{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T21:55:59Z","timestamp":1790805359606,"version":"4.1.0"},"reference-count":57,"publisher":"American Society for Clinical Investigation","issue":"2","license":[{"start":{"date-parts":[[2024,1,16]],"date-time":"2024-01-16T00:00:00Z","timestamp":1705363200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001665","name":"French National Research Agency","doi-asserted-by":"crossref","award":["ANR-17-CE18-0014"],"award-info":[{"award-number":["ANR-17-CE18-0014"]}],"id":[{"id":"10.13039\/501100001665","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100001665","name":"French National Research Agency","doi-asserted-by":"crossref","award":["ANR 22 CE17 0031"],"award-info":[{"award-number":["ANR 22 CE17 0031"]}],"id":[{"id":"10.13039\/501100001665","id-type":"DOI","asserted-by":"crossref"}]},{"name":"CALMIP center","award":["Grant 2023-p22027"],"award-info":[{"award-number":["Grant 2023-p22027"]}]},{"DOI":"10.13039\/501100001665","name":"French National Research Agency","doi-asserted-by":"crossref","award":["ANR-21-PRRD-0002-01"],"award-info":[{"award-number":["ANR-21-PRRD-0002-01"]}],"id":[{"id":"10.13039\/501100001665","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2024,1,16]]},"DOI":"10.1172\/jci172018","type":"journal-article","created":{"date-parts":[[2023,11,28]],"date-time":"2023-11-28T17:01:47Z","timestamp":1701190907000},"source":"Crossref","is-referenced-by-count":16,"title":["A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III"],"prefix":"10.1172","volume":"134","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1770-4368","authenticated-orcid":false,"given":"Antoine","family":"Gardin","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J\u00e9r\u00e9my","family":"Rouillon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7183-3710","authenticated-orcid":false,"given":"Valle","family":"Montalvo-Romeral","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lucille","family":"Rossiaud","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrice","family":"Vidal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9260-8296","authenticated-orcid":false,"given":"Romain","family":"Launay","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-9571-2902","authenticated-orcid":false,"given":"Mallaury","family":"Vie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Youssef","family":"Krimi Benchekroun","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J\u00e9r\u00e9mie","family":"Cosette","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B\u00e9rang\u00e8re","family":"Bertin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3004-7075","authenticated-orcid":false,"given":"Tiziana","family":"La Bella","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guillaume","family":"Dubreuil","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Justine","family":"Nozi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9474-6377","authenticated-orcid":false,"given":"Louisa","family":"Jauze","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Romain","family":"Fragnoud","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathalie","family":"Daniele","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laetitia","family":"Van Wittenberghe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5786-848X","authenticated-orcid":false,"given":"J\u00e9r\u00e9my","family":"Esque","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6280-4109","authenticated-orcid":false,"given":"Isabelle","family":"Andr\u00e9","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xavier","family":"Nissan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lucile","family":"Hoch","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9184-0210","authenticated-orcid":false,"given":"Giuseppe","family":"Ronzitti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"232","published-online":{"date-parts":[[2024,1,16]]},"reference":[{"key":"B1"},{"key":"B2","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-016-9932-2"},{"key":"B3","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgmr.2021.100821"},{"key":"B4","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12355"},{"key":"B5","doi-asserted-by":"publisher","DOI":"10.1186\/s40478-019-0815-2"},{"key":"B6","doi-asserted-by":"publisher","DOI":"10.1038\/s41436-019-0561-7"},{"key":"B7","doi-asserted-by":"publisher","DOI":"10.1097\/GIM.0b013e3181e655b6"},{"key":"B8","doi-asserted-by":"publisher","DOI":"10.1038\/s41573-019-0012-9"},{"key":"B9","doi-asserted-by":"publisher","DOI":"10.1016\/j.jhep.2021.12.015"},{"key":"B10","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"B11","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2019000811"},{"key":"B12","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2113708"},{"key":"B13","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)31868-8"},{"key":"B14","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2019.102"},{"key":"B15","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2017.12.019"},{"key":"B16","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2022.08.004"},{"key":"B17","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aav4523"},{"key":"B18","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2018.11.002"},{"issue":"4s1","key":"B19","article-title":"Autophagy