{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T07:53:30Z","timestamp":1791273210028,"version":"4.1.0"},"reference-count":60,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"funder":[{"DOI":"10.13039\/501100004243","name":"Prinses Beatrix Spierfonds","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004243","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100011641","name":"Muskelsvindfonden","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100011641","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100010275","name":"Deutsche Gesellschaft f\u00fcr Muskelkranke","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100010275","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100011640","name":"European Neuromuscular Centre","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100011640","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002426","name":"Fondazione Telethon","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100002426","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100011724","name":"Muscular Dystrophy UK","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100011724","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010767","name":"Stiftung f\u00fcr die Erforschung der Muskelkrankheiten","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100010767","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100013465","name":"French Muscular Dystrophy Association","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013465","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005693","name":"Parent Project Muscular Dystrophy","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005693","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","nmd-journal.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Neuromuscular Disorders"],"published-print":{"date-parts":[[2026,10]]},"DOI":"10.1016\/j.nmd.2026.107371","type":"journal-article","created":{"date-parts":[[2026,7,28]],"date-time":"2026-07-28T06:39:02Z","timestamp":1785220742000},"page":"107371","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"special_numbering":"C","title":["289th ENMC international workshop: assessing and managing emerging AAV related toxicities after gene therapy for neuromuscular disorders, 26 - 28 September 2025, Hoofddorp, The Netherlands"],"prefix":"10.1016","volume":"67","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1015-1529","authenticated-orcid":false,"given":"Rotem","family":"Orbach","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monica Rebeca","family":"Gil Garzon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3365-6933","authenticated-orcid":false,"given":"Hildegard","family":"B\u00fcning","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5930-2324","authenticated-orcid":false,"given":"Carsten","family":"B\u00f6nnemann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9102-5232","authenticated-orcid":false,"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carsten G.","family":"B\u00f6nnemann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hildegard","family":"B\u00fcning","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Basel T.","family":"Assaf","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katherine","family":"Beaverson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Serge","family":"Braun","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabriel","family":"Brooks","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barry","family":"Byrne","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana","family":"Buj-Bello","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Chamberlain","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jahannaz \u201cNaz\u201d","family":"Dastgir","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anil","family":"Dhawan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kevin M.","family":"Flanigan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pat","family":"Furlong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amitava \u201cTav\u201d","family":"Ganguli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monica Rebeca Gil","family":"Garzon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan","family":"Kirschner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bradley","family":"Hamilton","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharon","family":"Hesterlee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juliette","family":"Hordeaux","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Kajaste-Rudnitski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rohit","family":"Kohli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Genevieve \u201cJenny\u201d","family":"Laforet","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dan","family":"Levy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hugh","family":"McMillan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanis","family":"Mimouni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander P.","family":"Murphy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kanneboyina","family":"Nagaraju","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rosaline","family":"Quinlivan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rotem","family":"Orbach","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"Richardson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isabelle","family":"Richard","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuseppe","family":"Ronzitti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ulrike","family":"Schara-Schmidt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"Schwartz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qi","family":"Shen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth","family":"Vroom","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karim","family":"Wahbi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.nmd.2026.107371_bib0001","doi-asserted-by":"crossref","first-page":"884","DOI":"10.1016\/j.nmd.2023.09.008","article-title":"261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands","volume":"33","author":"Servais","year":"2023","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2026.107371_bib0002","doi-asserted-by":"crossref","first-page":"327","DOI":"10.3233\/JND-221639","article-title":"Meeting report: 2022 muscular dystrophy association summit on 'safety and challenges in gene transfer therapy","volume":"10","author":"Lek","year":"2023","journal-title":"J Neuromuscul Dis"},{"key":"10.1016\/j.nmd.2026.107371_bib0003","doi-asserted-by":"crossref","first-page":"1139","DOI":"10.3233\/JND-240002","article-title":"Meeting report: 2023 muscular dystrophy association summit on 'safety and challenges in gene therapy of neuromuscular diseases","volume":"11","author":"Lek","year":"2024","journal-title":"J Neuromuscul Dis"},{"key":"10.1016\/j.nmd.2026.107371_bib0004","doi-asserted-by":"crossref","first-page":"2294","DOI":"10.1056\/NEJMc2212912","article-title":"Dystrophin immunity after gene therapy for duchenne's muscular dystrophy","volume":"388","author":"Bonnemann","year":"2023","journal-title":"N Engl J Med"},{"key":"10.1016\/j.nmd.2026.107371_bib0005","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1038\/s41598-024-84077-w","article-title":"Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy","volume":"15","author":"Potter","year":"2025","journal-title":"Sci Rep"},{"key":"10.1016\/j.nmd.2026.107371_bib0006","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1208\/s12248-025-01192-w","article-title":"Assessment of immune responses against AAV encoded transgene products","volume":"28","author":"Gorovits","year":"2026","journal-title":"AAPS J"},{"key":"10.1016\/j.nmd.2026.107371_bib0007","doi-asserted-by":"crossref","first-page":"797","DOI":"10.1089\/hum.2013.092","article-title":"Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect","volume":"24","author":"Flanigan","year":"2013","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0008","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1089\/hum.2022.166","article-title":"T cell responses to dystrophin in a natural history study of duchenne muscular dystrophy","volume":"34","author":"Anthony","year":"2023","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0009","doi-asserted-by":"crossref","first-page":"342","DOI":"10.1038\/s41431-023-01516-4","article-title":"DAG1 haploinsufficiency is associated with sporadic and familial isolated or pauci-symptomatic hyperCKemia","volume":"32","author":"Traverso","year":"2024","journal-title":"Eur J Hum Genet"},{"key":"10.1016\/j.nmd.2026.107371_bib0010","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1089\/hum.2024.156","article-title":"Thrombotic microangiopathy associated with systemic adeno-associated virus gene transfer: review of reported cases","volume":"36","author":"Laforet","year":"2025","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0011","doi-asserted-by":"crossref","first-page":"4266","DOI":"10.1182\/bloodadvances.2021006419","article-title":"Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy","volume":"6","author":"Guillou","year":"2022","journal-title":"Blood Adv"},{"key":"10.1016\/j.nmd.2026.107371_bib0012","doi-asserted-by":"crossref","DOI":"10.1038\/mtm.2014.33","article-title":"B-Cell depletion is protective against anti-AAV capsid immune response: a human subject case