{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T15:46:24Z","timestamp":1790523984372,"version":"4.1.0"},"reference-count":75,"publisher":"Elsevier BV","issue":"9","license":[{"start":{"date-parts":[[2019,9,1]],"date-time":"2019-09-01T00:00:00Z","timestamp":1567296000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2019,9,1]],"date-time":"2019-09-01T00:00:00Z","timestamp":1567296000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2020,9,4]],"date-time":"2020-09-04T00:00:00Z","timestamp":1599177600000},"content-version":"vor","delay-in-days":369,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006108","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\/100000005","name":"U.S. Department of Defense","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000005","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":[[2019,9]]},"DOI":"10.1016\/j.ymthe.2019.06.012","type":"journal-article","created":{"date-parts":[[2019,7,3]],"date-time":"2019-07-03T13:22:08Z","timestamp":1562160128000},"page":"1568-1585","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":72,"title":["AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice"],"prefix":"10.1016","volume":"27","author":[{"given":"Michael E.","family":"Nance","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruicheng","family":"Shi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chady H.","family":"Hakim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nalinda B.","family":"Wasala","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yongping","family":"Yue","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiufang","family":"Pan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tracy","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolyn A.","family":"Robinson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sean X.","family":"Duan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gang","family":"Yao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"N. Nora","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shi-jie","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn R.","family":"Wagner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charles A.","family":"Gersbach","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dongsheng","family":"Duan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2019.06.012_bib1","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.ymthe.2019.06.012_bib2","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.2019.06.012_bib3","doi-asserted-by":"crossref","first-page":"479","DOI":"10.1016\/j.molmed.2016.04.002","article-title":"Satellite Cells in Muscular Dystrophy - Lost in Polarity","volume":"22","author":"Chang","year":"2016","journal-title":"Trends Mol. Med."},{"key":"10.1016\/j.ymthe.2019.06.012_bib4","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1038\/nrneurol.2017.126","article-title":"Genome engineering: a new approach to gene therapy for neuromuscular disorders","volume":"13","author":"Nelson","year":"2017","journal-title":"Nat. Rev. Neurol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib5","doi-asserted-by":"crossref","first-page":"795","DOI":"10.1038\/s41551-018-0320-0","article-title":"CRISPR alleviates muscular dystrophy in dogs","volume":"2","author":"Duan","year":"2018","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.ymthe.2019.06.012_bib6","doi-asserted-by":"crossref","first-page":"1205","DOI":"10.1152\/physrev.00046.2017","article-title":"Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing","volume":"98","author":"Zhang","year":"2018","journal-title":"Physiol. Rev."},{"key":"10.1016\/j.ymthe.2019.06.012_bib7","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.2019.06.012_bib8","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1126\/science.aad5143","article-title":"In\u00a0vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy","volume":"351","author":"Nelson","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2019.06.012_bib9","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.2019.06.012_bib10","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.2019.06.012_bib11","doi-asserted-by":"crossref","first-page":"14454","DOI":"10.1038\/ncomms14454","article-title":"Muscle-specific CRISPR\/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy","volume":"8","author":"Bengtsson","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2019.06.012_bib12","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1161\/CIRCRESAHA.117.310996","article-title":"In\u00a0Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice","volume":"121","author":"El Refaey","year":"2017","journal-title":"Circ. Res."},{"key":"10.1016\/j.ymthe.2019.06.012_bib13","doi-asserted-by":"crossref","first-page":"124297","DOI":"10.1172\/jci.insight.124297","article-title":"AAV CRISPR editing rescues cardiac and muscle function for 18\u00a0months in dystrophic mice","volume":"3","author":"Hakim","year":"2018","journal-title":"JCI Insight"},{"key":"10.1016\/j.ymthe.2019.06.012_bib14","doi-asserted-by":"crossref","first-page":"86","DOI":"10.1126\/science.aau1549","article-title":"Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy","volume":"362","author":"Amoasii","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2019.06.012_bib15","doi-asserted-by":"crossref","first-page":"535","DOI":"10.1089\/hum.2018.243","article-title":"Questions answered and unanswered by the first CRISPR editing study in a canine model of Duchenne muscular dystrophy","volume":"30","author":"Wasala","year":"2019","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2019.06.012_bib16","doi-asserted-by":"crossref","first-page":"2604","DOI":"10.1016\/j.ymthe.2018.08.010","article-title":"CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In\u00a0Vitro and In\u00a0Vivo","volume":"26","author":"Duch\u00eane","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.06.012_bib17","doi-asserted-by":"crossref","first-page":"e283","DOI":"10.1038\/mtna.2015.58","article-title":"Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method","volume":"5","author":"Iyombe-Engembe","year":"2016","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2019.06.012_bib18","doi-asserted-by":"crossref","first-page":"1888","DOI":"10.1038\/mt.2016.191","article-title":"Gene Editing for Duchenne Muscular Dystrophy Using the CRISPR\/Cas9 Technology: The Importance of Fine-tuning the Approach","volume":"24","author":"Tremblay","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.06.012_bib19","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1016\/j.coviro.2016.07.006","article-title":"Systemic delivery of adeno-associated viral vectors","volume":"21","author":"Duan","year":"2016","journal-title":"Curr. Opin. Virol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib20","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1089\/humc.2015.006","article-title":"Duchenne muscular dystrophy gene therapy in the canine model","volume":"26","author":"Duan","year":"2015","journal-title":"Hum. Gene Ther. Clin. Dev."},{"key":"10.1016\/j.ymthe.2019.06.012_bib21","doi-asserted-by":"crossref","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","article-title":"Single-dose gene-replacement therapy for spinal muscular atrophy","volume":"377","author":"Mendell","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2019.06.012_bib22","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.ymthe.2019.06.012_bib23","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.2019.06.012_bib24","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1038\/nrm3265","article-title":"Satellite cells, the engines of muscle repair","volume":"13","author":"Wang","year":"2011","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib25","doi-asserted-by":"crossref","first-page":"525","DOI":"10.1016\/j.gde.2006.08.008","article-title":"Myogenic progenitor cells and skeletal myogenesis in vertebrates","volume":"16","author":"Buckingham","year":"2006","journal-title":"Curr. Opin. Genet. Dev."},{"key":"10.1016\/j.ymthe.2019.06.012_bib26","doi-asserted-by":"crossref","first-page":"14038","DOI":"10.1038\/mtm.2014.38","article-title":"Adeno-associated viral (AAV) 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.2019.06.012_bib27","first-page":"387","article-title":"A review of muscle transplantation in mammals","volume":"27","author":"Carlson","year":"1978","journal-title":"Physiol. Bohemoslov."},{"key":"10.1016\/j.ymthe.2019.06.012_bib28","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1093\/oxfordjournals.bmb.a071631","article-title":"Skeletal muscle: regeneration and transplantation studies","volume":"36","author":"Sloper","year":"1980","journal-title":"Br. Med. Bull."},{"key":"10.1016\/j.ymthe.2019.06.012_bib29","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1016\/0022-510X(77)90138-1","article-title":"A host contribution to the regeneration of muscle grafts","volume":"33","author":"Partridge","year":"1977","journal-title":"J.\u00a0Neurol. Sci."},{"key":"10.1016\/j.ymthe.2019.06.012_bib30","doi-asserted-by":"crossref","first-page":"677","DOI":"10.1007\/BF00216211","article-title":"Isoenzyme studies of whole muscle grafts and movement of muscle precursor cells","volume":"230","author":"Grounds","year":"1983","journal-title":"Cell Tissue Res."