{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,3]],"date-time":"2026-11-03T00:20:29Z","timestamp":1793665229274,"version":"4.5.1"},"reference-count":41,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"funder":[{"DOI":"10.13039\/501100001674","name":"Fondation Leducq","doi-asserted-by":"publisher","award":["2023_Leducq-foundation_International-networks-excellence-program"],"award-info":[{"award-number":["2023_Leducq-foundation_International-networks-excellence-program"]}],"id":[{"id":"10.13039\/501100001674","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2026,4]]},"DOI":"10.1016\/j.ymthe.2025.12.042","type":"journal-article","created":{"date-parts":[[2025,12,24]],"date-time":"2025-12-24T08:04:10Z","timestamp":1766563450000},"page":"2012-2027","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":7,"title":["Transgene-induced cardiotoxicity in high-dose AAV gene transfer"],"prefix":"10.1016","volume":"34","author":[{"given":"Ariane","family":"Biquand","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Evelyne","family":"Gicquel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerome","family":"Poupiot","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marine","family":"Faivre","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephany","family":"Campuzano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tiffany","family":"Bourgeton","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre-Romain","family":"Le Brun","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philippe","family":"Veron","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anthony","family":"Brureau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6505-446X","authenticated-orcid":false,"given":"Isabelle","family":"Richard","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2025.12.042_bib1","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1016\/j.jpeds.2020.11.054","article-title":"Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series","volume":"231","author":"Chand","year":"2021","journal-title":"J. Pediatr."},{"key":"10.1016\/j.ymthe.2025.12.042_bib2","doi-asserted-by":"crossref","first-page":"1550","DOI":"10.1001\/jama.2015.3253","article-title":"Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome","volume":"313","author":"Hacein-Bey Abina","year":"2015","journal-title":"JAMA"},{"key":"10.1016\/j.ymthe.2025.12.042_bib3","doi-asserted-by":"crossref","first-page":"327","DOI":"10.3233\/JND-221639","article-title":"Meeting Report: 2022 Muscular Dystrophy Association Summit on \u2018Safety and Challenges in Gene Transfer Therapy\u2019","volume":"10","author":"Lek","year":"2023","journal-title":"J. Neuromuscul. Dis."},{"key":"10.1016\/j.ymthe.2025.12.042_bib4","doi-asserted-by":"crossref","first-page":"1203","DOI":"10.1056\/NEJMoa2307798","article-title":"Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy","volume":"389","author":"Lek","year":"2023","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.ymthe.2025.12.042_bib5","doi-asserted-by":"crossref","first-page":"592","DOI":"10.1038\/s41434-022-00341-6","article-title":"Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study","volume":"30","author":"Bitetti","year":"2023","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2025.12.042_bib6","doi-asserted-by":"crossref","first-page":"346","DOI":"10.1007\/s11897-022-00569-8","article-title":"Biomarkers for Myocarditis and Inflammatory Cardiomyopathy","volume":"19","author":"Suresh","year":"2022","journal-title":"Curr. Heart Fail. Rep."},{"key":"10.1016\/j.ymthe.2025.12.042_bib7","doi-asserted-by":"crossref","first-page":"1125","DOI":"10.1016\/S1474-4422(23)00313-7","article-title":"Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial","volume":"22","author":"Shieh","year":"2023","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.ymthe.2025.12.042_bib8","doi-asserted-by":"crossref","first-page":"955","DOI":"10.1002\/ana.26755","article-title":"Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged >\/=4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR)","volume":"94","author":"Zaidman","year":"2023","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymthe.2025.12.042_bib9","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aat6072","article-title":"Titin splicing regulates cardiotoxicity associated with calpain 3 gene therapy for limb-girdle muscular dystrophy type 2A","volume":"11","author":"Lostal","year":"2019","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2025.12.042_bib10","doi-asserted-by":"crossref","first-page":"2851","DOI":"10.1093\/hmg\/10.25.2851","article-title":"Mutations in the fukutin-related protein gene (FKRP) identify limb girdle muscular dystrophy 2I as a milder allelic variant of congenital muscular dystrophy MDC1C","volume":"10","author":"Brockington","year":"2001","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.12.042_bib11","doi-asserted-by":"crossref","DOI":"10.1242\/jcs.258684","article-title":"Titin M-line insertion sequence 7 is required for proper cardiac function in mice","volume":"134","author":"Biquand","year":"2021","journal-title":"J. Cell Sci."