{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,10]],"date-time":"2026-11-10T01:25:14Z","timestamp":1794273914752,"version":"4.9.0"},"reference-count":67,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,3,10]],"date-time":"2026-03-10T00:00:00Z","timestamp":1773100800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Nucleic Acids"],"published-print":{"date-parts":[[2026,6]]},"DOI":"10.1016\/j.omtn.2026.102901","type":"journal-article","created":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T17:08:37Z","timestamp":1773335317000},"page":"102901","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":1,"title":["AAV-mediated gene transfer of a novel microdystrophin ameliorates pathology and enhances muscle function in a mouse model of DMD"],"prefix":"10.1016","volume":"37","author":[{"given":"Lawrence","family":"Owusu","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"SunJung","family":"Kim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hiren","family":"Patel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven","family":"Foltz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gary N.Y.","family":"Chan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kwi Hye","family":"Kim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aaron","family":"Kopen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lin","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael W.","family":"Lawlor","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicholas","family":"Buss","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Justine J.","family":"Cunningham","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ye","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olivier","family":"Danos","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3869-9948","authenticated-orcid":false,"given":"Michele","family":"Fiscella","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2026.102901_bib1","doi-asserted-by":"crossref","first-page":"323","DOI":"10.1016\/0960-8966(91)90117-B","article-title":"Population frequencies of neuromuscular diseases--II. Amyotrophic lateral sclerosis (motor neurone disease)","volume":"1","author":"Emery","year":"1991","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.omtn.2026.102901_bib2","doi-asserted-by":"crossref","first-page":"304","DOI":"10.1002\/ana.23528","article-title":"Evidence-based path to newborn screening for Duchenne muscular dystrophy","volume":"71","author":"Mendell","year":"2012","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtn.2026.102901_bib3","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1002\/mus.23810","article-title":"Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy","volume":"48","author":"Mendell","year":"2013","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.omtn.2026.102901_bib4","doi-asserted-by":"crossref","first-page":"3011","DOI":"10.1038\/s41598-021-82725-z","article-title":"Newborn screening of duchenne muscular dystrophy specifically targeting deletions amenable to exon-skipping therapy","volume":"11","author":"Beckers","year":"2021","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtn.2026.102901_bib5","doi-asserted-by":"crossref","first-page":"50","DOI":"10.3390\/ijns8040050","article-title":"Newborn Screening for Duchenne Muscular Dystrophy: First Year Results of a Population-Based Pilot","volume":"8","author":"Hartnett","year":"2022","journal-title":"Int. J. Neonatal Screen."},{"key":"10.1016\/j.omtn.2026.102901_bib6","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1016\/0092-8674(87)90579-4","article-title":"Dystrophin: the protein product of the Duchenne muscular dystrophy locus","volume":"51","author":"Hoffman","year":"1987","journal-title":"Cell"},{"key":"10.1016\/j.omtn.2026.102901_bib7","series-title":"Molecular Mechanisms of Muscular Dystrophies","first-page":"14","article-title":"The Structure and Function of Dystrophin","author":"Abmayr","year":"2006"},{"key":"10.1016\/j.omtn.2026.102901_bib8","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.omtn.2026.102901_bib9","doi-asserted-by":"crossref","first-page":"653","DOI":"10.1016\/j.stem.2018.10.006","article-title":"Orienting Muscle Stem Cells for Regeneration in Homeostasis, Aging, and Disease","volume":"23","author":"Feige","year":"2018","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.omtn.2026.102901_bib10","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1038\/s41572-021-00248-3","article-title":"Duchenne muscular dystrophy","volume":"7","author":"Duan","year":"2021","journal-title":"Nat. Rev. Dis. Primers"},{"key":"10.1016\/j.omtn.2026.102901_bib11","doi-asserted-by":"crossref","first-page":"451","DOI":"10.1016\/S0140-6736(17)32160-8","article-title":"Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study","volume":"391","author":"McDonald","year":"2018","journal-title":"Lancet"},{"key":"10.1016\/j.omtn.2026.102901_bib12","first-page":"CD003725","article-title":"Corticosteroids for the treatment of Duchenne muscular dystrophy","volume":"2016","author":"Matthews","year":"2016","journal-title":"Cochrane Database Syst. Rev."