{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T02:31:40Z","timestamp":1791945100829,"version":"4.3.4"},"reference-count":33,"publisher":"Massachusetts Medical Society","issue":"10","license":[{"start":{"date-parts":[[2025,3,6]],"date-time":"2025-03-06T00:00:00Z","timestamp":1741219200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.nejmgroup.org\/legal\/terms-of-use.htm"}],"funder":[{"name":"Rocket Pharmaceuticals, Inc."}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["N Engl J Med"],"published-print":{"date-parts":[[2025,3,6]]},"DOI":"10.1056\/nejmoa2412392","type":"journal-article","created":{"date-parts":[[2024,11,18]],"date-time":"2024-11-18T15:46:08Z","timestamp":1731944768000},"page":"972-983","source":"Crossref","is-referenced-by-count":93,"title":["Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease"],"prefix":"10.1056","volume":"392","author":[{"given":"Barry","family":"Greenberg","sequence":"first","affiliation":[{"name":"University of California, San Diego Medical Center, La Jolla"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Taylor","sequence":"additional","affiliation":[{"name":"University of Colorado, Anschutz, Medical Center, Aurora"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Adler","sequence":"additional","affiliation":[{"name":"University of California, San Diego Medical Center, La Jolla"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven","family":"Colan","sequence":"additional","affiliation":[{"name":"Boston Children\u2019s Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Ricks","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals, Cranbury, NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Yarabe","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals, Cranbury, NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pavan","family":"Battiprolu","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals, Cranbury, NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gaurav","family":"Shah","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals, Cranbury, NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kinnari","family":"Patel","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals, Cranbury, NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Coggins","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals, Cranbury, NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susanna","family":"Carou-Keenan","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals, Cranbury, NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan D.","family":"Schwartz","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals, Cranbury, NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joseph W.","family":"Rossano","sequence":"additional","affiliation":[{"name":"Children\u2019s Hospital of Philadelphia, Philadelphia"},{"name":"Perelman School of Medicine, University of Pennsylvania, Philadelphia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"150","reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1097\/GIM.0b013e31820ad795"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCHEARTFAILURE.114.001105"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ijcard.2019.01.020"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2015.03.005"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.carpath.2016.07.005"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.recesp.2018.04.027"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCGEN.120.003117"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.31.1.51"},{"key":"e_1_3_5_10_2","doi-asserted-by":"publisher","DOI":"10.1038\/35022604"},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.10235"},{"key":"e_1_3_5_12_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00401-015-1385-4"},{"key":"e_1_3_5_13_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1808618116"},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.yjmcc.2017.05.007"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2009.371"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.healun.2014.08.002"},{"key":"e_1_3_5_17_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.cardfail.2021.11.007"},{"key":"e_1_3_5_18_2","first-page":"1080","article-title":"Pediatric heart transplantation-indications and outcomes in the current era.","volume":"6","author":"Thrush PT","year":"2014","unstructured":"Thrush PT, Hoffman TM. Pediatric heart transplantation-indications and outcomes in the current era. J Thorac Dis 2014;6:1080-1096.25132975","journal-title":"J Thorac Dis"},{"key":"e_1_3_5_19_2","doi-asserted-by":"publisher","DOI":"10.31486\/toj.19.0022"},{"key":"e_1_3_5_20_2","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aax1744"},{"key":"e_1_3_5_21_2","doi-asserted-by":"publisher","DOI":"10.1089\/humc.2013.208"},{"key":"e_1_3_5_22_2","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aat6072"},{"key":"e_1_3_5_23_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2016.130"},{"key":"e_1_3_5_24_2","doi-asserted-by":"publisher","DOI":"10.1038\/mtm.2015.7"},{"key":"e_1_3_5_25_2","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddv310"},{"key":"e_1_3_5_26_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2014.210"},{"key":"e_1_3_5_27_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2011.22"},{"key":"e_1_3_5_28_2","doi-asserted-by":"publisher","DOI":"10.1161\/01.RES.0000237661.18885.f6"},{"key":"e_1_3_5_29_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.1200708"},{"key":"e_1_3_5_30_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2014.03.032"},{"key":"e_1_3_5_31_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2018.02.014"},{"key":"e_1_3_5_32_2","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCOUTCOMES.115.001958"},{"key":"e_1_3_5_33_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2023.08.014"},{"key":"e_1_3_5_34_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI173510"}],"container-title":["New England Journal of Medicine"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.nejm.org\/doi\/pdf\/10.1056\/NEJMoa2412392","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,6,11]],"date-time":"2025-06-11T22:31:48Z","timestamp":1749681108000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.nejm.org\/doi\/10.1056\/NEJMoa2412392"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,3,6]]},"references-count":33,"journal-issue":{"issue":"10","published-print":{"date-parts":[[2025,3,6]]}},"alternative-id":["10.1056\/NEJMoa2412392"],"URL":"https:\/\/doi.org\/10.1056\/nejmoa2412392","relation":{},"ISSN":["0028-4793","1533-4406"],"issn-type":[{"value":"0028-4793","type":"print"},{"value":"1533-4406","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,3,6]]}}}