},{"key":"10.1016\/j.ymthe.2019.06.012_bib31","doi-asserted-by":"crossref","first-page":"325","DOI":"10.1002\/path.1711320404","article-title":"The contribution of exogenous cells to regenerating skeletal muscle: an isoenzyme study of muscle allografts in mice","volume":"132","author":"Grounds","year":"1980","journal-title":"J.\u00a0Pathol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib32","doi-asserted-by":"crossref","first-page":"386","DOI":"10.1007\/BF01578428","article-title":"Muscle precursor cells invade and repopulate freeze-killed muscles","volume":"8","author":"Morgan","year":"1987","journal-title":"J.\u00a0Muscle Res. Cell Motil."},{"key":"10.1016\/j.ymthe.2019.06.012_bib33","doi-asserted-by":"crossref","first-page":"6477","DOI":"10.4049\/jimmunol.174.10.6477","article-title":"Human lymphoid and myeloid cell development in NOD\/LtSz-scid IL2R gamma null mice engrafted with mobilized human hemopoietic stem cells","volume":"174","author":"Shultz","year":"2005","journal-title":"J.\u00a0Immunol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib34","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1073\/pnas.2637010100","article-title":"Shifting foci of hematopoiesis during reconstitution from single stem cells","volume":"101","author":"Cao","year":"2004","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2019.06.012_bib35","doi-asserted-by":"crossref","first-page":"6638","DOI":"10.1182\/blood-2009-02-204321","article-title":"Lymphoid-tissue-specific homing of bone-marrow-derived dendritic cells","volume":"113","author":"Creusot","year":"2009","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2019.06.012_bib36","first-page":"1","article-title":"Breeding of a non-obese, diabetic strain of mice","volume":"29","author":"Makino","year":"1980","journal-title":"Jikken Dobutsu"},{"key":"10.1016\/j.ymthe.2019.06.012_bib37","doi-asserted-by":"crossref","first-page":"1169","DOI":"10.1007\/s10620-014-3168-5","article-title":"NOD-scidIl2rg (tm1Wjl) and NOD-Rag1 (null) Il2rg (tm1Wjl) : a model for stromal cell-tumor cell interaction for human colon cancer","volume":"59","author":"Maykel","year":"2014","journal-title":"Dig. Dis. Sci."},{"key":"10.1016\/j.ymthe.2019.06.012_bib38","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1038\/nn.2467","article-title":"A robust and high-throughput Cre reporting and characterization system for the whole mouse brain","volume":"13","author":"Madisen","year":"2010","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.ymthe.2019.06.012_bib39","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1038\/ncb2015","article-title":"Muscle injury activates resident fibro\/adipogenic progenitors that facilitate myogenesis","volume":"12","author":"Joe","year":"2010","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib40","doi-asserted-by":"crossref","first-page":"3194","DOI":"10.1634\/stemcells.2007-1017","article-title":"Prospective isolation of skeletal muscle stem cells with a Pax7 reporter","volume":"26","author":"Bosnakovski","year":"2008","journal-title":"Stem Cells"},{"key":"10.1016\/j.ymthe.2019.06.012_bib41","doi-asserted-by":"crossref","first-page":"5310","DOI":"10.1172\/JCI70314","article-title":"Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression","volume":"123","author":"Mueller","year":"2013","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2019.06.012_bib42","doi-asserted-by":"crossref","first-page":"516","DOI":"10.1038\/mt.2008.283","article-title":"Detection of intact rAAV particles up to 6 years after successful gene transfer in the retina of dogs and primates","volume":"17","author":"Stieger","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.06.012_bib43","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1002\/ar.1091850303","article-title":"The number of satellite cells in normal human muscle","volume":"185","author":"Schmalbruch","year":"1976","journal-title":"Anat. Rec."},{"key":"10.1016\/j.ymthe.2019.06.012_bib44","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1002\/ar.1091890204","article-title":"The number of nuclei in adult rat muscles with special reference to satellite cells","volume":"189","author":"Schmalbruch","year":"1977","journal-title":"Anat. Rec."},{"key":"10.1016\/j.ymthe.2019.06.012_bib45","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1369\/jhc.7A7301.2007","article-title":"Pax7 reveals a greater frequency and concentration of satellite cells at the ends of growing skeletal muscle fibers","volume":"56","author":"Allouh","year":"2008","journal-title":"J.