{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T21:19:50Z","timestamp":1777670390100,"version":"3.51.4"},"reference-count":5,"publisher":"SAGE Publications","issue":"13-14","license":[{"start":{"date-parts":[[2023,7,1]],"date-time":"2023-07-01T00:00:00Z","timestamp":1688169600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.liebertpub.com\/nv\/resources-tools\/text-and-data-mining-policy\/121\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2023,7,1]]},"DOI":"10.1089\/hum.2023.29244.bfs","type":"journal-article","created":{"date-parts":[[2023,7,18]],"date-time":"2023-07-18T11:34:27Z","timestamp":1689680067000},"page":"588-591","source":"Crossref","is-referenced-by-count":3,"title":["Case Study Cites Immune Reaction to High Adeno-Associated Virus Dose in Explaining Duchenne Muscular Dystrophy Trial Death"],"prefix":"10.1177","volume":"34","author":[{"given":"Alex","family":"Philippidis","sequence":"first","affiliation":[{"name":"Mary Ann Liebert, Inc., Publishers, New Rochelle, New York, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","reference":[{"key":"p_4","doi-asserted-by":"crossref","unstructured":"Lek A, Wong B, Keeler A, etal Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-Delivered CRISPR-Transactivator. medRxiv 2023. Available from: https:\/\/ www.medrxiv.org\/content\/10.1101\/2023.05.16.232 89881v1 [Last accessed: June 14, 2023].","DOI":"10.1101\/2023.05.16.23289881"},{"key":"p_5","unstructured":"Verve Therapeutics. Verve Establishes Global Collaboration with Lilly to Advance Verve's In Vivo Gene Editing Program Targeting Lp(a) for the Treatment of Atherosclerotic Cardiovascular Disease. 2023. Available from: https:\/\/ir.vervetx .com\/news-releases\/news-release-details\/verveestablishes-global-collaboration-lilly-advance-ver ves-vivo [Last accessed: June 15, 2023]."},{"key":"p_6","volume":"202","author":"Philippidis A.","journal-title":"Biotechnology News"},{"key":"p_7","unstructured":"CRISPR Therapeutics and Vertex Pharmaceuticals. FDA Accepts Biologics License Applications for Exagamglogene Autotemcel (Exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia. 2023. Available from: http:\/\/ir .crisprtx.com\/news-releases\/news-release-details\/ fda-accepts-biologics-license-applications-exagamg logene [Last accessed: June 13, 2023]."},{"key":"p_8","unstructured":"Vertex Pharmaceuticals and CRISPR Therapeutics. Positive Results from Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology Association (EHA) Congress. 2023. Available from: https:\/\/investors .vrtx.com\/news-releases\/news-release-details\/posi tive-results-pivotal-trials-exa-cel-transfusion-depen dent [Last accessed: June 13, 2023]."}],"container-title":["Human Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.liebertpub.com\/doi\/full-xml\/10.1089\/hum.2023.29244.bfs","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.liebertpub.com\/doi\/pdf\/10.1089\/hum.2023.29244.bfs","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T07:56:37Z","timestamp":1777449397000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/full\/10.1089\/hum.2023.29244.bfs"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,7,1]]},"references-count":5,"journal-issue":{"issue":"13-14","published-print":{"date-parts":[[2023,7,1]]}},"alternative-id":["10.1089\/hum.2023.29244.bfs"],"URL":"https:\/\/doi.org\/10.1089\/hum.2023.29244.bfs","relation":{},"ISSN":["1043-0342","1557-7422"],"issn-type":[{"value":"1043-0342","type":"print"},{"value":"1557-7422","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,7,1]]}}}