},{"key":"10.1016\/j.omtn.2026.102901_bib13","doi-asserted-by":"crossref","first-page":"1592","DOI":"10.1056\/NEJM198906153202405","article-title":"Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy","volume":"320","author":"Mendell","year":"1989","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2026.102901_bib14","doi-asserted-by":"crossref","first-page":"1048","DOI":"10.1212\/WNL.0000000000001950","article-title":"Prednisone\/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study","volume":"85","author":"Bello","year":"2015","journal-title":"Neurology"},{"key":"10.1016\/j.omtn.2026.102901_bib15","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1089\/nat.2017.0682","article-title":"Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues","volume":"27","author":"Aartsma-Rus","year":"2017","journal-title":"Nucleic Acid Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib16","doi-asserted-by":"crossref","first-page":"213","DOI":"10.3233\/JND-180351","article-title":"Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy","volume":"6","author":"Khan","year":"2019","journal-title":"J. Neuromuscul. Dis."},{"key":"10.1016\/j.omtn.2026.102901_bib17","doi-asserted-by":"crossref","first-page":"e2270","DOI":"10.1212\/WNL.0000000000009233","article-title":"Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy","volume":"94","author":"Frank","year":"2020","journal-title":"Neurology"},{"key":"10.1016\/j.omtn.2026.102901_bib18","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1089\/nat.2016.0657","article-title":"FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga","volume":"27","author":"Aartsma-Rus","year":"2017","journal-title":"Nucleic Acid Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib19","doi-asserted-by":"crossref","first-page":"2689","DOI":"10.1016\/j.ymthe.2025.04.038","article-title":"Clinical applications of exon-skipping antisense oligonucleotides in neuromuscular diseases","volume":"33","author":"Torres-Masjoan","year":"2025","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib20","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.omtn.2026.102901_bib21","doi-asserted-by":"crossref","first-page":"733","DOI":"10.1089\/hum.2018.012","article-title":"Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients","volume":"29","author":"Duan","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib22","doi-asserted-by":"crossref","first-page":"2337","DOI":"10.1016\/j.ymthe.2018.07.011","article-title":"Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy","volume":"26","author":"Duan","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib23","series-title":"CENTER DIRECTOR DECISIONAL MEMO -ELEVIDYS","author":"Marks","year":"2023"},{"key":"10.1016\/j.omtn.2026.102901_bib24","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1016\/j.ymthe.2020.12.007","article-title":"Current Clinical Applications of In Vivo Gene Therapy with AAVs","volume":"29","author":"Mendell","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib25","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.omtn.2026.102901_bib26","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.omtn.2026.102901_bib27","doi-asserted-by":"crossref","first-page":"280","DOI":"10.1002\/ana.25951","article-title":"Very Low Residual Dystrophin Quantity Is Associated with Milder Dystrophinopathy","volume":"89","author":"de Feraudy","year":"2021","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtn.2026.102901_bib28","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.omtn.2026.102901_bib29","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.omtn.2026.102901_bib30","doi-asserted-by":"crossref","first-page":"623","DOI":"10.1016\/j.ymthe.2019.01.002","article-title":"Development of Novel Micro-dystrophins with Enhanced Functionality","volume":"27","author":"Ramos","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib31","doi-asserted-by":"crossref","first-page":"917","DOI":"10.1038\/s41573-023-00775-6","article-title":"Therapeutic approaches for Duchenne muscular dystrophy","volume":"22","author":"Roberts","year":"2023","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omtn.2026.102901_bib32","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1038\/s41434-025-00561-6","article-title":"AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects","volume":"32","author":"Chwalenia","year":"2025","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib33","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1002\/jgm.686","article-title":"Gene transfer into skeletal muscle using novel AAV serotypes","volume":"7","author":"Louboutin","year":"2005","journal-title":"J. Gene Med."