{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T14:50:24Z","timestamp":1790952624588,"version":"4.1.0"},"reference-count":35,"publisher":"Massachusetts Medical Society","issue":"24","license":[{"start":{"date-parts":[[2025,6,26]],"date-time":"2025-06-26T00:00:00Z","timestamp":1750896000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.nejmgroup.org\/legal\/terms-of-use.htm"}],"funder":[{"DOI":"10.13039\/501100007937","name":"National High Level Hospital Clinical Research Funding (High Quality Clinical Research Project of Peking University First Hospital","doi-asserted-by":"publisher","award":["2022CR69"],"award-info":[{"award-number":["2022CR69"]}],"id":[{"id":"10.13039\/501100007937","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["82171393"],"award-info":[{"award-number":["82171393"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["N Engl J Med"],"published-print":{"date-parts":[[2025,6,26]]},"DOI":"10.1056\/nejmoa2407766","type":"journal-article","created":{"date-parts":[[2025,6,25]],"date-time":"2025-06-25T17:00:33Z","timestamp":1750870833000},"page":"2438-2446","source":"Crossref","is-referenced-by-count":26,"title":["AAV9-Mediated Gene Therapy for Infantile-Onset Pompe\u2019s Disease"],"prefix":"10.1056","volume":"392","author":[{"given":"Xiuwei","family":"Ma","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lu","family":"Zhuang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wenhao","family":"Ma","sequence":"additional","affiliation":[{"name":"Genecradle Therapeutics, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7544-7569","authenticated-orcid":false,"given":"Jun","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yingying","family":"Mao","sequence":"additional","affiliation":[{"name":"Genecradle Therapeutics, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jianhua","family":"Wang","sequence":"additional","affiliation":[{"name":"Ultrasonic Department, Seventh Medical Center of Chinese PLA General Hospital, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaodong","family":"Wang","sequence":"additional","affiliation":[{"name":"Genecradle Therapeutics, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juan","family":"Xu","sequence":"additional","affiliation":[{"name":"Department of Pharmacy, Medical Supplies Center of Chinese PLA General Hospital, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shuangqing","family":"Yu","sequence":"additional","affiliation":[{"name":"Genecradle Therapeutics, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruijie","family":"Gu","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yongxia","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhongqiu","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xinyang","family":"Jiang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sheng","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fang","family":"He","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiao","family":"Yang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lina","family":"Zhu","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shan","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanping","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ting","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qiuping","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhijie","family":"Wu","sequence":"additional","affiliation":[{"name":"Genecradle Therapeutics, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cengceng","family":"Zhang","sequence":"additional","affiliation":[{"name":"Genecradle Therapeutics, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yiying","family":"Zhang","sequence":"additional","affiliation":[{"name":"Children\u2019s Hospital, Zhejiang University School of Medicine, Hangzhou, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoyan","family":"Dong","sequence":"additional","affiliation":[{"name":"Genecradle Therapeutics, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hui","family":"Xiong","sequence":"additional","affiliation":[{"name":"Department of Neurology, Beijing Children\u2019s Hospital, Capital Medical University, National Center for Children\u2019s Health, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaobing","family":"Wu","sequence":"additional","affiliation":[{"name":"Genecradle Therapeutics, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9622-4062","authenticated-orcid":false,"given":"Zhichun","family":"Feng","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Chinese People\u2019s Liberation Army (PLA) General Hospital, Beijing"},{"name":"Department of Pediatric Medicine, Seventh Medical Center of Chinese PLA General Hospital, Beijing"},{"name":"National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing"},{"name":"Beijing Key Laboratory of Pediatric Organ Failure, Beijing"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"150","reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-4642(21)00308-4"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-010-9144-0"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMe2211515"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1080\/14712598.2022.2067476"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.expneurol.2016.07.013"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2023.02.014"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1038\/mtm.2015.7"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2018.016"},{"key":"e_1_3_5_10_2","first-page":"24","article-title":"AAV vector mediated gene therapy in Pompe model mice.","volume":"42","author":"Wu Z","year":"2022","unstructured":"Wu Z, Ma W, Dong Z, Wu X, Yang Y, Sheng W. AAV vector mediated gene therapy in Pompe model mice. China Biotechnology 2022;42:24-34 (https:\/\/manu60.magtech.com.cn\/biotech\/CN\/10.13523\/j.cb.2202040#9).","journal-title":"China Biotechnology"},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1067\/mhj.2002.121265"},{"key":"e_1_3_5_12_2","unstructured":"University Medical Center Rotterdam. Pompe disease GAA variant database (https:\/\/www.pompevariantdatabase.clmz.nl\/pompe_mutations_list.php?orderby=aMut_ID1)."},{"key":"e_1_3_5_13_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41436-019-0527-9"},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2007.05.011"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-008-0714-0"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1007\/s13311-018-0655-y"},{"key":"e_1_3_5_17_2","unstructured":"Hammersmith infant neurological examination: guidance notes for completion of the proforma. London: Mac Keith Press December 2023 (https:\/\/www.mackeith.co.uk\/wp-content\/uploads\/2024\/01\/Hammersmith-Infant-Neurological-Examination-HINE-Guidance-notes-update-20.12.2023-v2.pdf)."},{"key":"e_1_3_5_18_2","unstructured":"World Health Organization. Motor development milestones (https:\/\/www.who.int\/tools\/child-growth-standards\/standards\/motor-development-milestones)."},{"key":"e_1_3_5_19_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27955"},{"key":"e_1_3_5_20_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"e_1_3_5_21_2","doi-asserted-by":"publisher","DOI":"10.1542\/peds.112.2.332"},{"key":"e_1_3_5_22_2","doi-asserted-by":"publisher","DOI":"10.1097\/00005072-196501000-00008"},{"key":"e_1_3_5_23_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2011.10.012"},{"key":"e_1_3_5_24_2","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.13740"},{"key":"e_1_3_5_25_2","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12736"},{"key":"e_1_3_5_26_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2200587"},{"key":"e_1_3_5_27_2","doi-asserted-by":"publisher","DOI":"10.2147\/TCRM.S362871"},{"key":"e_1_3_5_28_2","doi-asserted-by":"publisher","DOI":"10.1165\/rcmb.2022-0424MA"},{"key":"e_1_3_5_29_2","doi-asserted-by":"publisher","DOI":"10.1038\/d41586-023-03732-w"},{"key":"e_1_3_5_30_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41577-018-0065-8"},{"key":"e_1_3_5_31_2","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-014-9707-6"},{"key":"e_1_3_5_32_2","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2012.44"},{"key":"e_1_3_5_33_2","first-page":"100475","article-title":"Characterization of immune response in Cross-Reactive Immunological Material (CRIM)-positive infantile Pompe disease patients treated with enzyme replacement therapy.","volume":"20","author":"Desai AK","year":"2019","unstructured":"Desai AK, Kazi ZB, Bali DS, Kishnani PS. Characterization of immune response in Cross-Reactive Immunological Material (CRIM)-positive infantile Pompe disease patients treated with enzyme replacement therapy. Mol Genet Metab Rep 2019;20:100475-100475.31193175","journal-title":"Mol Genet Metab Rep"},{"key":"e_1_3_5_34_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1708483"},{"key":"e_1_3_5_35_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2012.250"},{"key":"e_1_3_5_36_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.162.3853.570"}],"container-title":["New England Journal of Medicine"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.nejm.org\/doi\/pdf\/10.1056\/NEJMoa2407766","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,1,23]],"date-time":"2026-01-23T18:36:26Z","timestamp":1769193386000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.nejm.org\/doi\/10.1056\/NEJMoa2407766"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,6,26]]},"references-count":35,"journal-issue":{"issue":"24","published-print":{"date-parts":[[2025,6,26]]}},"alternative-id":["10.1056\/NEJMoa2407766"],"URL":"https:\/\/doi.org\/10.1056\/nejmoa2407766","relation":{},"ISSN":["0028-4793","1533-4406"],"issn-type":[{"value":"0028-4793","type":"print"},{"value":"1533-4406","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,6,26]]}}}