study","volume":"1","author":"Corti","year":"2014","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.nmd.2026.107371_bib0013","doi-asserted-by":"crossref","first-page":"273","DOI":"10.1177\/2040620718791933","article-title":"Gene therapy for hemophilia: what does the future hold?","volume":"9","author":"Doshi","year":"2018","journal-title":"Ther Adv Hematol"},{"key":"10.1016\/j.nmd.2026.107371_bib0014","doi-asserted-by":"crossref","first-page":"2073","DOI":"10.1016\/j.ymthe.2020.06.001","article-title":"Long-Term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia B","volume":"28","author":"George","year":"2020","journal-title":"Mol Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0015","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1182\/blood-2013-01-306647","article-title":"Immune responses to AAV vectors: overcoming barriers to successful gene therapy","volume":"122","author":"Mingozzi","year":"2013","journal-title":"Blood"},{"key":"10.1016\/j.nmd.2026.107371_bib0016","doi-asserted-by":"crossref","DOI":"10.1016\/j.pharmthera.2019.107453","article-title":"Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery","volume":"207","author":"Nidetz","year":"2020","journal-title":"Pharmacol Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0017","doi-asserted-by":"crossref","first-page":"670","DOI":"10.3389\/fimmu.2020.00670","article-title":"Human immune responses to adeno-associated virus (AAV) vectors","volume":"11","author":"Ronzitti","year":"2020","journal-title":"Front Immunol"},{"key":"10.1016\/j.nmd.2026.107371_bib0018","doi-asserted-by":"crossref","first-page":"616","DOI":"10.1016\/j.ymthe.2023.01.010","article-title":"Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy","volume":"31","author":"Schulz","year":"2023","journal-title":"Mol Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0019","doi-asserted-by":"crossref","DOI":"10.1172\/JCI173510","article-title":"Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies","volume":"134","author":"Salabarria","year":"2024","journal-title":"J Clin Invest"},{"key":"10.1016\/j.nmd.2026.107371_bib0020","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1016\/j.omtm.2022.02.011","article-title":"Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations","volume":"25","author":"Mendell","year":"2022","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.nmd.2026.107371_bib0021","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2022.857276","article-title":"Overcoming the challenges imposed by humoral immunity to AAV vectors to achieve safe and efficient gene transfer in seropositive patients","volume":"13","author":"Gross","year":"2022","journal-title":"Front Immunol"},{"key":"10.1016\/j.nmd.2026.107371_bib0022","doi-asserted-by":"crossref","first-page":"2080","DOI":"10.1016\/j.ymthe.2024.05.004","article-title":"Engineered IgM and IgG cleaving enzymes for mitigating antibody neutralization and complement activation in AAV gene transfer","volume":"32","author":"Smith","year":"2024","journal-title":"Mol Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0023","article-title":"Enhancing AAV9-UFmuDys1 gene therapy efficacy through immunosuppression in mice with pre-existing immunity and enabling redosing strategies for duchenne muscular dystrophy","author":"Saha","year":"2025","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0024","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2024.101195","article-title":"Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74","volume":"32","author":"Potter","year":"2024","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.nmd.2026.107371_bib0025","doi-asserted-by":"crossref","first-page":"3694","DOI":"10.1038\/s41467-025-58778-3","article-title":"AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain","volume":"16","author":"Costa-Verdera","year":"2025","journal-title":"Nat Commun"},{"key":"10.1016\/j.nmd.2026.107371_bib0026","doi-asserted-by":"crossref","first-page":"214","DOI":"10.1038\/ni.2229","article-title":"Intrinsic antiviral immunity","volume":"13","author":"Yan","year":"2012","journal-title":"Nat Immunol"},{"key":"10.1016\/j.nmd.2026.107371_bib0028","doi-asserted-by":"crossref","first-page":"472","DOI":"10.1038\/s41591-025-04073-z","article-title":"Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy","volume":"32","author":"Buddle","year":"2026","journal-title":"Nat Med"},{"key":"10.1016\/j.nmd.2026.107371_bib0029","article-title":"Profiling neuroinflammatory markers and response to nusinersen in paediatric spinal muscular atrophy","volume":"14","author":"Zhang","year":"2024","journal-title":"Sci Rep"},{"key":"10.1016\/j.nmd.2026.107371_bib0030","doi-asserted-by":"crossref","DOI":"10.1172\/JCI153430","article-title":"Microvasculopathy in spinal muscular atrophy is driven by a reversible autonomous endothelial