{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,8]],"date-time":"2026-10-08T07:03:33Z","timestamp":1791443013602,"version":"4.3.3"},"reference-count":67,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2019,3,1]],"date-time":"2019-03-01T00:00:00Z","timestamp":1551398400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2019,1,9]],"date-time":"2019-01-09T00:00:00Z","timestamp":1546992000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"NIH","doi-asserted-by":"publisher","award":["AR065139"],"award-info":[{"award-number":["AR065139"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"NIH","doi-asserted-by":"publisher","award":["HL122332"],"award-info":[{"award-number":["HL122332"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005202","name":"Muscular Dystrophy Association","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005202","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Public Health Service, National Research Service Award","award":["T32 GM007270"],"award-info":[{"award-number":["T32 GM007270"]}]},{"DOI":"10.13039\/100000057","name":"National Institute of General Medical Sciences","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000057","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Solid Biosciences"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2019,3]]},"DOI":"10.1016\/j.ymthe.2019.01.002","type":"journal-article","created":{"date-parts":[[2019,2,1]],"date-time":"2019-02-01T11:42:37Z","timestamp":1549021357000},"page":"623-635","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":123,"title":["Development of Novel Micro-dystrophins with Enhanced Functionality"],"prefix":"10.1016","volume":"27","author":[{"given":"Julian N.","family":"Ramos","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katrin","family":"Hollinger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Niclas E.","family":"Bengtsson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James M.","family":"Allen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen D.","family":"Hauschka","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey S.","family":"Chamberlain","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2019.01.002_bib1","series-title":"Duchenne Muscular Dystrophy","author":"Emery","year":"2003"},{"key":"10.1016\/j.ymthe.2019.01.002_bib2","doi-asserted-by":"crossref","first-page":"14454","DOI":"10.1038\/ncomms14454","article-title":"Muscle-specific CRISPR\/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy","volume":"8","author":"Bengtsson","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2019.01.002_bib3","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1126\/science.aad5143","article-title":"In\u00a0vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy","volume":"351","author":"Nelson","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2019.01.002_bib4","doi-asserted-by":"crossref","first-page":"86","DOI":"10.1126\/science.aau1549","article-title":"Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy","volume":"362","author":"Amoasii","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2019.01.002_bib5","doi-asserted-by":"crossref","first-page":"2604","DOI":"10.1016\/j.ymthe.2018.08.010","article-title":"CRISPR-induced deletion with saCas9 restores dystrophin expression in dystrophic models in\u00a0vitro and in\u00a0vivo","volume":"26","author":"Duch\u00eane","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib6","doi-asserted-by":"crossref","first-page":"1125","DOI":"10.1016\/j.ymthe.2017.02.019","article-title":"Progress toward gene therapy for Duchenne muscular dystrophy","volume":"25","author":"Chamberlain","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib7","doi-asserted-by":"crossref","first-page":"1429","DOI":"10.1056\/NEJMoa1000228","article-title":"Dystrophin immunity in Duchenne\u2019s muscular dystrophy","volume":"363","author":"Mendell","year":"2010","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2019.01.002_bib8","doi-asserted-by":"crossref","first-page":"629","DOI":"10.1002\/ana.22251","article-title":"Sustained alpha-sarcoglycan\u00a0gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D","volume":"68","author":"Mendell","year":"2010","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib9","doi-asserted-by":"crossref","first-page":"828","DOI":"10.1038\/nm1085","article-title":"Systemic delivery of genes to striated muscles using adeno-associated viral vectors","volume":"10","author":"Gregorevic","year":"2004","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2019.01.002_bib10","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1038\/mt.2009.258","article-title":"Characterization of genome integrity for oversized recombinant AAV vector","volume":"18","author":"Dong","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib11","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1038\/mt.2010.205","article-title":"Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6","volume":"19","author":"Odom","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib12","doi-asserted-by":"crossref","first-page":"552","DOI":"10.1089\/hum.2013.210","article-title":"Full-length dystrophin reconstitution with adeno-associated viral vectors","volume":"25","author":"Lostal","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib13","doi-asserted-by":"crossref","first-page":"122","DOI":"10.1007\/BF00286703","article-title":"Characterization of patients with glycerol kinase deficiency utilizing cDNA probes for the Duchenne muscular dystrophy locus","volume":"83","author":"Towbin","year":"1989","journal-title":"Hum. Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib14","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1083\/jcb.134.1.93","article-title":"Forced expression of dystrophin deletion constructs reveals structure-function correlations","volume":"134","author":"Rafael","year":"1996","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib15","doi-asserted-by":"crossref","first-page":"1399","DOI":"10.1083\/jcb.150.6.1399","article-title":"Assembly of the dystrophin-associated protein complex\u00a0does not require the dystrophin COOH-terminal domain","volume":"150","author":"Crawford","year":"2000","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib16","first-page":"498","article-title":"The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion","volume":"45","author":"Koenig","year":"1989","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib17","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1038\/343180a0","article-title":"Very mild muscular dystrophy associated with the deletion of 46% of dystrophin","volume":"343","author":"England","year":"1990","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2019.01.002_bib18","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1093\/hmg\/3.6.919","article-title":"Half the dystrophin gene is apparently enough for a mild clinical course: confirmation of its potential\u00a0use for gene therapy","volume":"3","author":"Passos-Bueno","year":"1994","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib19","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1038\/nm0302-253","article-title":"Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy","volume":"8","author":"Harper","year":"2002","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2019.01.002_bib20","doi-asserted-by":"crossref","first-page":"13714","DOI":"10.1073\/pnas.240335297","article-title":"Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model","volume":"97","author":"Wang","year":"2000","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2019.01.002_bib21","doi-asserted-by":"crossref","first-page":"821","DOI":"10.1016\/j.ymthe.2004.07.025","article-title":"AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype","volume":"10","author":"Yoshimura","year":"2004","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib22","doi-asserted-by":"crossref","first-page":"1195","DOI":"10.1113\/jphysiol.2010.201921","article-title":"Lateral transmission of force is impaired in skeletal muscles of dystrophic mice and very old rats","volume":"589","author":"Ramaswamy","year":"2011","journal-title":"J.\u00a0Physiol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib23","series-title":"The Molecular Mechanisms of Muscular Dystrophies","first-page":"14","article-title":"The structure and function of dystrophin","author":"Abmayr","year":"2006"},{"key":"10.1016\/j.ymthe.2019.01.002_bib24","doi-asserted-by":"crossref","first-page":"661","DOI":"10.1083\/jcb.135.3.661","article-title":"A new model for the interaction of dystrophin with F-actin","volume":"135","author":"Rybakova","year":"1996","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib25","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/0014-5793(95)00398-S","article-title":"Dystrophin and utrophin: the missing links!","volume":"369","author":"Winder","year":"1995","journal-title":"FEBS Lett."},{"key":"10.1016\/j.ymthe.2019.01.002_bib26","series-title":"The Molecular Mechanisms of Muscular Dystrophies","first-page":"1","article-title":"Structure and function of the dystrophin-glycoprotein complex","author":"Ervasti","year":"2006"},{"key":"10.1016\/j.ymthe.2019.01.002_bib27","doi-asserted-by":"crossref","first-page":"5723","DOI":"10.1073\/pnas.1323842111","article-title":"Microtubule binding distinguishes dystrophin from utrophin","volume":"111","author":"Belanto","year":"2014","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2019.01.002_bib28","doi-asserted-by":"crossref","first-page":"546","DOI":"10.1016\/j.jmb.2009.04.025","article-title":"Mapping of the lipid-binding and stability properties of the central rod domain of human dystrophin","volume":"389","author":"Legardinier","year":"2009","journal-title":"J.\u00a0Mol. Biol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib29","first-page":"755","article-title":"The dystrophin glycoprotein complex regulates the epigenetic activation of muscle stem cell commitment","volume":"22","author":"Chang","year":"2018","journal-title":"Cell\u00a0Stem Cell"},{"key":"10.1016\/j.ymthe.2019.01.002_bib30","doi-asserted-by":"crossref","first-page":"2978","DOI":"10.1093\/hmg\/ddy197","article-title":"Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16-17","volume":"27","author":"Adams","year":"2018","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib31","doi-asserted-by":"crossref","first-page":"624","DOI":"10.1172\/JCI36612","article-title":"Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy","volume":"119","author":"Lai","year":"2009","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2019.01.002_bib32","doi-asserted-by":"crossref","first-page":"27305","DOI":"10.1074\/jbc.270.45.27305","article-title":"Identification and characterization of the dystrophin anchoring site on beta-dystroglycan","volume":"270","author":"Jung","year":"1995","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2019.01.002_bib33","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1152\/physrev.00028.2001","article-title":"Function and genetics of dystrophin and dystrophin-related proteins in muscle","volume":"82","author":"Blake","year":"2002","journal-title":"Physiol. Rev."