{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,27]],"date-time":"2026-10-27T22:03:13Z","timestamp":1793138593840,"version":"4.5.0"},"reference-count":203,"publisher":"Elsevier BV","issue":"5","license":[{"start":{"date-parts":[[2025,5,1]],"date-time":"2025-05-01T00:00:00Z","timestamp":1746057600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,5,1]],"date-time":"2025-05-01T00:00:00Z","timestamp":1746057600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,4,5]],"date-time":"2025-04-05T00:00:00Z","timestamp":1743811200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000005","name":"US Department of Defense","doi-asserted-by":"publisher","award":["MD220097"],"award-info":[{"award-number":["MD220097"]}],"id":[{"id":"10.13039\/100000005","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"}]},{"DOI":"10.13039\/100014269","name":"Philippe Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014269","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000069","name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases","doi-asserted-by":"publisher","award":["P50AR065139"],"award-info":[{"award-number":["P50AR065139"]}],"id":[{"id":"10.13039\/100000069","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000069","name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases","doi-asserted-by":"publisher","award":["R01AR040864"],"award-info":[{"award-number":["R01AR040864"]}],"id":[{"id":"10.13039\/100000069","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007393","name":"Association Fran\u00e7aise contre les Myopathies","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007393","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100013465","name":"AFM-T\u00e9l\u00e9thon","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013465","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100023456","name":"Defeat Duchenne Canada","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100023456","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100007492","name":"Fondation Bettencourt Schueller","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100007492","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2025,5]]},"DOI":"10.1016\/j.ymthe.2025.03.065","type":"journal-article","created":{"date-parts":[[2025,4,2]],"date-time":"2025-04-02T23:30:19Z","timestamp":1743636619000},"page":"2035-2051","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":38,"title":["The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy"],"prefix":"10.1016","volume":"33","author":[{"given":"Niclas E.","family":"Bengtsson","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hichem","family":"Tasfaout","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.2025.03.065_bib1","doi-asserted-by":"crossref","first-page":"842","DOI":"10.1038\/316842a0","article-title":"Detection of deletions spanning the Duchenne muscular dystrophy locus using a tightly linked DNA segment","volume":"316","author":"Monaco","year":"1985","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib2","doi-asserted-by":"crossref","first-page":"646","DOI":"10.1038\/323646a0","article-title":"Isolation of candidate cDNA clones for portions of the Duchenne muscular dystrophy gene","volume":"323","author":"Monaco","year":"1986","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib3","doi-asserted-by":"crossref","first-page":"434","DOI":"10.1038\/328434a0","article-title":"A cDNA clone from the Duchenne\/Becker muscular dystrophy gene","volume":"328","author":"Burghes","year":"1987","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib4","series-title":"Duchenne Muscular Dystrophy","author":"Emery","year":"2003"},{"key":"10.1016\/j.ymthe.2025.03.065_bib5","series-title":"Current Neurology","first-page":"65","article-title":"Duchenne Muscular Dystrophy","author":"Chamberlain","year":"1990"},{"key":"10.1016\/j.ymthe.2025.03.065_bib6","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1016\/0092-8674(87)90504-6","article-title":"Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals","volume":"50","author":"Koenig","year":"1987","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2025.03.065_bib7","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1016\/0092-8674(88)90383-2","article-title":"The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein","volume":"53","author":"Koenig","year":"1988","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2025.03.065_bib8","doi-asserted-by":"crossref","first-page":"1657","DOI":"10.1002\/humu.21114","article-title":"Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort","volume":"30","author":"Flanigan","year":"2009","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.ymthe.2025.03.065_bib9","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/0888-7543(88)90113-9","article-title":"An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus","volume":"2","author":"Monaco","year":"1988","journal-title":"Genomics"},{"key":"10.1016\/j.ymthe.2025.03.065_bib10","doi-asserted-by":"crossref","first-page":"755","DOI":"10.1126\/science.3055295","article-title":"Frame-shift deletions in patients with Duchenne and Becker muscular dystrophy","volume":"242","author":"Malhotra","year":"1988","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.065_bib11","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.2025.03.065_bib12","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1212\/WNL.39.4.465","article-title":"Molecular and clinical correlations of deletions leading to Duchenne and Becker muscular dystrophies","volume":"39","author":"Baumbach","year":"1989","journal-title":"Neurology"},{"key":"10.1016\/j.ymthe.2025.03.065_bib13","first-page":"54","article-title":"Exploring the molecular basis for variability among patients with Becker muscular dystrophy: dystrophin gene and protein studies","volume":"49","author":"Beggs","year":"1991","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib14","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.2025.03.065_bib15","doi-asserted-by":"crossref","first-page":"860","DOI":"10.1136\/jmg.28.12.860","article-title":"Becker muscular dystrophy patient with a large intragenic dystrophin deletion: Implications for functional minigenes and gene therapy","volume":"28","author":"Love","year":"1991","journal-title":"J.\u00a0Med. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib16","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 use for gene therapy","volume":"3","author":"Passos-Bueno","year":"1994","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib17","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1016\/j.nmd.2024.04.004","article-title":"Long-term clinical follow-up of a family with Becker muscular dystrophy associated with a large deletion in the DMD gene","volume":"39","author":"Davies","year":"2024","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.ymthe.2025.03.065_bib18","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1038\/322073a0","article-title":"Analysis of deletions in DNA from patients with Becker and Duchenne muscular dystrophy","volume":"322","author":"Kunkel","year":"1986","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib19","doi-asserted-by":"crossref","first-page":"11141","DOI":"10.1093\/nar\/16.23.11141","article-title":"Deletion screening of the Duchenne muscular dystrophy locus via multiplex DNA amplification","volume":"16","author":"Chamberlain","year":"1988","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2025.03.065_bib20","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1007\/BF00205170","article-title":"Detection of 98% of DMD\/BMD gene deletions by polymerase chain reaction","volume":"86","author":"Beggs","year":"1990","journal-title":"Hum. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib21","doi-asserted-by":"crossref","first-page":"2609","DOI":"10.1001\/jama.1992.03480190051030","article-title":"Diagnosis of Duchenne and Becker muscular dystrophies by polymerase chain reaction: A multicenter study","volume":"267","author":"Chamberlain","year":"1992","journal-title":"JAMA"},{"key":"10.1016\/j.ymthe.2025.03.065_bib22","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.ymthe.2025.03.065_bib23","doi-asserted-by":"crossref","first-page":"1292","DOI":"10.1073\/pnas.86.4.1292","article-title":"Recovery of induced mutations for X chromosome-linked muscular dystrophy in mice","volume":"86","author":"Chapman","year":"1989","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2025.03.065_bib24","doi-asserted-by":"crossref","first-page":"1578","DOI":"10.1126\/science.2662404","article-title":"The molecular basis of muscular dystrophy in the mdx mouse: a point mutation","volume":"244","author":"Sicinski","year":"1989","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.065_bib25","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1038\/ng0593-87","article-title":"New mdx mutation disrupts expression of muscle and nonmuscle isoforms of dystrophin","volume":"4","author":"Cox","year":"1993","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib26","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.2025.03.065_bib27","doi-asserted-by":"crossref","first-page":"466","DOI":"10.1038\/333466a0","article-title":"The Duchenne muscular dystrophy gene product is localized in sarcolemma of human skeletal muscle","volume":"333","author":"Zubrzycka-Gaarn","year":"1988","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib28","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.ymthe.2025.03.065_bib29","doi-asserted-by":"crossref","first-page":"1363","DOI":"10.1056\/NEJM198805263182104","article-title":"Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy","volume":"318","author":"Hoffman","year":"1988","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib30","doi-asserted-by":"crossref","first-page":"1121","DOI":"10.1016\/0092-8674(91)90035-W","article-title":"Membrane organization of the dystrophin-glycoprotein complex","volume":"66","author":"Ervasti","year":"1991","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2025.03.065_bib31","doi-asserted-by":"crossref","first-page":"696","DOI":"10.1038\/355696a0","article-title":"Primary structure of dystrophin-associated glycoproteins linking dystrophin to the extracellular matrix","volume":"355","author":"Ibraghimov-Beskrovnaya","year":"1992","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib32","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1016\/0092-8674(95)90344-5","article-title":"Three muscular dystrophies: loss of cytoskeleton-extracellular matrix linkage","volume":"80","author":"Campbell","year":"1995","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2025.03.065_bib33","doi-asserted-by":"crossref","first-page":"1252","DOI":"10.1038\/s41586-024-08310-2","article-title":"Structure and assembly of the dystrophin glycoprotein complex","volume":"637","author":"Wan","year":"2025","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib34","doi-asserted-by":"crossref","first-page":"1261","DOI":"10.1038\/s41586-024-08324-w","article-title":"Native DGC structure rationalizes muscular dystrophy-causing mutations","volume":"637","author":"Liu","year":"2025","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib35","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.2025.03.065_bib36","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.2025.03.065_bib37","doi-asserted-by":"crossref","first-page":"1325","DOI":"10.1083\/jcb.145.6.1325","article-title":"alpha-Dystroglycan is a laminin receptor involved in extracellular matrix assembly on myotubes and muscle cell viability","volume":"145","author":"Montanaro","year":"1999","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib38","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.2025.03.065_bib39","doi-asserted-by":"crossref","first-page":"334","DOI":"10.1038\/349334a0","article-title":"Expression of recombinant dystrophin and its localization to the cell membrane","volume":"349","author":"Lee","year":"1991","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib40","first-page":"62","article-title":"The muscle creatine kinase gene is regulated by multiple upstream elements, including a muscle-specific enhancer","volume":"8","author":"Jaynes","year":"1988","journal-title":"Mol. Cell. Biol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib41","doi-asserted-by":"crossref","first-page":"725","DOI":"10.1038\/364725a0","article-title":"Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity [see comments]","volume":"364","author":"Cox","year":"1993","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib42","doi-asserted-by":"crossref","first-page":"S23","DOI":"10.1016\/S0960-8966(02)00078-0","article-title":"Viral vectors for gene transfer of micro-mini-or full-length dystrophin","volume":"12","author":"Scott","year":"2002","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.ymthe.2025.03.065_bib43","doi-asserted-by":"crossref","first-page":"1465","DOI":"10.1126\/science.1690918","article-title":"Direct gene transfer into mouse muscle in\u00a0vivo","volume":"247","author":"Wolff","year":"1990","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.065_bib44","doi-asserted-by":"crossref","first-page":"693","DOI":"10.1203\/00006450-199506000-00004","article-title":"Expression of deletion-containing dystrophins in mdx muscle: implications for gene therapy and dystrophin function","volume":"37","author":"Fritz","year":"1995","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.ymthe.2025.03.065_bib45","doi-asserted-by":"crossref","first-page":"815","DOI":"10.1038\/352815a0","article-title":"Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs","volume":"352","author":"Acsadi","year":"1991","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib46","doi-asserted-by":"crossref","first-page":"1065","DOI":"10.1089\/hum.2004.15.1065","article-title":"Phase