{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T10:32:44Z","timestamp":1790418764781,"version":"4.1.0"},"reference-count":81,"publisher":"SAGE Publications","issue":"s2","license":[{"start":{"date-parts":[[2021,11,30]],"date-time":"2021-11-30T00:00:00Z","timestamp":1638230400000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JND"],"published-print":{"date-parts":[[2021,11,30]]},"abstract":"<jats:p>Adult skeletal muscle is a relatively stable tissue, as the multinucleated muscle fibres contain post-mitotic myonuclei. During early postnatal life, muscle growth occurs by the addition of skeletal muscle stem cells (satellite cells) or their progeny to growing muscle fibres. In Duchenne muscular dystrophy, which we shall use as an example of muscular dystrophies, the muscle fibres lack dystrophin and undergo necrosis. Satellite-cell mediated regeneration occurs, to repair and replace the necrotic muscle fibres, but as the regenerated muscle fibres still lack dystrophin, they undergo further cycles of degeneration and regeneration. AAV gene therapy is a promising approach for treating Duchenne muscular dystrophy. But for a single dose of, for example, AAV coding for microdystrophin, to be effective, the treated myonuclei must persist, produce sufficient dystrophin and a sufficient number of nuclei must be targeted. This latter point is crucial as AAV vector remains episomal and does not replicate in dividing cells. Here, we describe and compare the growth of skeletal muscle in rodents and in humans and discuss the evidence that myofibre necrosis and regeneration leads to the loss of viral genomes within skeletal muscle. In addition, muscle growth is expected to lead to the dilution of the transduced nuclei especially in case of very early intervention, but it is not clear if growth could result in insufficient dystrophin to prevent muscle fibre breakdown. This should be the focus of future studies.<\/jats:p>","DOI":"10.3233\/jnd-210683","type":"journal-article","created":{"date-parts":[[2021,7,30]],"date-time":"2021-07-30T10:59:52Z","timestamp":1627642792000},"page":"S317-S324","source":"Crossref","is-referenced-by-count":17,"title":["Changes in Myonuclear Number During Postnatal Growth \u2013 Implications for AAV Gene Therapy for Muscular Dystrophy"],"prefix":"10.1177","volume":"8","author":[{"given":"Jennifer","family":"Morgan","sequence":"first","affiliation":[{"name":"The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK"},{"name":"National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[{"name":"The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK"},{"name":"National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","reference":[{"issue":"1","key":"10.3233\/JND-210683_ref1","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1046\/j.1469-7580.2003.00139.x","article-title":"The formation of skeletal muscle: From somite to limb","volume":"202","author":"Buckingham","year":"2003","journal-title":"J Anat"},{"key":"10.3233\/JND-210683_ref2","doi-asserted-by":"crossref","unstructured":"Morgan J , Partridge T . 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Prepubertal skeletal muscle growth requires Pax7-expressing satellite cell-derived myonuclear contribution. 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Adeno-associated viral (AAV) vectors do not efficiently target muscle satellite cells. Mol Ther Methods Clin Dev. 2014;1.","DOI":"10.1038\/mtm.2014.38"},{"issue":"6271","key":"10.3233\/JND-210683_ref42","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1126\/science.aad5177","article-title":"In vivo gene editing in dystrophic mouse muscle and muscle stem cells","volume":"351","author":"Tabebordbar","year":"2016","journal-title":"Science"},{"key":"10.3233\/JND-210683_ref43","doi-asserted-by":"crossref","unstructured":"Meng J , Counsell J , Morgan JE . Effects of Mini-Dystrophin on Dystrophin-Deficient, Human Skeletal Muscle-Derived Cells. 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AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice. JCI Insight. 2018;3(23).","DOI":"10.1172\/jci.insight.124297"},{"key":"10.3233\/JND-210683_ref52","doi-asserted-by":"crossref","unstructured":"Amoasii L , Long C , Li H , Mireault AA , Shelton JM , Sanchez-Ortiz E , et al. Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy. 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Ther"},{"issue":"5","key":"10.3233\/JND-210683_ref56","doi-asserted-by":"crossref","first-page":"1332","DOI":"10.2353\/ajpath.2008.071042","article-title":"Preservation of muscle force in Mdx3cv mice correlates with low-level expression of a near full-length dystrophin protein","volume":"172","author":"Li","year":"2008","journal-title":"Am J Pathol."},{"issue":"11-12","key":"10.3233\/JND-210683_ref57","doi-asserted-by":"crossref","first-page":"913","DOI":"10.1016\/j.nmd.2007.07.005","article-title":"Dystrophin levels as low as 30%are sufficient to avoid muscular dystrophy in the human","volume":"17","author":"Neri","year":"2007","journal-title":"Neuromuscul Disord"},{"issue":"1","key":"10.3233\/JND-210683_ref58","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1038\/mt.2010.213","article-title":"Physiological characterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense 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