},{"key":"10.1016\/j.ymthe.2019.01.002_bib34","doi-asserted-by":"crossref","first-page":"693","DOI":"10.1093\/hmg\/ddh087","article-title":"ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to beta-dystroglycan","volume":"13","author":"Ishikawa-Sakurai","year":"2004","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib35","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1038\/ng1294-333","article-title":"Dp71 can restore the dystrophin-associated glycoprotein complex in muscle but fails to prevent dystrophy","volume":"8","author":"Cox","year":"1994","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib36","doi-asserted-by":"crossref","first-page":"4975","DOI":"10.1074\/jbc.270.10.4975","article-title":"Identification of alpha-syntrophin binding to syntrophin triplet, dystrophin, and utrophin","volume":"270","author":"Yang","year":"1995","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2019.01.002_bib37","doi-asserted-by":"crossref","first-page":"1451","DOI":"10.1089\/10430340260185085","article-title":"Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice","volume":"13","author":"Watchko","year":"2002","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib38","doi-asserted-by":"crossref","first-page":"16105","DOI":"10.1038\/ncomms16105","article-title":"Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy","volume":"8","author":"Le Guiner","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2019.01.002_bib39","doi-asserted-by":"crossref","first-page":"744","DOI":"10.1038\/sj.gt.3302686","article-title":"Restoration of all dystrophin protein interactions by functional domains in trans does not rescue dystrophy","volume":"13","author":"Gardner","year":"2006","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib40","doi-asserted-by":"crossref","first-page":"873","DOI":"10.1083\/jcb.134.4.873","article-title":"Transgenic mdx mice expressing dystrophin with a deletion in the actin-binding domain display a \u201cmild Becker\u201d phenotype","volume":"134","author":"Corrado","year":"1996","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib41","doi-asserted-by":"crossref","first-page":"3975","DOI":"10.1093\/hmg\/ddn301","article-title":"Molecular and cellular adaptations to chronic myotendinous strain injury in mdx mice expressing a truncated dystrophin","volume":"17","author":"Banks","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib42","doi-asserted-by":"crossref","first-page":"e1000958","DOI":"10.1371\/journal.pgen.1000958","article-title":"The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins","volume":"6","author":"Banks","year":"2010","journal-title":"PLoS Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib43","doi-asserted-by":"crossref","first-page":"320","DOI":"10.1038\/sj.mt.6300027","article-title":"Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle","volume":"15","author":"Salva","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib44","doi-asserted-by":"crossref","first-page":"743","DOI":"10.1016\/0092-8674(95)90471-9","article-title":"Nitric oxide synthase complexed with dystrophin and absent from skeletal muscle sarcolemma in Duchenne muscular dystrophy","volume":"82","author":"Brenman","year":"1995","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2019.01.002_bib45","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1038\/nature07414","article-title":"Sarcolemma-localized nNOS is required to maintain activity after mild exercise","volume":"456","author":"Kobayashi","year":"2008","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2019.01.002_bib46","doi-asserted-by":"crossref","first-page":"15090","DOI":"10.1073\/pnas.95.25.15090","article-title":"Impaired metabolic modulation of alpha-adrenergic vasoconstriction in dystrophin-deficient skeletal muscle","volume":"95","author":"Thomas","year":"1998","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2019.01.002_bib47","doi-asserted-by":"crossref","first-page":"363","DOI":"10.1083\/jcb.200905048","article-title":"Dystrophin is a microtubule-associated protein","volume":"186","author":"Prins","year":"2009","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib48","doi-asserted-by":"crossref","first-page":"2090","DOI":"10.1093\/hmg\/ddy113","article-title":"Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins","volume":"27","author":"Nelson","year":"2018","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib49","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1186\/s13395-015-0040-z","article-title":"In\u00a0vitro stability of therapeutically relevant, internally truncated dystrophins","volume":"5","author":"McCourt","year":"2015","journal-title":"Skelet. Muscle"},{"key":"10.1016\/j.ymthe.2019.01.002_bib50","doi-asserted-by":"crossref","first-page":"e40066","DOI":"10.1371\/journal.pone.0040066","article-title":"The crystal structures of dystrophin and utrophin spectrin repeats: implications for domain boundaries","volume":"7","author":"Muthu","year":"2012","journal-title":"PLoS ONE"},{"key":"10.1016\/j.ymthe.2019.01.002_bib51","doi-asserted-by":"crossref","first-page":"1501","DOI":"10.1038\/mt.2012.111","article-title":"Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies","volume":"20","author":"Wang","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib52","doi-asserted-by":"crossref","first-page":"5880","DOI":"10.1093\/hmg\/ddv310","article-title":"Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus","volume":"24","author":"Yue","year":"2015","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib53","doi-asserted-by":"crossref","first-page":"1427","DOI":"10.1038\/mt.2009.116","article-title":"Evaluation of vascular delivery methodologies