I Study of Dystrophin Plasmid-Based Gene Therapy in Duchenne\/Becker Muscular Dystrophy","volume":"15","author":"Romero","year":"2004","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib47","doi-asserted-by":"crossref","first-page":"626","DOI":"10.1172\/JCI115902","article-title":"Widespread long-term gene transfer to mouse skeletal muscles and heart","volume":"90","author":"Stratford-Perricaudet","year":"1992","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2025.03.065_bib48","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1038\/361647a0","article-title":"Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice","volume":"361","author":"Ragot","year":"1993","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib49","doi-asserted-by":"crossref","first-page":"579","DOI":"10.1093\/hmg\/3.4.579","article-title":"A differential efficiency of adenovirus-mediated in\u00a0vivo gene transfer into skeletal muscle cells of different maturity","volume":"3","author":"Acsadi","year":"1994","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib50","doi-asserted-by":"crossref","first-page":"5222","DOI":"10.1128\/JVI.75.11.5222-5229.2001","article-title":"Biology of E1-deleted adenovirus vectors in nonhuman primate muscle","volume":"75","author":"Zoltick","year":"2001","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib51","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1006\/mthe.2001.0305","article-title":"A death in the laboratory: the politics of the Gelsinger aftermath","volume":"3","author":"Carmen","year":"2001","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib52","doi-asserted-by":"crossref","first-page":"3854","DOI":"10.1073\/pnas.92.9.3854","article-title":"Rescue, propagation, and partial purification of a helper virus-dependent adenovirus vector","volume":"92","author":"Mitani","year":"1995","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2025.03.065_bib53","doi-asserted-by":"crossref","first-page":"13565","DOI":"10.1073\/pnas.93.24.13565","article-title":"A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal","volume":"93","author":"Parks","year":"1996","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2025.03.065_bib54","doi-asserted-by":"crossref","first-page":"913","DOI":"10.1093\/hmg\/5.7.913","article-title":"Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells","volume":"5","author":"Kumar-Singh","year":"1996","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib55","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1038\/7439","article-title":"Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector","volume":"5","author":"Greelish","year":"1999","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib56","doi-asserted-by":"crossref","first-page":"12979","DOI":"10.1073\/pnas.202300099","article-title":"Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin","volume":"99","author":"DelloRusso","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2025.03.065_bib57","doi-asserted-by":"crossref","first-page":"717","DOI":"10.1093\/hmg\/2.6.717","article-title":"Direct retroviral-mediated transfer of a dystrophin minigene into mdx mouse muscle in\u00a0vivo","volume":"2","author":"Dunckley","year":"1993","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib58","doi-asserted-by":"crossref","first-page":"1719","DOI":"10.1093\/hmg\/11.15.1719","article-title":"Stable micro-dystrophin gene transfer using an integrating adeno-retroviral hybrid vector ameliorates the dystrophic pathology in mdx mouse muscle","volume":"11","author":"Roberts","year":"2002","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib59","doi-asserted-by":"crossref","first-page":"1441","DOI":"10.1089\/104303403769211655","article-title":"Correction of the dystrophic phenotype by in\u00a0vivo targeting of muscle progenitor cells","volume":"14","author":"Kobinger","year":"2003","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib60","doi-asserted-by":"crossref","first-page":"206","DOI":"10.1038\/mt.2009.253","article-title":"Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression","volume":"18","author":"Kimura","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib61","doi-asserted-by":"crossref","first-page":"2062","DOI":"10.1016\/j.cell.2023.03.033","article-title":"Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery","volume":"186","author":"Hindi","year":"2023","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2025.03.065_bib62","doi-asserted-by":"crossref","first-page":"14082","DOI":"10.1073\/pnas.93.24.14082","article-title":"Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein","volume":"93","author":"Kessler","year":"1996","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2025.03.065_bib63","doi-asserted-by":"crossref","first-page":"8098","DOI":"10.1128\/jvi.70.11.8098-8108.1996","article-title":"Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector","volume":"70","author":"Xiao","year":"1996","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib64","first-page":"97","article-title":"Use of adeno-associated virus as a general transduction vector for mammalian cells","volume":"158","author":"Muzyczka","year":"1992","journal-title":"Curr. Top. Microbiol. Immunol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib65","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.2025.03.065_bib66","doi-asserted-by":"crossref","first-page":"1095","DOI":"10.1093\/hmg\/11.9.1095","article-title":"Expression of Dp260 in muscle tethers the actin cytoskeleton to the dystrophin-glycoprotein complex and partially prevents dystrophy","volume":"11","author":"Warner","year":"2002","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib67","doi-asserted-by":"crossref","first-page":"1251","DOI":"10.1093\/hmg\/4.8.1251","article-title":"Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice","volume":"4","author":"Phelps","year":"1995","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib68","doi-asserted-by":"crossref","first-page":"1245","DOI":"10.1093\/hmg\/4.8.1245","article-title":"Expression of human full-length and minidystrophin in transgenic mdx mice: implications for gene therapy of Duchenne muscular dystrophy","volume":"4","author":"Wells","year":"1995","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib69","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.2025.03.065_bib70","doi-asserted-by":"crossref","first-page":"1807","DOI":"10.1093\/hmg\/11.16.1807","article-title":"Spectrin-like repeats from dystrophin and alpha-actinin-2 are not functionally