induction improves dual AAV vector efficacy in a mouse model of glycogen storage disease type III [Poster ASGCT 2023]","volume":"31","author":"Jauze","year":"2023","journal-title":"Mol Ther"},{"key":"B20","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2020.05.034"},{"key":"B21","doi-asserted-by":"publisher","DOI":"10.1172\/jci.insight.152970"},{"key":"B22","doi-asserted-by":"publisher","DOI":"10.1016\/j.jhep.2020.11.001"},{"key":"B23","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2020.182"},{"key":"B24","article-title":"Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa\u2212\/\u2212 mice","author":"Sellier","journal-title":"J Inherit Metab Dis"},{"key":"B25","doi-asserted-by":"publisher","DOI":"10.32607\/actanaturae.11063"},{"key":"B26","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2008.104"},{"key":"B27","doi-asserted-by":"publisher","DOI":"10.1038\/mtm.2016.49"},{"key":"B28","doi-asserted-by":"publisher","DOI":"10.1038\/ncomms11229"},{"key":"B29","doi-asserted-by":"publisher","DOI":"10.1038\/s41586-021-03819-2"},{"key":"B30","doi-asserted-by":"publisher","DOI":"10.1016\/j.jmb.2021.167336"},{"key":"B31","doi-asserted-by":"publisher","DOI":"10.1038\/jhg.2016.24"},{"key":"B32","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2017.08.010"},{"key":"B33","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.61547"},{"key":"B34","doi-asserted-by":"publisher","DOI":"10.1006\/mgme.1999.2953"},{"key":"B35","doi-asserted-by":"publisher","DOI":"10.1002\/mgg3.1444"},{"key":"B36","doi-asserted-by":"publisher","DOI":"10.1038\/jhg.2013.117"},{"key":"B37","doi-asserted-by":"publisher","DOI":"10.1097\/CND.0000000000000212"},{"key":"B38","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0270373"},{"key":"B39","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddv214"},{"key":"B40","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2020.08.018"},{"key":"B41","doi-asserted-by":"publisher","DOI":"10.3233\/JND-221639"},{"key":"B42","doi-asserted-by":"publisher","DOI":"10.3389\/fcell.2023.1163427"},{"key":"B43","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2019.255"},{"key":"B44","doi-asserted-by":"publisher","DOI":"10.1038\/ncomms16105"},{"key":"B45","doi-asserted-by":"publisher","DOI":"10.1002\/hep.30535"},{"key":"B46"},{"key":"B47","doi-asserted-by":"publisher","DOI":"10.1038\/nrd.2016.245"},{"key":"B48","unstructured":"Grunewald S, et al. mRNA-3927 therapy for propionic acidemia: interim data from a phase 1\/2 study. Paper presented at: the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting; May 16\u201320, 2023; Los Angeles, California, USA. https:\/\/s29.q4cdn.com\/435878511\/files\/doc_presentations\/2023\/May\/18\/mrna-3927-pa-p101-ia-oral_asgct2023_final.pdf Accessed November 13, 2023"},{"key":"B49","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2018.12.011"},{"key":"B50","unstructured":"Mingozzi F, et al., inventor; Cabinet Becker et Associes, assignee. Treatment of glycogen storage disease type III. International Publication No. WO2018162748A1. September 13, 2018"},{"key":"B51","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-017-17204-5"},{"key":"B52","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aam6375"},{"key":"B53","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2011.063"},{"key":"B54","doi-asserted-by":"publisher","DOI":"10.1038\/nmeth.2019"},{"key":"B55","doi-asserted-by":"publisher","DOI":"10.1016\/j.softx.2015.06.001"},{"key":"B56","doi-asserted-by":"publisher","DOI":"10.1002\/jcc.23354"},{"key":"B57","doi-asserted-by":"publisher","DOI":"10.1002\/jcc.21787"}],"container-title":["Journal of Clinical Investigation"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.jci.org\/articles\/view\/172018\/files\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,15]],"date-time":"2024-01-15T19:04:14Z","timestamp":1705345454000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.jci.org\/articles\/view\/172018"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,1,16]]},"references-count":57,"journal-issue":{"issue":"2","published-online":{"date-parts":[[2024,1,16]]},"published-print":{"date-parts":[[2024,1,16]]}},"alternative-id":["10.1172\/JCI172018"],"URL":"https:\/\/doi.org\/10.1172\/jci172018","relation":{},"ISSN":["1558-8238"],"issn-type":[{"value":"1558-8238","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,1,16]]},"article-number":"e172018"}}