to enhance\u00a0rAAV6-mediated gene transfer to canine striated musculature","volume":"17","author":"Gregorevic","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib54","doi-asserted-by":"crossref","first-page":"1859","DOI":"10.1091\/mbc.11.5.1859","article-title":"A role for nitric oxide in muscle repair: nitric oxide-mediated activation of muscle satellite cells","volume":"11","author":"Anderson","year":"2000","journal-title":"Mol. Biol. Cell"},{"key":"10.1016\/j.ymthe.2019.01.002_bib55","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1083\/jcb.200105110","article-title":"A nitric oxide synthase transgene ameliorates muscular dystrophy in mdx mice","volume":"155","author":"Wehling","year":"2001","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib56","doi-asserted-by":"crossref","first-page":"1331","DOI":"10.1038\/mt.2010.308","article-title":"Transcription factor rational design improves directed differentiation of human mesenchymal stem cells into skeletal myocytes","volume":"19","author":"Gon\u00e7alves","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib57","doi-asserted-by":"crossref","first-page":"759","DOI":"10.1089\/hum.2008.197","article-title":"Partial rescue of growth failure in growth hormone (GH)-deficient\u00a0mice by a single injection of a double-stranded adeno-associated viral vector expressing the GH gene driven by a muscle-specific regulatory cassette","volume":"20","author":"Martari","year":"2009","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib58","doi-asserted-by":"crossref","first-page":"1149","DOI":"10.1093\/hmg\/5.8.1149","article-title":"Differential expression of dystrophin isoforms in strains of mdx mice with different mutations","volume":"5","author":"Im","year":"1996","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2019.01.002_bib59","doi-asserted-by":"crossref","first-page":"445","DOI":"10.1007\/978-1-61779-343-1_26","article-title":"Analysis of fiber-type differences in reporter gene expression of \u03b2-gal transgenic muscle","volume":"798","author":"Tai","year":"2012","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib60","doi-asserted-by":"crossref","first-page":"216","DOI":"10.1016\/j.omtm.2017.06.006","article-title":"A five-repeat micro-dystrophin gene ameliorated dystrophic phenotype in the severe Dba\/2j-mdx model of Duchenne muscular dystrophy","volume":"6","author":"Hakim","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2019.01.002_bib61","doi-asserted-by":"crossref","first-page":"1086","DOI":"10.1038\/sj.mt.6300144","article-title":"Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure","volume":"15","author":"Townsend","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib62","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1038\/mt.2008.264","article-title":"Cardiac expression of a mini-dystrophin that normalizes skeletal muscle force only partially restores heart function in aged Mdx mice","volume":"17","author":"Bostick","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.01.002_bib63","doi-asserted-by":"crossref","first-page":"787","DOI":"10.1038\/nm1439","article-title":"rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice","volume":"12","author":"Gregorevic","year":"2006","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2019.01.002_bib64","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1007\/978-1-4939-7374-3_18","article-title":"AAV6 vector production and purification for muscle gene therapy","volume":"1687","author":"Halbert","year":"2018","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib65","doi-asserted-by":"crossref","first-page":"2263","DOI":"10.1016\/S0002-9440(10)64502-6","article-title":"Improved contractile function of the mdx dystrophic mouse diaphragm muscle after insulin-like growth factor-I administration","volume":"161","author":"Gregorevic","year":"2002","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib66","doi-asserted-by":"crossref","first-page":"177","DOI":"10.1002\/jmor.1052210207","article-title":"Relationship between muscle fiber types and sizes and muscle architectural properties in the mouse hindlimb","volume":"221","author":"Burkholder","year":"1994","journal-title":"J.\u00a0Morphol."},{"key":"10.1016\/j.ymthe.2019.01.002_bib67","doi-asserted-by":"crossref","first-page":"725","DOI":"10.1111\/j.1440-1681.2007.04865.x","article-title":"Diaphragm muscle strip preparation for evaluation of gene therapies in mdx mice","volume":"35","author":"Faulkner","year":"2008","journal-title":"Clin. Exp. Pharmacol. Physiol."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S152500161930005X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S152500161930005X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2019,8,7]],"date-time":"2019-08-07T17:41:46Z","timestamp":1565199706000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S152500161930005X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,3]]},"references-count":67,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2019,3]]}},"alternative-id":["S152500161930005X"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2019.01.002","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2019,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Development of Novel Micro-dystrophins with Enhanced Functionality","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2019.01.002","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2019.01.019","name":"associatedlink","label":"CrossRef DOI link to the associated document"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2019 The Authors.","name":"copyright","label":"Copyright"}]}}