},{"key":"10.1016\/j.omtn.2026.102901_bib34","doi-asserted-by":"crossref","first-page":"321","DOI":"10.1038\/nbt1073","article-title":"Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart","volume":"23","author":"Wang","year":"2005","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2026.102901_bib35","doi-asserted-by":"crossref","first-page":"1125","DOI":"10.1016\/S1474-4422(23)00313-7","article-title":"Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial","volume":"22","author":"Shieh","year":"2023","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.omtn.2026.102901_bib36","doi-asserted-by":"crossref","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.omtn.2026.102901_bib37","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1038\/6981","article-title":"Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences","volume":"17","author":"Li","year":"1999","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2026.102901_bib38","doi-asserted-by":"crossref","first-page":"1138","DOI":"10.1089\/hum.2020.325","article-title":"Dose-Dependent Microdystrophin Expression Enhancement in Cardiac Muscle by a Cardiac-Specific Regulatory Element","volume":"32","author":"Malerba","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib39","doi-asserted-by":"crossref","first-page":"848","DOI":"10.1038\/s41598-022-04892-x","article-title":"Therapeutic potential of highly functional codon-optimized microutrophin for muscle-specific expression","volume":"12","author":"Starikova","year":"2022","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtn.2026.102901_bib40","doi-asserted-by":"crossref","first-page":"47","DOI":"10.32607\/actanaturae.11063","article-title":"Muscle-Specific Promoters for Gene Therapy","volume":"13","author":"Skopenkova","year":"2021","journal-title":"Acta Nat."},{"key":"10.1016\/j.omtn.2026.102901_bib41","doi-asserted-by":"crossref","first-page":"1189","DOI":"10.1073\/pnas.81.4.1189","article-title":"X chromosome-linked muscular dystrophy (mdx) in the mouse","volume":"81","author":"Bulfield","year":"1984","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2026.102901_bib42","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1016\/S0092-8674(00)80533-4","article-title":"Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy","volume":"90","author":"Grady","year":"1997","journal-title":"Cell"},{"key":"10.1016\/j.omtn.2026.102901_bib43","doi-asserted-by":"crossref","first-page":"1153","DOI":"10.1002\/(SICI)1097-4598(199809)21:9<1153::AID-MUS6>3.0.CO;2-6","article-title":"Dystrophy and myogenesis in mdx diaphragm muscle","volume":"21","author":"Anderson","year":"1998","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.omtn.2026.102901_bib44","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1016\/j.omtm.2021.02.024","article-title":"Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice","volume":"21","author":"Hamm","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtn.2026.102901_bib45","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1002\/jgm.1602","article-title":"Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog","volume":"13","author":"Koo","year":"2011","journal-title":"J. Gene Med."},{"key":"10.1016\/j.omtn.2026.102901_bib46","doi-asserted-by":"crossref","first-page":"3515","DOI":"10.1016\/j.ymthe.2022.09.015","article-title":"AAV vectors: The Rubik's cube of human gene therapy","volume":"30","author":"Pupo","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib47","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1002\/mus.24679","article-title":"T2 relaxation times are increased in Skeletal muscle of DMD but not BMD patients","volume":"53","author":"Wokke","year":"2016","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.omtn.2026.102901_bib48","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.omtn.2026.102901_bib49","doi-asserted-by":"crossref","DOI":"10.3389\/fcell.2023.1167762","article-title":"Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy","volume":"11","author":"Mendell","year":"2023","journal-title":"Front. Cell Dev. Biol."},{"key":"10.1016\/j.omtn.2026.102901_bib50","doi-asserted-by":"crossref","first-page":"29","DOI":"10.3233\/JND-150113","article-title":"Current Translational Research and Murine Models For Duchenne Muscular Dystrophy","volume":"3","author":"Rodrigues","year":"2016","journal-title":"J. Neuromuscul. Dis."},{"key":"10.1016\/j.omtn.2026.102901_bib51","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1007\/BF00687043","article-title":"Skeletal muscle pathology in X chromosome-linked muscular dystrophy (mdx) mouse","volume":"69","author":"Tanabe","year":"1986","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.omtn.2026.102901_bib52","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1016\/j.yjmcc.2014.01.009","article-title":"Low dystrophin levels in heart can delay heart failure in mdx mice","volume":"69","author":"van Putten","year":"2014","journal-title":"J. Mol. Cell. Cardiol."