{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T11:19:50Z","timestamp":1788520790972,"version":"build-2803163510"},"reference-count":29,"publisher":"SAGE Publications","issue":"5","license":[{"start":{"date-parts":[[2026,1,19]],"date-time":"2026-01-19T00:00:00Z","timestamp":1768780800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,1,19]],"date-time":"2026-01-19T00:00:00Z","timestamp":1768780800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2026,9]]},"abstract":"<jats:p>\n                    This meeting report summarizes the presentations and discussions held at the summit on \u2018Challenges in Gene Therapy\u2019 hosted by the Muscular Dystrophy Association (MDA) in 2024. Topics broadly cover\n                    <jats:italic toggle=\"yes\">in vitro<\/jats:italic>\n                    and\n                    <jats:italic toggle=\"yes\">in vivo<\/jats:italic>\n                    models for understanding of gene therapy related immune responses, strategies to improve safety and efficacy of next-generation gene therapies, and clinical site readiness for gene therapy and post-treatment monitoring. This year's summit also marked the launch of MDA's gene therapy support network \u2013 a national resource for patients and clinicians in the MDA care center network aimed at providing sites with the information and resources needed to deliver genetic medicines within our neuromuscular community. A series of recommendations around 8 topic areas including predicting immune responses, long-term follow up of treated individuals, and ethical considerations was derived from content presented at the meeting.\n                  <\/jats:p>","DOI":"10.1177\/22143602251364325","type":"journal-article","created":{"date-parts":[[2026,1,19]],"date-time":"2026-01-19T09:47:41Z","timestamp":1768816061000},"page":"1159-1175","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["Meeting report: 2024 Muscular Dystrophy Association summit on \u2018Safety and challenges in gene therapy of neuromuscular diseases\u2019"],"prefix":"10.1177","volume":"13","author":[{"given":"Angela","family":"Lek","sequence":"first","affiliation":[{"name":"Muscular Dystrophy Association"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Evrim","family":"Atas","sequence":"additional","affiliation":[{"name":"Muscular Dystrophy Association"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian","family":"Lin","sequence":"additional","affiliation":[{"name":"Muscular Dystrophy Association"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharon E","family":"Hesterlee","sequence":"additional","affiliation":[{"name":"Muscular Dystrophy Association"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carsten G","family":"B\u00f6nnemann","sequence":"additional","affiliation":[{"name":"National Institutes of Health"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barry J","family":"Byrne","sequence":"additional","affiliation":[{"name":"University of Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,1,19]]},"reference":[{"key":"e_1_3_3_2_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2008.186"},{"key":"e_1_3_3_3_2","doi-asserted-by":"publisher","DOI":"10.1002\/humu.22758"},{"key":"e_1_3_3_4_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2023.026"},{"key":"e_1_3_3_5_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2021.047"},{"key":"e_1_3_3_6_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2012.26"},{"key":"e_1_3_3_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2021.03.014"},{"key":"e_1_3_3_8_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2023.101144"},{"key":"e_1_3_3_9_2","doi-asserted-by":"publisher","DOI":"10.1002\/ijch.201100094"},{"key":"e_1_3_3_10_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-240002"},{"key":"e_1_3_3_11_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMc2212912"},{"key":"e_1_3_3_12_2","doi-asserted-by":"crossref","unstructured":"Khan S Haegel H Hollenstein H et al. 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J Neuromuscul Dis 2021; 8: S325\u2013S340.","journal-title":"J Neuromuscul Dis"},{"key":"e_1_3_3_16_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-019-0594-0"},{"key":"e_1_3_3_17_2","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2023.1094279"},{"key":"e_1_3_3_18_2","doi-asserted-by":"publisher","DOI":"10.1177\/17562864221149781"},{"key":"e_1_3_3_19_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2021.243"},{"key":"e_1_3_3_20_2","unstructured":"CureDuchenne Muscular Dystrophy Association and Parent Project Muscular Dystrophy Announce Collaborative Project to Focus on Re-Dosing Gene Therapy in Duchenne Muscular Dystrophy n.d. https:\/\/www.mda.org\/press-releases\/cureduchenne-mda-and-parent-project-muscular-dystrophy-announce-collaborative-project-to-focus-on-re-dosing-gene-therapy-in-duchenne-muscular-dystrophy"},{"key":"e_1_3_3_21_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.lanepe.2023.100817"},{"key":"e_1_3_3_22_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI153430"},{"key":"e_1_3_3_23_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2307952"},{"key":"e_1_3_3_24_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2024.101349"},{"key":"e_1_3_3_25_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41587-021-00989-2"},{"key":"e_1_3_3_26_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.immuni.2021.03.003"},{"key":"e_1_3_3_27_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-022-28531-1"},{"key":"e_1_3_3_28_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2023.113493"},{"key":"e_1_3_3_29_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2020.11.054"},{"key":"e_1_3_3_30_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(23)00175-4"}],"container-title":["Journal of Neuromuscular Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/22143602251364325","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/22143602251364325","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/22143602251364325","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T11:07:44Z","timestamp":1788520064000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/22143602251364325"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,1,19]]},"references-count":29,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2026,9]]}},"alternative-id":["10.1177\/22143602251364325"],"URL":"https:\/\/doi.org\/10.1177\/22143602251364325","relation":{},"ISSN":["2214-3599","2214-3602"],"issn-type":[{"value":"2214-3599","type":"print"},{"value":"2214-3602","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,1,19]]}}}