{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,5,13]],"date-time":"2025-05-13T15:52:27Z","timestamp":1747151547021,"version":"3.40.5"},"reference-count":61,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2020,11,3]],"date-time":"2020-11-03T00:00:00Z","timestamp":1604361600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Pulmonology"],"published-print":{"date-parts":[[2021,4]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Gene therapy is an attractive approach being intensively studied to prevent muscle deterioration in patients with Duchenne muscular dystrophy. While clinical trials are only in early stages, initial reports are promising for its effects on ambulation. Cardiopulmonary failure, however, is the most common cause of mortality in Duchenne muscular dystrophy (DMD) patients, and little is known regarding the prospects for gene therapy on alleviating DMD\u2010associated cardiomyopathy and respiratory failure. Here we review current knowledge regarding effects of gene therapy on DMD cardiomyopathy and discuss respiratory endpoints that should be considered as outcome measures in future clinical trials.<\/jats:p>","DOI":"10.1002\/ppul.25079","type":"journal-article","created":{"date-parts":[[2020,11,3]],"date-time":"2020-11-03T18:35:12Z","timestamp":1604428512000},"page":"729-737","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["The effect of emerging molecular and genetic therapies on cardiopulmonary disease in Duchenne muscular dystrophy"],"prefix":"10.1002","volume":"56","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0145-9705","authenticated-orcid":false,"given":"Douglas A.","family":"McKim","sequence":"first","affiliation":[{"name":"Division of Respiratory Medicine, CANVent Respiratory Rehabilitation Services, The Ottawa Hospital Research Institute University of Ottawa  Ottawa Ontario Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8595-3577","authenticated-orcid":false,"given":"Timothy P.","family":"Cripe","sequence":"additional","affiliation":[{"name":"Division of Pediatric Hematology, Oncology, Blood and Marrow Transplant, Nationwide Children's Hospital Ohio State University College of Medicine  Columbus Ohio USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0524-9908","authenticated-orcid":false,"given":"Linda H.","family":"Cripe","sequence":"additional","affiliation":[{"name":"Division of Pediatric Cardiology, Nationwide Children's Hospital Ohio State University College of Medicine  Columbus Ohio USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2020,11,3]]},"reference":[{"doi-asserted-by":"publisher","key":"e_1_2_11_2_1","DOI":"10.1002\/ana.23528"},{"doi-asserted-by":"publisher","key":"e_1_2_11_3_1","DOI":"10.1038\/sj.embor.7400221"},{"doi-asserted-by":"publisher","key":"e_1_2_11_4_1","DOI":"10.1001\/jamaneurol.2020.1484"},{"doi-asserted-by":"publisher","key":"e_1_2_11_5_1","DOI":"10.1007\/s11010-020-03834-3"},{"doi-asserted-by":"publisher","key":"e_1_2_11_6_1","DOI":"10.1093\/hmg\/ddz148"},{"doi-asserted-by":"publisher","key":"e_1_2_11_7_1","DOI":"10.1038\/mt.2008.76"},{"doi-asserted-by":"publisher","key":"e_1_2_11_8_1","DOI":"10.1089\/hum.2018.085"},{"doi-asserted-by":"publisher","key":"e_1_2_11_9_1","DOI":"10.1089\/hum.2015.107"},{"issue":"102","key":"e_1_2_11_10_1","first-page":"49","article-title":"Viral expression cassette elements to enhance transgene target specificity and expression in gene therapy","volume":"19","author":"Powell SK","year":"2015","journal-title":"Discov Med"},{"doi-asserted-by":"publisher","key":"e_1_2_11_11_1","DOI":"10.1038\/mt.2011.124"},{"doi-asserted-by":"publisher","key":"e_1_2_11_12_1","DOI":"10.1016\/j.ymthe.2018.07.011"},{"doi-asserted-by":"publisher","key":"e_1_2_11_13_1","DOI":"10.1038\/mt.2011.154"},{"doi-asserted-by":"publisher","key":"e_1_2_11_14_1","DOI":"10.1038\/sj.mt.6300027"},{"doi-asserted-by":"publisher","key":"e_1_2_11_15_1","DOI":"10.1001\/jamaneurol.2020.1484"},{"doi-asserted-by":"publisher","key":"e_1_2_11_16_1","DOI":"10.1016\/j.ymthe.2019.01.005"},{"doi-asserted-by":"publisher","key":"e_1_2_11_17_1","DOI":"10.1016\/j.omtm.2020.03.011"},{"doi-asserted-by":"publisher","key":"e_1_2_11_18_1","DOI":"10.1126\/science.aad5143"},{"doi-asserted-by":"publisher","key":"e_1_2_11_19_1","DOI":"10.1172\/jci.insight.124297"},{"doi-asserted-by":"publisher","key":"e_1_2_11_20_1","DOI":"10.1038\/s41591-019-0344-3"},{"doi-asserted-by":"publisher","key":"e_1_2_11_21_1","DOI":"10.1016\/j.ymthe.2020.04.017"},{"doi-asserted-by":"publisher","key":"e_1_2_11_22_1","DOI":"10.1183\/13993003.01418-2017"},{"doi-asserted-by":"publisher","key":"e_1_2_11_23_1","DOI":"10.2340\/16501977-2144"},{"doi-asserted-by":"publisher","key":"e_1_2_11_24_1","DOI":"10.1016\/j.nmd.2018.07.004"},{"doi-asserted-by":"publisher","key":"e_1_2_11_25_1","DOI":"10.1002\/ppul.23172"},{"doi-asserted-by":"publisher","key":"e_1_2_11_26_1","DOI":"10.1002\/ppul.21204"},{"doi-asserted-by":"publisher","key":"e_1_2_11_27_1","DOI":"10.1164\/ajrccm.164.12.2103052"},{"doi-asserted-by":"publisher","key":"e_1_2_11_28_1","DOI":"10.1164\/rccm.200307-885ST"},{"doi-asserted-by":"publisher","key":"e_1_2_11_29_1","DOI":"10.1164\/rccm.201703-0507WS"},{"doi-asserted-by":"publisher","key":"e_1_2_11_30_1","DOI":"10.1002\/mus.880040213"},{"doi-asserted-by":"publisher","key":"e_1_2_11_31_1","DOI":"10.1016\/S1474-4422(18)30025-5"},{"doi-asserted-by":"publisher","key":"e_1_2_11_32_1","DOI":"10.1177\/0883073810371003"},{"doi-asserted-by":"publisher","key":"e_1_2_11_33_1","DOI":"10.1155\/2011\/139769"},{"doi-asserted-by":"publisher","key":"e_1_2_11_34_1","DOI":"10.1183\/23120541.00135-2016"},{"doi-asserted-by":"publisher","key":"e_1_2_11_35_1","DOI":"10.1016\/j.apmr.2012.02.024"},{"doi-asserted-by":"publisher","key":"e_1_2_11_36_1","DOI":"10.1002\/mus.25089"},{"doi-asserted-by":"publisher","key":"e_1_2_11_37_1","DOI":"10.1513\/AnnalsATS.201507-475BC"},{"issue":"2","key":"e_1_2_11_38_1","first-page":"117","article-title":"Survival in Duchenne muscular dystrophy","volume":"31","author":"Rall S","year":"2012","journal-title":"Acta Myol"},{"issue":"2","key":"e_1_2_11_39_1","first-page":"121","article-title":"Improvement of survival in Duchenne muscular dystrophy: retrospective analysis of 835 patients","volume":"31","author":"Passamano L","year":"2012","journal-title":"Acta Myol"},{"doi-asserted-by":"publisher","key":"e_1_2_11_40_1","DOI":"10.1016\/j.nmd.2010.09.006"},{"doi-asserted-by":"publisher","key":"e_1_2_11_41_1","DOI":"10.1155\/2013\/406163"},{"doi-asserted-by":"publisher","key":"e_1_2_11_42_1","DOI":"10.1183\/09031936.06.00004906"},{"doi-asserted-by":"publisher","key":"e_1_2_11_43_1","DOI":"10.1016\/S0022-510X(99)00045-3"},{"doi-asserted-by":"publisher","key":"e_1_2_11_44_1","DOI":"10.1136\/thx.2007.084574"},{"doi-asserted-by":"publisher","key":"e_1_2_11_45_1","DOI":"10.1378\/chest.119.5.1371"},{"doi-asserted-by":"publisher","key":"e_1_2_11_46_1","DOI":"10.1136\/thx.2004.039388"},{"doi-asserted-by":"publisher","key":"e_1_2_11_47_1","DOI":"10.1097\/00002060-200206000-00003"},{"doi-asserted-by":"publisher","key":"e_1_2_11_48_1","DOI":"10.1177\/1479972314562209"},{"doi-asserted-by":"publisher","key":"e_1_2_11_49_1","DOI":"10.1183\/09031936.00127712"},{"doi-asserted-by":"publisher","key":"e_1_2_11_50_1","DOI":"10.2217\/cer-2019-0171"},{"doi-asserted-by":"publisher","key":"e_1_2_11_51_1","DOI":"10.1002\/ppul.24205"},{"doi-asserted-by":"publisher","key":"e_1_2_11_52_1","DOI":"10.1007\/s00415-011-5979-z"},{"doi-asserted-by":"publisher","key":"e_1_2_11_53_1","DOI":"10.1073\/pnas.1605336113"},{"doi-asserted-by":"publisher","key":"e_1_2_11_54_1","DOI":"10.3233\/JND-180351"},{"doi-asserted-by":"publisher","key":"e_1_2_11_55_1","DOI":"10.1002\/ppul.22688"},{"doi-asserted-by":"publisher","key":"e_1_2_11_56_1","DOI":"10.1016\/S0140-6736(15)60025-3"},{"doi-asserted-by":"publisher","key":"e_1_2_11_57_1","DOI":"10.1002\/ppul.23215"},{"doi-asserted-by":"publisher","key":"e_1_2_11_58_1","DOI":"10.1016\/0002-9343(87)90897-7"},{"doi-asserted-by":"publisher","key":"e_1_2_11_59_1","DOI":"10.1016\/S0003-9993(97)90001-0"},{"doi-asserted-by":"publisher","key":"e_1_2_11_60_1","DOI":"10.1164\/rccm.200512-1841OC"},{"doi-asserted-by":"publisher","key":"e_1_2_11_61_1","DOI":"10.1007\/s11999-015-4239-4"},{"doi-asserted-by":"publisher","key":"e_1_2_11_62_1","DOI":"10.1002\/ajmg.a.38413"}],"container-title":["Pediatric Pulmonology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ppul.25079","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/ppul.25079","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ppul.25079","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,1]],"date-time":"2023-09-01T23:34:34Z","timestamp":1693611274000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ppul.25079"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,11,3]]},"references-count":61,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2021,4]]}},"alternative-id":["10.1002\/ppul.25079"],"URL":"https:\/\/doi.org\/10.1002\/ppul.25079","archive":["Portico"],"relation":{},"ISSN":["8755-6863","1099-0496"],"issn-type":[{"type":"print","value":"8755-6863"},{"type":"electronic","value":"1099-0496"}],"subject":[],"published":{"date-parts":[[2020,11,3]]},"assertion":[{"value":"2020-07-31","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-08-28","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-11-03","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}