interchangeable","volume":"11","author":"Harper","year":"2002","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib71","doi-asserted-by":"crossref","first-page":"1725","DOI":"10.1093\/hmg\/3.10.1725","article-title":"Prevention of dystrophic pathology in mdx mice by a truncated dystrophin isoform","volume":"3","author":"Rafael","year":"1994","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib72","doi-asserted-by":"crossref","first-page":"1399","DOI":"10.1083\/jcb.150.6.1399","article-title":"Assembly of the dystrophin-associated protein complex does not require the dystrophin COOH-terminal domain","volume":"150","author":"Crawford","year":"2000","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib73","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.2025.03.065_bib75","first-page":"2855","article-title":"Transcriptional regulation of the muscle creatine kinase gene and regulated expression in transfected mouse myoblasts","volume":"6","author":"Jaynes","year":"1986","journal-title":"Mol. Cell. Biol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib76","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.2025.03.065_bib77","doi-asserted-by":"crossref","first-page":"1265","DOI":"10.1016\/S0006-291X(02)00362-5","article-title":"Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene","volume":"293","author":"Sakamoto","year":"2002","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib78","doi-asserted-by":"crossref","first-page":"851","DOI":"10.1089\/hum.2008.058","article-title":"Adeno-Associated\u00a0Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice","volume":"19","author":"Bostick","year":"2008","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib79","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.2025.03.065_bib80","doi-asserted-by":"crossref","first-page":"2105","DOI":"10.1093\/hmg\/ddm158","article-title":"Functional capacity of dystrophins carrying deletions in the N-terminal actin-binding domain","volume":"16","author":"Banks","year":"2007","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib81","doi-asserted-by":"crossref","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.2025.03.065_bib82","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.ymthe.2025.03.065_bib83","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.2025.03.065_bib84","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1006\/mthe.2000.0089","article-title":"Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors","volume":"2","author":"Hauser","year":"2000","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib85","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.2025.03.065_bib86","doi-asserted-by":"crossref","first-page":"525","DOI":"10.1006\/mthe.2001.0496","article-title":"Immune evasion by muscle-specific gene expression in dystrophic muscle","volume":"4","author":"Hartigan-O'Connor","year":"2001","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib87","doi-asserted-by":"crossref","first-page":"777","DOI":"10.1089\/10430340050015671","article-title":"Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-beta-gamma-or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors","volume":"11","author":"Stedman","year":"2000","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib88","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1002\/ana.21732","article-title":"Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins","volume":"66","author":"Mendell","year":"2009","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib89","doi-asserted-by":"crossref","first-page":"629","DOI":"10.1002\/ana.22251","article-title":"Sustained alpha-sarcoglycan gene 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.2025.03.065_bib90","doi-asserted-by":"crossref","first-page":"1429","DOI":"10.1056\/NEJMoa1000228","article-title":"Dystrophin immunity in Duchenne's muscular dystrophy","volume":"363","author":"Mendell","year":"2010","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib91","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1038\/mt.2011.237","article-title":"Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector","volume":"20","author":"Bowles","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib92","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1016\/j.pediatrneurol.2024.01.003","article-title":"Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy","volume":"153","author":"Mendell","year":"2024","journal-title":"Pediatr. Neurol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib93","doi-asserted-by":"crossref","first-page":"1122","DOI":"10.1001\/jamaneurol.2020.1484","article-title":"Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial","volume":"77","author":"Mendell","year":"2020","journal-title":"JAMA Neurol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib94","first-page":"553","article-title":"IGNITE DMD Study of SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy: Long-Term Outcomes and Biomarker Update","volume":"30","author":"Morris","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib95","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.ymthe.2025.03.065_bib96","doi-asserted-by":"crossref","first-page":"R102","DOI":"10.1093\/hmg\/ddz128","article-title":"AAV-based gene therapies for the muscular dystrophies","volume":"28","author":"Crudele","year":"2019","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib97","doi-asserted-by":"crossref","first-page":"1379","DOI":"10.1089\/hum.2011.020","article-title":"Delivery of AAV2\/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of alpha1-syntrophin and alpha-dystrobrevin in skeletal muscles of mdx mice","volume":"22","author":"Koo","year":"2011","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib98","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.2025.03.065_bib99","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.ymthe.2025.03.065_bib100","doi-asserted-by":"crossref","first-page":"1854","DOI":"10.1161\/01.CIR.87.6.1854","article-title":"X-linked dilated cardiomyopathy: Molecular genetic evidence of linkage to the Duchenne muscular dystrophy (dystrophin) gene at the Xp21 locus","volume":"87","author":"Towbin","year":"1993","journal-title":"Circulation"},{"key":"10.1016\/j.ymthe.2025.03.065_bib101","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1016\/j.tcm.2009.04.006","article-title":"Cardiac consequences to skeletal muscle-centric therapeutics for Duchenne muscular dystrophy","volume":"19","author":"Townsend","year":"2009","journal-title":"Trends Cardiovasc. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib102","doi-asserted-by":"crossref","first-page":"832","DOI":"10.1038\/mt.2008.52","article-title":"Emergent dilated cardiomyopathy caused by targeted repair of dystrophic skeletal muscle","volume":"16","author":"Townsend","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib103","doi-asserted-by":"crossref","first-page":"1203","DOI":"10.1056\/NEJMoa2307798","article-title":"Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy","volume":"389","author":"Lek","year":"2023","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib104","doi-asserted-by":"crossref","first-page":"884","DOI":"10.1016\/j.nmd.2023.09.008","article-title":"261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands","volume":"33","author":"Servais","year":"2023","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.ymthe.2025.03.065_bib105","doi-asserted-by":"crossref","first-page":"2294","DOI":"10.1056\/NEJMc2212912","article-title":"Dystrophin Immunity after Gene Therapy for Duchenne's Muscular Dystrophy","volume":"388","author":"Bonnemann","year":"2023","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib106","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2022.999021","article-title":"Pre-existing humoral immunity and complement pathway contribute to immunogenicity of adeno-associated virus (AAV) vector in human blood","volume":"13","author":"Smith","year":"2022","journal-title":"Front. Immunol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib107","doi-asserted-by":"crossref","first-page":"1944","DOI":"10.1038\/mt.2008.207","article-title":"A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs","volume":"16","author":"Yue","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib108","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.ymthe.2025.03.065_bib109","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1016\/j.omtm.2023.05.017","article-title":"Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy","volume":"30","author":"Le Guiner","year":"2023","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.03.065_bib110","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.ymthe.2025.03.065_bib111","doi-asserted-by":"crossref","first-page":"192","DOI":"10.1038\/s41586-024-07710-8","article-title":"Split intein-mediated protein trans-splicing to express large dystrophins","volume":"632","author":"Tasfaout","year":"2024","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib112","doi-asserted-by":"crossref","first-page":"5432","DOI":"10.1038\/s41467-020-19230-w","article-title":"Identification of a myotropic AAV by massively parallel in\u00a0vivo evaluation of barcoded capsid variants","volume":"11","author":"Weinmann","year":"2020","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib113","doi-asserted-by":"crossref","first-page":"4919","DOI":"10.1016\/j.cell.2021.08.028","article-title":"Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species","volume":"184","author":"Tabebordbar","year":"2021","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2025.03.065_bib114","doi-asserted-by":"crossref","first-page":"5947","DOI":"10.1038\/s41467-022-33745-4","article-title":"Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing","volume":"13","author":"Gonzalez","year":"2022","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib115","doi-asserted-by":"crossref","first-page":"1539","DOI":"10.1038\/mt.2008.149","article-title":"Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin\/utrophin-deficient mice","volume":"16","author":"Odom","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib116","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1016\/j.omtm.2018.10.005","article-title":"Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2\/mdx Mice","volume":"11","author":"Kennedy","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.03.065_bib117","doi-asserted-by":"crossref","first-page":"1505","DOI":"10.1038\/s41591-019-0594-0","article-title":"Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models","volume":"25","author":"Song","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib118","first-page":"20","article-title":"Deimmunized micro-dystrophin vectors blunt patient immunity in vitro & restore cardiac functional deficits in vivo in mdx4cv DMD mice","volume":"30","author":"Odom","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib119","doi-asserted-by":"crossref","first-page":"4481","DOI":"10.1016\/j.csbj.2022.08.034","article-title":"Obtaining protein foldability information from computational models of AlphaFold2 and RoseTTAFold","volume":"20","author":"Liu","year":"2022","journal-title":"Comput. Struct. Biotechnol. J."},{"key":"10.1016\/j.ymthe.2025.03.065_bib120","doi-asserted-by":"crossref","first-page":"825","DOI":"10.1021\/acs.biochem.0c00912","article-title":"Modeling Immunity with Rosetta: Methods for Antibody and Antigen Design","volume":"60","author":"Schoeder","year":"2021","journal-title":"Biochemistry"},{"key":"10.1016\/j.ymthe.2025.03.065_bib121","doi-asserted-by":"crossref","first-page":"66","DOI":"10.1016\/S0076-6879(04)83004-0","article-title":"Protein structure prediction using Rosetta","volume":"383","author":"Rohl","year":"2004","journal-title":"Methods Enzymol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib122","doi-asserted-by":"crossref","first-page":"737","DOI":"10.1089\/hum.2017.144","article-title":"Cardiac-Specific Expression of DeltaH2-R15 Mini-Dystrophin Normalized All Electrocardiogram Abnormalities and the End-Diastolic Volume in a 23-Month-Old Mouse Model of Duchenne Dilated Cardiomyopathy","volume":"29","author":"Wasala","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib123","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.abn4704","article-title":"Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders","volume":"8","author":"El Andari","year":"2022","journal-title":"Sci. Adv."},{"key":"10.1016\/j.ymthe.2025.03.065_bib124","doi-asserted-by":"crossref","first-page":"7965","DOI":"10.1038\/s41467-024-52002-4","article-title":"An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across species","volume":"15","author":"Vu Hong","year":"2024","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib125","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2022.1001263","article-title":"rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis","volume":"13","author":"Shen","year":"2022","journal-title":"Front. Immunol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib146","doi-asserted-by":"crossref","first-page":"1435","DOI":"10.1038\/nbt1153","article-title":"Efficient in\u00a0vivo gene expression by trans-splicing adeno-associated viral vectors","volume":"23","author":"Lai","year":"2005","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib147","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1089\/hum.2011.131","article-title":"Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma","volume":"23","author":"Zhang","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib126","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1089\/hum.2013.164","article-title":"Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice","volume":"25","author":"Koo","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib127","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.2025.03.065_bib128","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1089\/hum.2017.095","article-title":"Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model","volume":"29","author":"Kodippili","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib129","doi-asserted-by":"crossref","DOI":"10.3390\/ijms241411421","article-title":"Assessment