},{"key":"10.1016\/j.ymthe.2025.12.042_bib12","doi-asserted-by":"crossref","first-page":"850","DOI":"10.1177\/0192623311413788","article-title":"Morphologic aspects of rodent cardiotoxicity in a retrospective evaluation of National Toxicology Program studies","volume":"39","author":"Jokinen","year":"2011","journal-title":"Toxicol. Pathol."},{"key":"10.1016\/j.ymthe.2025.12.042_bib13","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1161\/01.RES.80.2.269","article-title":"Dystrophin is not a specific component of the cardiac costamere","volume":"80","author":"Stevenson","year":"1997","journal-title":"Circ. Res."},{"key":"10.1016\/j.ymthe.2025.12.042_bib14","doi-asserted-by":"crossref","DOI":"10.1111\/cpr.12562","article-title":"Syntrophins entangled in cytoskeletal meshwork: Helping to hold it all together","volume":"52","author":"Bhat","year":"2019","journal-title":"Cell Prolif."},{"key":"10.1016\/j.ymthe.2025.12.042_bib15","doi-asserted-by":"crossref","DOI":"10.1098\/rstb.2016.0525","article-title":"The heat-shock, or HSF1-mediated proteotoxic stress, response in cancer: from proteomic stability to oncogenesis","volume":"373","author":"Dai","year":"2018","journal-title":"Philos. Trans. R. Soc. Lond. B Biol. Sci."},{"key":"10.1016\/j.ymthe.2025.12.042_bib16","first-page":"1971","article-title":"Downregulation of GSDMD attenuates tumor proliferation via the intrinsic mitochondrial apoptotic pathway and inhibition of EGFR\/Akt signaling and predicts a good prognosis in non-small cell lung cancer","volume":"40","author":"Gao","year":"2018","journal-title":"Oncol. Rep."},{"key":"10.1016\/j.ymthe.2025.12.042_bib17","doi-asserted-by":"crossref","DOI":"10.3390\/cells10030515","article-title":"Heme Oxgenase-1, a Cardinal Modulator of Regulated Cell Death and Inflammation","volume":"10","author":"Ryter","year":"2021","journal-title":"Cells"},{"key":"10.1016\/j.ymthe.2025.12.042_bib18","first-page":"291","article-title":"BiP: Master Regulator of the Unfolded Protein Response and Crucial Factor in Flavivirus Biology Yale","volume":"90","author":"Lewy","year":"2017","journal-title":"J. Biol. Med."},{"key":"10.1016\/j.ymthe.2025.12.042_bib19","doi-asserted-by":"crossref","DOI":"10.1017\/S1462399409001203","article-title":"Sarcoglycanopathies: molecular pathogenesis and therapeutic prospects","volume":"11","author":"Sandona","year":"2009","journal-title":"Expert Rev. Mol. Med."},{"key":"10.1016\/j.ymthe.2025.12.042_bib20","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1038\/sj.cdd.4400476","article-title":"Emerging roles of caspase-3 in apoptosis","volume":"6","author":"Porter","year":"1999","journal-title":"Cell Death Differ."},{"key":"10.1016\/j.ymthe.2025.12.042_bib21","doi-asserted-by":"crossref","first-page":"1248","DOI":"10.1038\/ijo.2011.233","article-title":"Investigating the involvement of the ATF6alpha pathway of the unfolded protein response in adipogenesis","volume":"36","author":"Lowe","year":"2012","journal-title":"Int. J. Obes."},{"key":"10.1016\/j.ymthe.2025.12.042_bib22","doi-asserted-by":"crossref","first-page":"545","DOI":"10.1038\/nrm2938","article-title":"Heat shock factors: integrators of cell stress, development and lifespan","volume":"11","author":"Akerfelt","year":"2010","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.ymthe.2025.12.042_bib23","doi-asserted-by":"crossref","first-page":"12","DOI":"10.2183\/pjab.85.12","article-title":"The proteasome: overview of structure and functions","volume":"85","author":"Tanaka","year":"2009","journal-title":"Proc. Jpn. Acad. Ser. B Phys. Biol. Sci."},{"key":"10.1016\/j.ymthe.2025.12.042_bib24","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1016\/S0076-6879(08)03612-4","article-title":"Monitoring autophagic degradation of p62\/SQSTM1","volume":"452","author":"Bjorkoy","year":"2009","journal-title":"Methods Enzymol."},{"key":"10.1016\/j.ymthe.2025.12.042_bib25","doi-asserted-by":"crossref","first-page":"571","DOI":"10.1038\/cdd.2010.191","article-title":"The Beclin 1 network regulates autophagy and apoptosis","volume":"18","author":"Kang","year":"2011","journal-title":"Cell Death Differ."},{"key":"10.1016\/j.ymthe.2025.12.042_bib26","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2022.975803","article-title":"Immunogenicity and toxicity of AAV gene therapy","volume":"13","author":"Ertl","year":"2022","journal-title":"Front. Immunol."