{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T03:05:54Z","timestamp":1791947154720,"version":"4.3.4"},"reference-count":81,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2022,9,3]],"date-time":"2022-09-03T00:00:00Z","timestamp":1662163200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004923","name":"French Muscular Dystrophy Association","doi-asserted-by":"publisher","award":["Strategic pole MNH Decrypt"],"award-info":[{"award-number":["Strategic pole MNH Decrypt"]}],"id":[{"id":"10.13039\/501100004923","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001677","name":"Institut National de la Sant\u00e9 et de la Recherche M\u00e9dicale","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001677","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100001819","name":"International Rett Syndrome Foundation","doi-asserted-by":"publisher","award":["IRSF"],"award-info":[{"award-number":["IRSF"]}],"id":[{"id":"10.13039\/100001819","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Develop Med Child Neuro"],"published-print":{"date-parts":[[2023,2]]},"abstract":"<jats:sec>\n                    <jats:label\/>\n                    <jats:p>\n                      Rett syndrome (RTT) is an X\u2010linked neurogenetic disorder caused by mutations of the\n                      <jats:italic>MECP2<\/jats:italic>\n                      (methyl\u2010CpG\u2010binding protein 2) gene. Over two decades of work established MeCP2 as a protein with pivotal roles in the regulation of the epigenome, neuronal physiology, synaptic maintenance, and behaviour. Given the genetic aetiology of RTT and the proof of concept of its reversal in a mouse model, considerable efforts have been made to design therapeutic approaches to re\u2010express MeCP2. By being at the forefront of the development of innovative gene therapies, research on RTT is of paramount importance for the treatment of monogenic neurological diseases. Here we discuss the recent advances and challenges of promising genetic strategies for the treatment of RTT including gene replacement therapies, gene\/RNA editing strategies, and reactivation of the silenced X chromosome.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>What this paper adds<\/jats:title>\n                    <jats:p>\n                      <jats:list list-type=\"bullet\">\n                        <jats:list-item>\n                          <jats:p>Recent advances shed light on the promises of gene replacement therapy with new vectors designed to control the levels of MeCP2 expression.<\/jats:p>\n                        <\/jats:list-item>\n                        <jats:list-item>\n                          <jats:p>New developments in DNA\/RNA editing approaches or reactivation of the silenced X chromosome open the possibility to re\u2010express the native MeCP2 locus at endogenous levels.<\/jats:p>\n                        <\/jats:list-item>\n                        <jats:list-item>\n                          <jats:p>Current strategies still face limitations in transduction efficiency and future work is needed to improve brain delivery.<\/jats:p>\n                        <\/jats:list-item>\n                      <\/jats:list>\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/dmcn.15383","type":"journal-article","created":{"date-parts":[[2022,9,3]],"date-time":"2022-09-03T07:48:20Z","timestamp":1662191300000},"page":"162-170","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":38,"title":["State\u2010of\u2010the\u2010art therapies for Rett syndrome"],"prefix":"10.1111","volume":"65","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6204-6771","authenticated-orcid":false,"given":"Nicolas","family":"Panayotis","sequence":"first","affiliation":[{"name":"Department of Biomolecular Sciences Weizmann Institute of Science  Rehovot Israel"},{"name":"Department of Molecular Neuroscience Weizmann Institute of Science  Rehovot Israel"},{"name":"Universit\u00e9 Paris Cit\u00e9, CNRS, Saints\u2010P\u00e8res Paris Institute for the Neurosciences  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yann","family":"Ehinger","sequence":"additional","affiliation":[{"name":"Department of Neurology University of California San Francisco  San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie Solenne","family":"Felix","sequence":"additional","affiliation":[{"name":"MMG, INSERM Aix Marseille University  Marseille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9721-8844","authenticated-orcid":false,"given":"Jean\u2010Christophe","family":"Roux","sequence":"additional","affiliation":[{"name":"MMG, INSERM Aix Marseille University  Marseille 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