\u00a0Histochem. Cytochem."},{"key":"10.1016\/j.ymthe.2019.06.012_bib46","doi-asserted-by":"crossref","first-page":"534","DOI":"10.1152\/jappl.2001.91.2.534","article-title":"Myogenic satellite cells: physiology to molecular biology","volume":"91","author":"Hawke","year":"2001","journal-title":"J.\u00a0Appl. Physiol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib47","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.2019.06.012_bib48","doi-asserted-by":"crossref","first-page":"3495","DOI":"10.1128\/JVI.77.6.3495-3504.2003","article-title":"Genetic fate of recombinant adeno-associated virus vector genomes in muscle","volume":"77","author":"Schnepp","year":"2003","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib49","doi-asserted-by":"crossref","first-page":"202","DOI":"10.1038\/ncb3477","article-title":"A Twist2-dependent progenitor cell contributes to adult skeletal muscle","volume":"19","author":"Liu","year":"2017","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib50","doi-asserted-by":"crossref","first-page":"390","DOI":"10.1038\/43919","article-title":"Dystrophin expression in the mdx mouse restored by stem cell transplantation","volume":"401","author":"Gussoni","year":"1999","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2019.06.012_bib51","doi-asserted-by":"crossref","first-page":"2298","DOI":"10.1089\/scd.2012.0647","article-title":"Role of pericytes in skeletal muscle regeneration and fat accumulation","volume":"22","author":"Birbrair","year":"2013","journal-title":"Stem Cells Dev."},{"key":"10.1016\/j.ymthe.2019.06.012_bib52","doi-asserted-by":"crossref","first-page":"2045","DOI":"10.4161\/cc.9.11.11854","article-title":"Fibro\/adipogenic progenitors: a\u00a0double-edged sword in skeletal muscle regeneration","volume":"9","author":"Natarajan","year":"2010","journal-title":"Cell Cycle"},{"key":"10.1016\/j.ymthe.2019.06.012_bib53","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1038\/ncb2025","article-title":"Identification and characterization of a non-satellite cell muscle resident progenitor during postnatal development","volume":"12","author":"Mitchell","year":"2010","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib54","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1089\/104303401750148784","article-title":"A new immunodeficient mouse model for human myoblast transplantation","volume":"12","author":"Cooper","year":"2001","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2019.06.012_bib55","doi-asserted-by":"crossref","first-page":"128","DOI":"10.1203\/00006450-199207000-00025","article-title":"The frequency of revertants in mdx mouse genetic models for Duchenne muscular dystrophy","volume":"32","author":"Danko","year":"1992","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.ymthe.2019.06.012_bib56","series-title":"Recent Advances in Skeletal Muscle Differentiation","first-page":"93","article-title":"The enigma of the \u2018dystrophin revertant\u2019 muscle fibre","author":"Partridge","year":"2008"},{"key":"10.1016\/j.ymthe.2019.06.012_bib57","doi-asserted-by":"crossref","first-page":"2679","DOI":"10.1242\/jcs.03000","article-title":"Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as an index of muscle regeneration","volume":"119","author":"Yokota","year":"2006","journal-title":"J.\u00a0Cell Sci."},{"key":"10.1016\/j.ymthe.2019.06.012_bib58","doi-asserted-by":"crossref","first-page":"985","DOI":"10.1083\/jcb.148.5.985","article-title":"Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion","volume":"148","author":"Lu","year":"2000","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib59","doi-asserted-by":"crossref","first-page":"139","DOI":"10.1634\/stemcells.20-2-139","article-title":"Analysis of different promoter systems for efficient transgene expression in mouse embryonic stem cell lines","volume":"20","author":"Chung","year":"2002","journal-title":"Stem Cells"},{"key":"10.1016\/j.ymthe.2019.06.012_bib60","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1038\/nature11438","article-title":"The aged niche disrupts muscle stem cell quiescence","volume":"490","author":"Chakkalakal","year":"2012","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2019.06.012_bib61","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.2019.06.012_bib62","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.2019.06.012_bib63","doi-asserted-by":"crossref","first-page":"1611","DOI":"10.1002\/stem.1402","article-title":"A new immuno-, dystrophin-deficient model, the NSG-mdx(4Cv) mouse, provides evidence for functional improvement