/jats:italic> \u00bb<jats:sup>1<\/jats:sup> de cette classe de mol\u00e9cules. Depuis lors, une dizaine d\u2019oligonucl\u00e9otides ont re\u00e7u des autorisations de mise sur le march\u00e9 (AMM), et de nombreux autres font actuellement l\u2019objet d\u2019un d\u00e9veloppement clinique. Dans cet article, nous d\u00e9crivons diff\u00e9rents oligonucl\u00e9otides th\u00e9rapeutiques, ainsi que leurs modes d\u2019action et leur brevetabilit\u00e9.<\/jats:p>","DOI":"10.1051\/medsci\/2023005","type":"journal-article","created":{"date-parts":[[2023,2,17]],"date-time":"2023-02-17T09:03:35Z","timestamp":1676624615000},"page":"111-118","source":"Crossref","is-referenced-by-count":2,"title":["M\u00e9canismes d\u2019action et brevetabilit\u00e9 des oligonucl\u00e9otides th\u00e9rapeutiques"],"prefix":"10.1051","volume":"39","author":[{"given":"Nicolas","family":"Crouvezier","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne-C\u00e9line","family":"Marie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lara","family":"Moumn\u00e9","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"250","published-online":{"date-parts":[[2023,2,17]]},"reference":[{"key":"R1","doi-asserted-by":"crossref","first-page":"714","DOI":"10.1016\/j.cmet.2018.03.004","volume":"27","author":"Crook","year":"2018","journal-title":"Cell Metab"},{"key":"R2","doi-asserted-by":"crossref","first-page":"17337","DOI":"10.1073\/pnas.0607015103","volume":"103","author":"Li","year":"2006","journal-title":"Proc Natl Acad Sci USA"},{"key":"R3","doi-asserted-by":"crossref","first-page":"260","DOI":"10.3390\/pharmaceutics14020260","volume":"14","author":"Moumn\u00e9","year":"2022","journal-title":"Pharmaceutics"},{"key":"R4","doi-asserted-by":"crossref","first-page":"2075","DOI":"10.1016\/j.ymthe.2017.06.002","volume":"25","author":"Liang","year":"2017","journal-title":"Mol Ther"},{"key":"R5","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1146\/annurev-med-041217-010829","volume":"70","author":"Bennett","year":"2019","journal-title":"Annu Rev Med"},{"key":"R6","doi-asserted-by":"crossref","first-page":"806","DOI":"10.1038\/35888","volume":"391","author":"Fire","year":"1998","journal-title":"Nature"},{"key":"R7","doi-asserted-by":"crossref","first-page":"e252","DOI":"10.1038\/mtna.2015.23","volume":"4","author":"Lam","year":"2015","journal-title":"Mol Ther Nucleic Acids"},{"key":"R8","doi-asserted-by":"crossref","first-page":"494","DOI":"10.1038\/35078107","volume":"411","author":"Elbashir","year":"2001","journal-title":"Nature"},{"key":"R9","doi-asserted-by":"crossref","first-page":"1625","DOI":"10.1007\/s40265-018-0983-6","volume":"78","author":"Hoy","year":"2018","journal-title":"Drugs"},{"key":"R10","doi-asserted-by":"crossref","first-page":"101","DOI":"10.1038\/s41392-020-0207-x","volume":"5","author":"Hu","year":"2020","journal-title":"Signal Transduct Target Ther"},{"key":"R11","doi-asserted-by":"crossref","first-page":"673","DOI":"10.1038\/s41573-020-0075-7","volume":"19","author":"Roberts","year":"2020","journal-title":"Nat Rev Drug Discov"},{"key":"R12","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1089\/nat.2018.0736","volume":"28","author":"Springer","year":"2018","journal-title":"Nucleic Acid Ther"},{"key":"R13","doi-asserted-by":"crossref","first-page":"232","DOI":"10.31662\/jmaj.2021-0019","volume":"4","author":"Matsuo","year":"2021","journal-title":"JMA J"},{"key":"R14","doi-asserted-by":"crossref","first-page":"785","DOI":"10.1038\/s41591-021-01297-7","volume":"27","author":"Cideciyan","year":"2021","journal-title":"Nat Med"},{"key":"R15","doi-asserted-by":"crossref","first-page":"1359","DOI":"10.1126\/science.354.6318.1359","volume":"354","author":"Wadman","year":"2016","journal-title":"Science"},{"key":"R16","doi-asserted-by":"crossref","first-page":"216","DOI":"10.1002\/hep.26669","volume":"59","author":"Reebye","year":"2014","journal-title":"Hepatology"},{"key":"R17","doi-asserted-by":"crossref","first-page":"3936","DOI":"10.1158\/1078-0432.CCR-20-0414","volume":"26","author":"Sarker","year":"2020","journal-title":"Clin Cancer Res"}],"container-title":["m\u00e9decine\/sciences"],"original-title":[],"link":[{"URL":"https:\/\/www.medecinesciences.org\/10.1051\/medsci\/2023005\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,2,17]],"date-time":"2023-02-17T09:04:40Z","timestamp":1676624680000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.medecinesciences.org\/10.1051\/medsci\/2023005"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,2]]},"references-count":17,"journal-issue":{"issue":"2"},"alternative-id":["msc220265"],"URL":"https:\/\/doi.org\/10.1051\/medsci\/2023005","relation":{},"ISSN":["0767-0974","1958-5381"],"issn-type":[{"value":"0767-0974","type":"print"},{"value":"1958-5381","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,2]]}}}