},{"key":"10.1016\/j.omtn.2026.102901_bib53","first-page":"440","article-title":"Normal and dystrophin-deficient muscle fibers in carriers of the gene for Duchenne muscular dystrophy","volume":"133","author":"Bonilla","year":"1988","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.omtn.2026.102901_bib54","doi-asserted-by":"crossref","first-page":"578","DOI":"10.1002\/jcsm.12506","article-title":"Uniform sarcolemmal dystrophin expression is required to prevent extracellular microRNA release and improve dystrophic pathology","volume":"11","author":"van Westering","year":"2020","journal-title":"J. Cachexia Sarcopenia Muscle"},{"key":"10.1016\/j.omtn.2026.102901_bib55","first-page":"625","article-title":"Sutructural Protein","volume":"3","author":"Nelson","year":"2021"},{"key":"10.1016\/j.omtn.2026.102901_bib56","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.omtn.2026.102901_bib57","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2025.101519","article-title":"Enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain","volume":"33","author":"Foltz","year":"2025","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtn.2026.102901_bib58","doi-asserted-by":"crossref","first-page":"543","DOI":"10.1016\/S0892-0362(03)00073-4","article-title":"Factors affecting grip strength testing","volume":"25","author":"Maurissen","year":"2003","journal-title":"Neurotoxicol. Teratol."},{"key":"10.1016\/j.omtn.2026.102901_bib59","doi-asserted-by":"crossref","first-page":"502","DOI":"10.1016\/j.nmd.2009.05.003","article-title":"Developing standard procedures for murine and canine efficacy studies of DMD therapeutics: report of two expert workshops on \u201cPre-clinical testing for Duchenne dystrophy\u201d: Washington DC, October 27th-28th 2007 and Z\u00fcrich, June 30th-July 1st 2008","volume":"19","author":"Nagaraju","year":"2009","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.omtn.2026.102901_bib60","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1016\/0165-0270(95)00049-6","article-title":"Quantitative measurement of muscle strength in the mouse","volume":"62","author":"Smith","year":"1995","journal-title":"J. Neurosci. Methods"},{"key":"10.1016\/j.omtn.2026.102901_bib61","doi-asserted-by":"crossref","first-page":"3710","DOI":"10.1073\/pnas.90.8.3710","article-title":"Dystrophin protects the sarcolemma from stresses developed during muscle contraction","volume":"90","author":"Petrof","year":"1993","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2026.102901_bib62","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1186\/1471-2202-11-92","article-title":"Automated quantitative gait analysis in animal models of movement disorders","volume":"11","author":"Vandeputte","year":"2010","journal-title":"BMC Neurosci."},{"key":"10.1016\/j.omtn.2026.102901_bib63","doi-asserted-by":"crossref","first-page":"340","DOI":"10.1016\/j.bbr.2016.04.044","article-title":"High-speed video gait analysis reveals early and characteristic locomotor phenotypes in mouse models of neurodegenerative movement disorders","volume":"311","author":"Preisig","year":"2016","journal-title":"Behav. Brain Res."},{"key":"10.1016\/j.omtn.2026.102901_bib64","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1089\/nat.2019.0824","article-title":"Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in mdx Mice","volume":"30","author":"Datson","year":"2020","journal-title":"Nucleic Acid Ther."},{"key":"10.1016\/j.omtn.2026.102901_bib65","doi-asserted-by":"crossref","first-page":"1209","DOI":"10.2217\/bmm-2019-0242","article-title":"Orthogonal analysis of dystrophin protein and mRNA as a surrogate outcome for drug development","volume":"13","author":"Uaesoontrachoon","year":"2019","journal-title":"Biomark. Med."},{"key":"10.1016\/j.omtn.2026.102901_bib66","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.abo1815","article-title":"Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy","volume":"15","author":"Birch","year":"2023","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtn.2026.102901_bib67","doi-asserted-by":"crossref","DOI":"10.3389\/fphys.2023.1166206","article-title":"Prolonged voluntary wheel running reveals unique adaptations in mdx mice treated with microdystrophin constructs \u00b1 the nNOS-binding site","volume":"14","author":"Hamm","year":"2023","journal-title":"Front. Physiol."}],"container-title":["Molecular Therapy Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253126000727?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253126000727?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,11,10]],"date-time":"2026-11-10T01:16:18Z","timestamp":1794273378000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253126000727"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,6]]},"references-count":67,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2026,6]]}},"alternative-id":["S2162253126000727"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2026.102901","relation":{},"ISSN":["2162-2531"],"issn-type":[{"value":"2162-2531","type":"print"}],"subject":[],"published":{"date-parts":[[2026,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"AAV-mediated gene transfer of a novel microdystrophin ameliorates pathology and enhances muscle function in a mouse model of DMD","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2026.102901","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Author(s). Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"102901"}}