cell defect","volume":"132","author":"Zhou","year":"2022","journal-title":"J Clin Invest"},{"key":"10.1016\/j.nmd.2026.107371_bib0031","doi-asserted-by":"crossref","DOI":"10.1016\/j.nmd.2025.106208","article-title":"Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy","volume":"54","author":"Wolff","year":"2025","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2026.107371_bib0032","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2022.1001263","article-title":"rAAV immunogenicity, toxicity, and durability in 255 clinical trials: a meta-analysis","volume":"13","author":"Shen","year":"2022","journal-title":"Front Immunol"},{"key":"10.1016\/j.nmd.2026.107371_bib0033","doi-asserted-by":"crossref","first-page":"560","DOI":"10.1016\/j.jhep.2020.11.001","article-title":"Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy","volume":"74","author":"Chand","year":"2021","journal-title":"J Hepatol"},{"key":"10.1016\/j.nmd.2026.107371_bib0034","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1016\/j.jpeds.2020.05.044","article-title":"Subacute liver failure following gene replacement therapy for spinal muscular atrophy type 1","volume":"225","author":"Feldman","year":"2020","journal-title":"J Pediatr"},{"key":"10.1016\/j.nmd.2026.107371_bib0035","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2024.101296","article-title":"Subacute liver injury in two young infants following gene replacement therapy for spinal muscular atrophy","volume":"32","author":"Ables","year":"2024","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.nmd.2026.107371_bib0036","doi-asserted-by":"crossref","DOI":"10.1177\/10430342261423168","article-title":"Learnings from patient mortality after delandistrogene moxeparvovec administration: a report of two cases and expert committee considerations for future mitigation and management","author":"Shieh","year":"2026","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0037","article-title":"Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom","volume":"37","author":"Gowda","year":"2024","journal-title":"Lancet Reg Health Eur"},{"key":"10.1016\/j.nmd.2026.107371_bib0038","doi-asserted-by":"crossref","DOI":"10.1172\/JCI173702","article-title":"Hepatocyte-intrinsic SMN deficiency drives metabolic dysfunction and liver steatosis in spinal muscular atrophy","volume":"134","author":"Leow","year":"2024","journal-title":"J Clin Invest"},{"key":"10.1016\/j.nmd.2026.107371_bib0039","doi-asserted-by":"crossref","first-page":"828","DOI":"10.1038\/gt.2014.61","article-title":"A dystrophic muscle broadens the contribution and activation of immune cells reacting to rAAV gene transfer","volume":"21","author":"Ferrand","year":"2014","journal-title":"Gene Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0040","doi-asserted-by":"crossref","first-page":"S43","DOI":"10.1542\/peds.2018-0333F","article-title":"Obesity and endocrine management of the patient with duchenne muscular dystrophy","volume":"142","author":"Weber","year":"2018","journal-title":"Pediatrics"},{"key":"10.1016\/j.nmd.2026.107371_bib0041","doi-asserted-by":"crossref","DOI":"10.1016\/j.ebiom.2023.104894","article-title":"Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial","volume":"99","author":"Lawlor","year":"2024","journal-title":"EBioMedicine"},{"key":"10.1016\/j.nmd.2026.107371_bib0042","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.nmd.2026.107371_bib0043","doi-asserted-by":"crossref","first-page":"eadr1483","DOI":"10.1126\/scitranslmed.adr1483","article-title":"AAV8 gene therapy and dietary insults together precipitate cholestatic liver disease in a mouse model of X-linked myotubular myopathy","volume":"18","author":"Pannia","year":"2026","journal-title":"Sci Transl Med"},{"key":"10.1016\/j.nmd.2026.107371_bib0044","doi-asserted-by":"crossref","DOI":"10.1212\/WNL.0000000000210268","article-title":"Safety and efficacy of IV onasemnogene abeparvovec for pediatric patients with spinal muscular atrophy: the phase 3b SMART study","volume":"104","author":"McMillan","year":"2025","journal-title":"Neurology"},{"key":"10.1016\/j.nmd.2026.107371_bib0045","doi-asserted-by":"crossref","first-page":"1519","DOI":"10.1002\/acn3.50855","article-title":"Abnormal fatty acid metabolism is a core component of spinal muscular atrophy","volume":"6","author":"Deguise","year":"2019","journal-title":"Ann Clin Transl Neurol"},{"key":"10.1016\/j.nmd.2026.107371_bib0046","doi-asserted-by":"crossref","first-page":"448","DOI":"10.1016\/S2352-4642(25)00380-3","article-title":"Safety of in vivo gene therapy in children: mechanisms and management of liver injury","volume":"10","author":"Jeyaraj","year":"2026","journal-title":"Lancet Child Adolesc