},{"key":"10.1016\/j.ymthe.2025.12.042_bib27","doi-asserted-by":"crossref","first-page":"808","DOI":"10.1089\/hum.2020.167","article-title":"Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology","volume":"31","author":"Hordeaux","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.12.042_bib28","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aba9188","article-title":"MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates","volume":"12","author":"Hordeaux","year":"2020","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2025.12.042_bib29","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.omtm.2018.06.004","article-title":"Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques","volume":"10","author":"Hordeaux","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.12.042_bib30","doi-asserted-by":"crossref","first-page":"1047","DOI":"10.1007\/s00428-007-0515-3","article-title":"Biochemical and ultrastructural evidence of endoplasmic reticulum stress in LGMD2I","volume":"451","author":"Boito","year":"2007","journal-title":"Virchows Arch."},{"key":"10.1016\/j.ymthe.2025.12.042_bib31","doi-asserted-by":"crossref","first-page":"519","DOI":"10.1111\/nan.12684","article-title":"Protein homeostasis in LGMDR9 (LGMD2I) - The role of ubiquitin-proteasome and autophagy-lysosomal system","volume":"47","author":"Franekova","year":"2021","journal-title":"Neuropathol. Appl. Neurobiol."},{"key":"10.1016\/j.ymthe.2025.12.042_bib32","doi-asserted-by":"crossref","first-page":"250","DOI":"10.3389\/fmicb.2014.00250","article-title":"Cellular unfolded protein response against viruses used in gene therapy","volume":"5","author":"Sen","year":"2014","journal-title":"Front. Microbiol."},{"key":"10.1016\/j.ymthe.2025.12.042_bib33","doi-asserted-by":"crossref","DOI":"10.1038\/mtm.2016.45","article-title":"100-fold but not 50-fold dystrophin overexpression aggravates electrocardiographic defects in the mdx model of Duchenne muscular dystrophy","volume":"3","author":"Yue","year":"2016","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.12.042_bib34","doi-asserted-by":"crossref","first-page":"725","DOI":"10.1038\/364725a0","article-title":"Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity","volume":"364","author":"Cox","year":"1993","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.12.042_bib35","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.165869","article-title":"Potential limitations of microdystrophin gene therapy for Duchenne muscular dystrophy","volume":"9","author":"Hart","year":"2024","journal-title":"JCI Insight"},{"key":"10.1016\/j.ymthe.2025.12.042_bib36","article-title":"Evaluation of the DBA\/2J mouse as a potential background strain for genetic models of cardiomyopathy","volume":"1","author":"Hart","year":"2022","journal-title":"J. Mol. Cell. Cardiol. Plus"},{"key":"10.1016\/j.ymthe.2025.12.042_bib37","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1016\/j.yjmcc.2009.08.013","article-title":"Cardiac strong inward rectifier potassium channels","volume":"48","author":"Anumonwo","year":"2010","journal-title":"J. Mol. Cell. Cardiol."},{"key":"10.1016\/j.ymthe.2025.12.042_bib38","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1161\/01.RES.0000237466.13252.5e","article-title":"Cardiac sodium channel Nav1.5 is regulated by a multiprotein complex composed of syntrophins and dystrophin","volume":"99","author":"Gavillet","year":"2006","journal-title":"Circ. Res."},{"key":"10.1016\/j.ymthe.2025.12.042_bib39","doi-asserted-by":"crossref","first-page":"294","DOI":"10.1161\/CIRCRESAHA.110.228312","article-title":"SAP97 and dystrophin macromolecular complexes determine two pools of cardiac sodium channels Nav1.5 in cardiomyocytes","volume":"108","author":"Petitprez","year":"2011","journal-title":"Circ. Res."},{"key":"10.1016\/j.ymthe.2025.12.042_bib40","article-title":"Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy","author":"Le Guiner","year":"2025","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.12.042_bib41","doi-asserted-by":"crossref","DOI":"10.3390\/ijms23042016","article-title":"Co-Administration of Simvastatin Does Not Potentiate the Benefit of Gene Therapy in the mdx Mouse Model for Duchenne Muscular Dystrophy","volume":"23","author":"Bourg","year":"2022","journal-title":"Int. J. Mol. Sci."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001625010652?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001625010652?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T15:36:19Z","timestamp":1775057779000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001625010652"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4]]},"references-count":41,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2026,4]]}},"alternative-id":["S1525001625010652"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2025.12.042","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2026,4]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Transgene-induced cardiotoxicity in high-dose AAV gene transfer","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2025.12.042","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}