of Therapeutic Potential of a Dual AAV Approach for Duchenne Muscular Dystrophy","volume":"24","author":"Albini","year":"2023","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ymthe.2025.03.065_bib130","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.2025.03.065_bib131","doi-asserted-by":"crossref","first-page":"762","DOI":"10.1126\/science.adp8179","article-title":"Ribozyme-activated mRNA trans-ligation enables large gene delivery to treat muscular dystrophies","volume":"386","author":"Lindley","year":"2024","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.065_bib132","doi-asserted-by":"crossref","first-page":"958","DOI":"10.1089\/hum.2008.009","article-title":"Protein trans-splicing as a means for viral vector-mediated in\u00a0vivo gene therapy","volume":"19","author":"Li","year":"2008","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib133","article-title":"Expression of full-length dystrophin reverses muscular dystrophy defects in young and old mdx4cv mice.","author":"Tasfaout","year":"2025","journal-title":"manuscript in revision"},{"key":"10.1016\/j.ymthe.2025.03.065_bib134","article-title":"Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice","volume":"15","author":"Zhou","year":"2024","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib135","doi-asserted-by":"crossref","DOI":"10.3390\/ijms251910444","article-title":"In Silico Structural Prediction for the Generation of Novel Performant Midi-Dystrophins Based on Intein-Mediated Dual AAV Approach","volume":"25","author":"Palmieri","year":"2024","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ymthe.2025.03.065_bib136","doi-asserted-by":"crossref","first-page":"306","DOI":"10.1038\/nm0397-306","article-title":"Recombinant adeno-associated virus for muscle directed gene therapy","volume":"3","author":"Fisher","year":"1997","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib137","doi-asserted-by":"crossref","first-page":"5804","DOI":"10.1073\/pnas.94.11.5804","article-title":"Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus","volume":"94","author":"Herzog","year":"1997","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2025.03.065_bib138","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1038\/ng1094-148","article-title":"Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain","volume":"8","author":"Kaplitt","year":"1994","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib139","doi-asserted-by":"crossref","first-page":"8568","DOI":"10.1128\/JVI.72.11.8568-8577.1998","article-title":"Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue","volume":"72","author":"Duan","year":"1998","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib140","doi-asserted-by":"crossref","first-page":"9468","DOI":"10.1128\/JVI.73.11.9468-9477.1999","article-title":"Concatamerization of adeno-associated virus circular genomes occurs through intermolecular recombination","volume":"73","author":"Yang","year":"1999","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib141","doi-asserted-by":"crossref","first-page":"6716","DOI":"10.1073\/pnas.97.12.6716","article-title":"Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy","volume":"97","author":"Yan","year":"2000","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2025.03.065_bib142","doi-asserted-by":"crossref","first-page":"599","DOI":"10.1038\/75087","article-title":"Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization","volume":"6","author":"Sun","year":"2000","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib143","doi-asserted-by":"crossref","first-page":"527","DOI":"10.1038\/75390","article-title":"Increasing the size of rAAV-mediated expression cassettes in\u00a0vivo by intermolecular joining of two complementary vectors","volume":"18","author":"Nakai","year":"2000","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib144","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1006\/mthe.2001.0456","article-title":"Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison","volume":"4","author":"Duan","year":"2001","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib145","doi-asserted-by":"crossref","first-page":"716","DOI":"10.1006\/mthe.2002.0607","article-title":"Expression of human factor VIII by splicing between dimerized AAV vectors","volume":"5","author":"Chao","year":"2002","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib148","doi-asserted-by":"crossref","first-page":"124","DOI":"10.1038\/sj.mt.6300322","article-title":"A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner","volume":"16","author":"Ghosh","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib149","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1089\/hum.2006.128","article-title":"Hybrid adeno-associated virus bearing nonhomologous inverted terminal repeats enhances dual-vector reconstruction of minigenes in\u00a0vivo","volume":"18","author":"Yan","year":"2007","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib150","doi-asserted-by":"crossref","first-page":"6244","DOI":"10.1038\/ncomms7244","article-title":"Multiplex CRISPR\/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy","volume":"6","author":"Ousterout","year":"2015","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib151","doi-asserted-by":"crossref","first-page":"1184","DOI":"10.1126\/science.1254445","article-title":"Prevention of muscular dystrophy in mice by CRISPR\/Cas9-mediated editing of germline DNA","volume":"345","author":"Long","year":"2014","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.065_bib152","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/j.stemcr.2014.10.013","article-title":"Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9","volume":"4","author":"Li","year":"2015","journal-title":"Stem Cel. Rep."