following allogeneic satellite cell transplantation","volume":"31","author":"Arpke","year":"2013","journal-title":"Stem Cells"},{"key":"10.1016\/j.ymthe.2019.06.012_bib64","doi-asserted-by":"crossref","first-page":"13104","DOI":"10.1073\/pnas.0702778104","article-title":"Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction","volume":"104","author":"Wang","year":"2007","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2019.06.012_bib65","first-page":"2633","article-title":"Genomic removal of a therapeutic mini-dystrophin gene from adult mice elicits a Duchenne muscular dystrophy-like phenotype","volume":"25","author":"Wasala","year":"2016","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2019.06.012_bib66","doi-asserted-by":"crossref","first-page":"6381","DOI":"10.1128\/JVI.78.12.6381-6388.2004","article-title":"Clades of Adeno-associated viruses are widely disseminated in human tissues","volume":"78","author":"Gao","year":"2004","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib67","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1007\/978-1-61779-343-1_15","article-title":"Recombinant adeno-associated viral vector production and purification","volume":"798","author":"Shin","year":"2012","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib68","doi-asserted-by":"crossref","first-page":"467","DOI":"10.1113\/jphysiol.2003.045328","article-title":"Number and spatial distribution of nuclei in the muscle fibres of normal mice studied in\u00a0vivo","volume":"551","author":"Bruusgaard","year":"2003","journal-title":"J.\u00a0Physiol."},{"key":"10.1016\/j.ymthe.2019.06.012_bib69","doi-asserted-by":"crossref","first-page":"1450","DOI":"10.1172\/JCI34022","article-title":"In\u00a0vivo time-lapse microscopy reveals no loss of murine myonuclei during weeks of muscle atrophy","volume":"118","author":"Bruusgaard","year":"2008","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2019.06.012_bib70","doi-asserted-by":"crossref","first-page":"e0147669","DOI":"10.1371\/journal.pone.0147669","article-title":"Musculoskeletal Geometry, Muscle Architecture and Functional Specialisations of the Mouse Hindlimb","volume":"11","author":"Charles","year":"2016","journal-title":"PLoS ONE"},{"key":"10.1016\/j.ymthe.2019.06.012_bib71","doi-asserted-by":"crossref","first-page":"1333","DOI":"10.1002\/mrm.20476","article-title":"Determination of mouse skeletal muscle architecture using three-dimensional diffusion tensor imaging","volume":"53","author":"Heemskerk","year":"2005","journal-title":"Magn. Reson. Med."},{"key":"10.1016\/j.ymthe.2019.06.012_bib72","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1159\/000147970","article-title":"Muscle fiber length and moment arm coordination during dorsi- and plantarflexion in the mouse hindlimb","volume":"159","author":"Lieber","year":"1997","journal-title":"Acta Anat. (Basel)"},{"key":"10.1016\/j.ymthe.2019.06.012_bib73","doi-asserted-by":"crossref","first-page":"3180","DOI":"10.1093\/hmg\/ddu028","article-title":"Human skeletal muscle xenograft as a new preclinical model for muscle disorders","volume":"23","author":"Zhang","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2019.06.012_bib74","doi-asserted-by":"crossref","first-page":"e88280","DOI":"10.1371\/journal.pone.0088280","article-title":"Characterization of 65 epitope-specific dystrophin monoclonal antibodies in canine and murine models of duchenne muscular dystrophy by immunostaining and western blot","volume":"9","author":"Kodippili","year":"2014","journal-title":"PLoS ONE"},{"key":"10.1016\/j.ymthe.2019.06.012_bib75","doi-asserted-by":"crossref","first-page":"750","DOI":"10.1038\/mt.2012.283","article-title":"Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy","volume":"21","author":"Shin","year":"2013","journal-title":"Mol. Ther."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001619303077?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001619303077?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,26]],"date-time":"2025-10-26T17:55:23Z","timestamp":1761501323000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001619303077"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,9]]},"references-count":75,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2019,9]]}},"alternative-id":["S1525001619303077"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2019.06.012","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2019,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2019.06.012","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2019 The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}