{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,18]],"date-time":"2026-09-18T20:44:22Z","timestamp":1789764262452,"version":"4.0.1"},"reference-count":7,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,10,28]],"date-time":"2024-10-28T00:00:00Z","timestamp":1730073600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100017510","name":"UK Dementia Research Institute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100017510","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100018987","name":"Ministerul Cercet\u0103rii, Inov\u0103rii \u015fi Digitaliz\u0103rii","doi-asserted-by":"publisher","award":["PNRR-III-C9-2022-I8-66"],"award-info":[{"award-number":["PNRR-III-C9-2022-I8-66"]}],"id":[{"id":"10.13039\/100018987","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100018987","name":"Ministerul Cercet\u0103rii, Inov\u0103rii \u015fi Digitaliz\u0103rii","doi-asserted-by":"publisher","award":["760114"],"award-info":[{"award-number":["760114"]}],"id":[{"id":"10.13039\/100018987","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002283","name":"Alzheimer\u2019s Research UK","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100002283","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000320","name":"Alzheimer's Society","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000320","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Nucleic Acids"],"published-print":{"date-parts":[[2024,12]]},"DOI":"10.1016\/j.omtn.2024.102377","type":"journal-article","created":{"date-parts":[[2024,11,14]],"date-time":"2024-11-14T09:41:16Z","timestamp":1731577276000},"page":"102377","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":6,"title":["CRISPR-Cas9-directed gene therapy for spinocerebellar ataxia type 1"],"prefix":"10.1016","volume":"35","author":[{"given":"Mihai","family":"Micl\u0103u\u0219","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2872-4468","authenticated-orcid":false,"given":"Gabriel","family":"Balmus","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2024.102377_bib1","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.123193","article-title":"Antisense oligonucleotide\u2013mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles","volume":"3","author":"Friedrich","year":"2018","journal-title":"JCI Insight"},{"key":"10.1016\/j.omtn.2024.102377_bib2","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1038\/d41586-020-00655-8","article-title":"CRISPR treatment inserted directly into the body for first time","volume":"579","author":"Ledford","year":"2020","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2024.102377_bib3","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtn.2024.102317","article-title":"Cas9 Editing of ATXN1 in Spinocerebellar Ataxia type 1 Mice and human iPSC-derived neurons","volume":"35","author":"Fagan","year":"2024","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2024.102377_bib4","doi-asserted-by":"crossref","first-page":"1235","DOI":"10.1016\/j.neuron.2018.02.013","article-title":"ATXN1-CIC Complex Is the Primary Driver of Cerebellar Pathology in Spinocerebellar Ataxia Type 1 through a Gain-of-Function Mechanism","volume":"97","author":"Rousseaux","year":"2018","journal-title":"Neuron"},{"key":"10.1016\/j.omtn.2024.102377_bib5","doi-asserted-by":"crossref","first-page":"915","DOI":"10.1016\/j.neuron.2023.02.030","article-title":"Disruption of the ATXN1-CIC complex reveals the role of additional nuclear ATXN1 interactors in spinocerebellar ataxia type 1","volume":"111","author":"Coffin","year":"2023","journal-title":"Neuron"},{"key":"10.1016\/j.omtn.2024.102377_bib6","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1016\/j.neuron.2022.11.017","article-title":"Decreasing mutant ATXN1 nuclear localization improves a spectrum of SCA1-like phenotypes and brain region transcriptomic profiles","volume":"111","author":"Handler","year":"2023","journal-title":"Neuron"},{"key":"10.1016\/j.omtn.2024.102377_bib7","doi-asserted-by":"crossref","first-page":"1159","DOI":"10.1016\/j.cell.2019.07.043","article-title":"Loss of Ataxin-1 Potentiates Alzheimer\u2019s Pathogenesis by Elevating Cerebral BACE1 Transcription","volume":"178","author":"Suh","year":"2019","journal-title":"Cell"}],"container-title":["Molecular Therapy Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253124002646?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253124002646?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,5,15]],"date-time":"2026-05-15T09:56:10Z","timestamp":1778838970000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253124002646"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,12]]},"references-count":7,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2024,12]]}},"alternative-id":["S2162253124002646"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2024.102377","relation":{},"ISSN":["2162-2531"],"issn-type":[{"value":"2162-2531","type":"print"}],"subject":[],"published":{"date-parts":[[2024,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"CRISPR-Cas9-directed gene therapy for spinocerebellar ataxia type 1","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2024.102377","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"simple-article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 The Author(s). Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"102377"}}