Health"},{"key":"10.1016\/j.nmd.2026.107371_bib0047","doi-asserted-by":"crossref","DOI":"10.1542\/peds.2025-074192","article-title":"ALT\/CK ratio as an early marker of liver injury after gene therapy in duchenne muscular dystrophy","volume":"157","author":"Drakou","year":"2026","journal-title":"Pediatrics"},{"key":"10.1016\/j.nmd.2026.107371_bib0048","article-title":"Transgene-induced cardiotoxicity in high-dose AAV gene transfer","author":"Biquand","year":"2025","journal-title":"Mol Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0049","doi-asserted-by":"crossref","first-page":"140","DOI":"10.1016\/j.ymthe.2025.10.041","article-title":"Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy","volume":"34","author":"Le Guiner","year":"2026","journal-title":"Mol Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0050","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.nmd.2026.107371_bib0051","doi-asserted-by":"crossref","first-page":"1094","DOI":"10.1161\/CIRCULATIONAHA.113.001340","article-title":"Restriction of calpain3 expression to the skeletal muscle prevents cardiac toxicity and corrects pathology in a murine model of limb-girdle muscular dystrophy","volume":"128","author":"Roudaut","year":"2013","journal-title":"Circulation"},{"key":"10.1016\/j.nmd.2026.107371_bib0052","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.nmd.2026.107371_bib0053","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2023.1094279","article-title":"Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase","volume":"14","author":"Hordeaux","year":"2023","journal-title":"Front Immunol"},{"key":"10.1016\/j.nmd.2026.107371_bib0054","doi-asserted-by":"crossref","first-page":"952","DOI":"10.1016\/j.ymthe.2024.02.002","article-title":"High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury","volume":"32","author":"Hordeaux","year":"2024","journal-title":"Mol Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0055","doi-asserted-by":"crossref","first-page":"2339","DOI":"10.1016\/j.ymthe.2025.05.017","article-title":"Lung endothelial transduction in a patient that succumbed to acute respiratory distress syndrome following high-dose rAAV9 gene therapy","volume":"33","author":"Hordeaux","year":"2025","journal-title":"Mol Ther"},{"key":"10.1016\/j.nmd.2026.107371_bib0056","doi-asserted-by":"crossref","first-page":"1092","DOI":"10.1056\/NEJMoa2307952","article-title":"Intrathecal gene therapy for giant axonal neuropathy","volume":"390","author":"Bharucha-Goebel","year":"2024","journal-title":"N Engl J Med"},{"key":"10.1016\/j.nmd.2026.107371_bib0057","doi-asserted-by":"crossref","DOI":"10.1016\/j.nmd.2025.105849","article-title":"68PSuspected dorsal root ganglion toxicity after intratecal gene therapy","volume":"53","author":"Carrera","year":"2025","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2026.107371_bib0058","doi-asserted-by":"crossref","first-page":"481","DOI":"10.1038\/s41591-025-04103-w","article-title":"Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial","volume":"32","author":"Proud","year":"2026","journal-title":"Nat Med"},{"key":"10.1016\/j.nmd.2026.107371_bib0059","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1056\/NEJMoa2005056","article-title":"SOD1 suppression with adeno-associated virus and MicroRNA in familial ALS","volume":"383","author":"Mueller","year":"2020","journal-title":"N Engl J Med"},{"key":"10.1016\/j.nmd.2026.107371_bib0060","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.nmd.2026.107371_bib0061","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"}],"container-title":["Neuromuscular Disorders"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0960896626010394?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0960896626010394?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T07:25:37Z","timestamp":1791271537000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0960896626010394"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,10]]},"references-count":60,"alternative-id":["S0960896626010394"],"URL":"https:\/\/doi.org\/10.1016\/j.nmd.2026.107371","relation":{},"ISSN":["0960-8966"],"issn-type":[{"value":"0960-8966","type":"print"}],"subject":[],"published":{"date-parts":[[2026,10]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"289th ENMC international workshop: assessing and managing emerging AAV related toxicities after gene therapy for neuromuscular disorders, 26 - 28 September 2025, Hoofddorp, The Netherlands","name":"articletitle","label":"Article Title"},{"value":"Neuromuscular Disorders","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.nmd.2026.107371","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"simple-article","name":"content_type","label":"Content Type"},{"name":"copyright","label":"Copyright"}],"article-number":"107371"}}