},{"key":"10.1016\/j.ymthe.2025.03.065_bib153","doi-asserted-by":"crossref","first-page":"400","DOI":"10.1126\/science.aad5725","article-title":"Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy","volume":"351","author":"Long","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.065_bib154","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.2025.03.065_bib155","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1126\/science.aad5177","article-title":"In\u00a0vivo gene editing in dystrophic mouse muscle and muscle stem cells","volume":"351","author":"Tabebordbar","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.065_bib156","doi-asserted-by":"crossref","first-page":"R9","DOI":"10.1093\/hmg\/ddv420","article-title":"Progress and prospects of gene therapy clinical trials for the muscular dystrophies","volume":"25","author":"Bengtsson","year":"2016","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.03.065_bib157","doi-asserted-by":"crossref","first-page":"564","DOI":"10.1038\/mt.2015.192","article-title":"CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice","volume":"24","author":"Xu","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib158","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.2025.03.065_bib159","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aan8081","article-title":"Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy","volume":"9","author":"Amoasii","year":"2017","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib160","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.2025.03.065_bib161","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1038\/s41591-019-0738-2","article-title":"Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy","volume":"26","author":"Moretti","year":"2020","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib162","doi-asserted-by":"crossref","first-page":"2176","DOI":"10.1016\/j.ymthe.2022.02.003","article-title":"Comparison of dystrophin expression following gene editing and gene replacement in an aged preclinical DMD animal model","volume":"30","author":"Bengtsson","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib163","doi-asserted-by":"crossref","first-page":"6769","DOI":"10.1038\/s41467-021-26830-7","article-title":"Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models","volume":"12","author":"Hakim","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib164","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1038\/s41591-018-0326-x","article-title":"Identification of preexisting adaptive immunity to Cas9 proteins in humans","volume":"25","author":"Charlesworth","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib165","doi-asserted-by":"crossref","first-page":"3497","DOI":"10.1038\/s41467-018-05843-9","article-title":"Cas9 immunity creates challenges for CRISPR gene editing therapies","volume":"9","author":"Crudele","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib166","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1038\/s41591-019-0344-3","article-title":"Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy","volume":"25","author":"Nelson","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib167","doi-asserted-by":"crossref","first-page":"1070","DOI":"10.1016\/j.ymthe.2020.11.003","article-title":"Dystrophin gene-editing stability is dependent on dystrophin levels in skeletal but not cardiac muscles","volume":"29","author":"Bengtsson","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib168","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1083\/jcb.9.2.493","article-title":"Satellite cell of skeletal muscle fibers","volume":"9","author":"Mauro","year":"1961","journal-title":"J.\u00a0Biophys. Biochem. Cytol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib169","doi-asserted-by":"crossref","first-page":"1254","DOI":"10.1016\/j.celrep.2019.03.105","article-title":"In situ modification of tissue stem and progenitor cell genomes","volume":"27","author":"Goldstein","year":"2019","journal-title":"Cell Rep."},{"key":"10.1016\/j.ymthe.2025.03.065_bib170","doi-asserted-by":"crossref","first-page":"320","DOI":"10.1016\/j.omtm.2020.09.016","article-title":"In\u00a0vivo gene editing of muscle stem cells with adeno-associated viral vectors in a mouse model of Duchenne muscular dystrophy","volume":"19","author":"Kwon","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.03.065_bib171","doi-asserted-by":"crossref","first-page":"1568","DOI":"10.1016\/j.ymthe.2019.06.012","article-title":"AAV9 edits muscle stem cells in normal and dystrophic adult mice","volume":"27","author":"Nance","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib172","doi-asserted-by":"crossref","first-page":"533","DOI":"10.1016\/j.stem.2016.01.021","article-title":"A single crispr-cas9 deletion strategy that targets the majority of DMD patients restores dystrophin function in hIPSC-derived muscle cells","volume":"18","author":"Young","year":"2016","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2025.03.065_bib173","doi-asserted-by":"crossref","first-page":"1796","DOI":"10.1126\/science.1104297","article-title":"Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping","volume":"306","author":"Goyenvalle","year":"2004","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.065_bib174","doi-asserted-by":"crossref","first-page":"2120","DOI":"10.1038\/mt.2012.181","article-title":"Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping","volume":"20","author":"Vulin","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib175","doi-asserted-by":"crossref","first-page":"1037","DOI":"10.1016\/j.omtm.2020.04.025","article-title":"Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice","volume":"17","author":"Aupy","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.03.065_bib176","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1016\/j.omtm.2022.07.005","article-title":"Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse","volume":"26","author":"Wein","year":"2022","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.03.065_bib177","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2023.101144","article-title":"Persistence of exon 2 skipping and dystrophin expression at 18\u00a0months after U7snRNA-mediated therapy in the Dup2 mouse model","volume":"31","author":"Gushchina","year":"2023","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.03.065_bib178","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.202013228","article-title":"Targeted genome editing in\u00a0vivo corrects a Dmd duplication restoring wild-type dystrophin expression","volume":"13","author":"Maino","year":"2021","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.ymthe.2025.03.065_bib179","doi-asserted-by":"crossref","first-page":"882","DOI":"10.1089\/hum.2020.286","article-title":"Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping","volume":"32","author":"Gushchina","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib180","doi-asserted-by":"crossref","first-page":"824","DOI":"10.1038\/s41587-020-0561-9","article-title":"Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors","volume":"38","author":"Anzalone","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib181","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib182","doi-asserted-by":"crossref","first-page":"883","DOI":"10.1038\/s41587-020-0453-z","article-title":"Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity","volume":"38","author":"Richter","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib183","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.abg4910","article-title":"Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing","volume":"7","author":"Chemello","year":"2021","journal-title":"Sci. Adv."},{"key":"10.1016\/j.ymthe.2025.03.065_bib184","doi-asserted-by":"crossref","first-page":"3719","DOI":"10.1038\/s41467-021-23996-y","article-title":"Efficient precise in\u00a0vivo base editing in adult dystrophic mice","volume":"12","author":"Xu","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib185","doi-asserted-by":"crossref","first-page":"5927","DOI":"10.1038\/s41467-024-50340-x","article-title":"Adenine base editing-mediated exon skipping restores dystrophin in humanized Duchenne mouse model","volume":"15","author":"Lin","year":"2024","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib186","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":"Duchene","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib187","doi-asserted-by":"crossref","first-page":"4439","DOI":"10.1038\/s41467-019-12449-2","article-title":"High levels of AAV vector integration into CRISPR-induced DNA breaks","volume":"10","author":"Hanlon","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib188","doi-asserted-by":"crossref","first-page":"218","DOI":"10.1038\/s41587-021-01025-z","article-title":"Precise genomic deletions using paired prime editing","volume":"40","author":"Choi","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib189","doi-asserted-by":"crossref","DOI":"10.3390\/ijms23116160","article-title":"Prime editing permits the introduction of specific mutations in the gene responsible for Duchenne muscular dystrophy","volume":"23","author":"Happi Mbakam","year":"2022","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ymthe.2025.03.065_bib190","doi-asserted-by":"crossref","first-page":"3243","DOI":"10.1016\/j.ymthe.2021.09.003","article-title":"Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy","volume":"29","author":"Pickar-Oliver","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2025.03.065_bib191","doi-asserted-by":"crossref","first-page":"7761","DOI":"10.1093\/nar\/gkac567","article-title":"Large-scale genome editing based on high-capacity adenovectors and CRISPR-Cas9 nucleases rescues full-length dystrophin synthesis in DMD muscle cells","volume":"50","author":"Tasca","year":"2022","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2025.03.065_bib192","doi-asserted-by":"crossref","first-page":"486","DOI":"10.1016\/j.omtm.2023.08.009","article-title":"CRISPR-Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice","volume":"30","author":"Stephenson","year":"2023","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.03.065_bib193","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1038\/s41587-021-01133-w","article-title":"Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing","volume":"40","author":"Anzalone","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2025.03.065_bib194","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1038\/s41551-024-01227-1","article-title":"Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing","volume":"9","author":"Pandey","year":"2025","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.ymthe.2025.03.065_bib195","doi-asserted-by":"crossref","first-page":"7101","DOI":"10.1038\/s41467-021-26714-w","article-title":"Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice","volume":"12","author":"Kenjo","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib196","doi-asserted-by":"crossref","first-page":"3232","DOI":"10.1038\/s41467-020-17029-3","article-title":"Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing","volume":"11","author":"Wei","year":"2020","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib197","doi-asserted-by":"crossref","first-page":"839","DOI":"10.1126\/science.aav4294","article-title":"Programmed DNA destruction by miniature CRISPR-Cas14 enzymes","volume":"362","author":"Harrington","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.065_bib198","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1126\/science.abb1400","article-title":"CRISPR-CasPhi from huge phages is a hypercompact genome editor","volume":"369","author":"Pausch","year":"2020","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2025.03.065_bib199","doi-asserted-by":"crossref","first-page":"4333","DOI":"10.1016\/j.molcel.2021.08.008","article-title":"Engineered miniature CRISPR-Cas system for mammalian genome regulation and editing","volume":"81","author":"Xu","year":"2021","journal-title":"Mol. Cell"},{"key":"10.1016\/j.ymthe.2025.03.065_bib200","doi-asserted-by":"crossref","first-page":"2046","DOI":"10.1038\/s41467-023-37829-7","article-title":"Engineered CRISPR-OsCas12f1 and RhCas12f1 with robust activities and expanded target range for genome editing","volume":"14","author":"Kong","year":"2023","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2025.03.065_bib201","doi-asserted-by":"crossref","DOI":"10.1172\/JCI162809","article-title":"Mini-dCas13X-mediated RNA editing restores dystrophin expression in a humanized mouse model of Duchenne muscular dystrophy","volume":"133","author":"Li","year":"2023","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2025.03.065_bib202","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1038\/s41586-021-03770-2","article-title":"Regulated control of gene therapies by drug-induced splicing","volume":"596","author":"Monteys","year":"2021","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2025.03.065_bib203","doi-asserted-by":"crossref","DOI":"10.1038\/mtm.2014.38","article-title":"Adeno-associated viral vectors do not efficiently target muscle satellite cells","volume":"1","author":"Arnett","year":"2014","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.03.065_bib204","first-page":"550","article-title":"Targeting muscle stem cells for permanent correction of dystrophin expression in Duchenne muscular dystrophy","volume":"32","author":"Hauschka","year":"2024","journal-title":"Mol. Ther."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001625002746?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001625002746?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T16:39:56Z","timestamp":1792687196000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001625002746"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,5]]},"references-count":203,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2025,5]]}},"alternative-id":["S1525001625002746"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2025.03.065","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2025,5]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2025.03.065","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}