{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,26]],"date-time":"2026-02-26T15:57:48Z","timestamp":1772121468129,"version":"3.50.1"},"reference-count":68,"publisher":"Elsevier BV","issue":"11-12","license":[{"start":{"date-parts":[[2022,12,1]],"date-time":"2022-12-01T00:00:00Z","timestamp":1669852800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2022,12,1]],"date-time":"2022-12-01T00:00:00Z","timestamp":1669852800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2022,10,31]],"date-time":"2022-10-31T00:00:00Z","timestamp":1667174400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","nmd-journal.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Neuromuscular Disorders"],"published-print":{"date-parts":[[2022,12]]},"DOI":"10.1016\/j.nmd.2022.10.007","type":"journal-article","created":{"date-parts":[[2022,10,31]],"date-time":"2022-10-31T21:33:27Z","timestamp":1667252007000},"page":"908-922","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":5,"title":["CRISPR-mediated correction of skeletal muscle Ca2+ handling in a novel DMD patient-derived pluripotent stem cell model"],"prefix":"10.1016","volume":"32","author":[{"given":"Cristina","family":"Morera","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jihee","family":"Kim","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Amaia","family":"Paredes-Redondo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Muriel","family":"Nobles","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Denis","family":"Rybin","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Robert","family":"Moccia","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anna","family":"Kowala","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jinhong","family":"Meng","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Seth","family":"Garren","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pentao","family":"Liu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer E","family":"Morgan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nicolas","family":"Christoforou","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jane","family":"Owens","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andrew","family":"Tinker","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0435-7694","authenticated-orcid":false,"given":"Yung-Yao","family":"Lin","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.nmd.2022.10.007_bib0001","doi-asserted-by":"crossref","first-page":"702","DOI":"10.1093\/glycob\/cwv021","article-title":"Matriglycan: a novel polysaccharide that links dystroglycan to the basement membrane","volume":"25","author":"Yoshida-Moriguchi","year":"2015","journal-title":"Glycobiology"},{"key":"10.1016\/j.nmd.2022.10.007_bib0002","doi-asserted-by":"crossref","first-page":"1575","DOI":"10.1002\/mus.1192","article-title":"The dystrophin-glycoprotein complex, cellular signaling, and the regulation of cell survival in the muscular dystrophies","volume":"24","author":"Rando","year":"2001","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.nmd.2022.10.007_bib0003","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1152\/physrev.00007.2015","article-title":"Absence of dystrophin disrupts skeletal muscle signaling: roles of Ca 2+, reactive oxygen species, and nitric oxide in the development of muscular dystrophy","volume":"96","author":"Allen","year":"2016","journal-title":"Physiol Rev"},{"key":"10.1016\/j.nmd.2022.10.007_bib0004","doi-asserted-by":"crossref","first-page":"2025","DOI":"10.1016\/S0140-6736(19)32910-1","article-title":"Muscular dystrophies","volume":"394","author":"Mercuri","year":"2019","journal-title":"Lancet"},{"key":"10.1016\/j.nmd.2022.10.007_bib0005","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1016\/S1474-4422(03)00585-4","article-title":"Dystrophin and mutations: one gene, several proteins, multiple phenotypes","volume":"2","author":"Muntoni","year":"2003","journal-title":"Lancet Neurol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0006","doi-asserted-by":"crossref","first-page":"395","DOI":"10.1002\/humu.22758","article-title":"The TREAT-NMD DMD global database: analysis of more than 7000 Duchenne muscular dystrophy mutations","volume":"36","author":"Bladen","year":"2015","journal-title":"Hum Mutat"},{"key":"10.1016\/j.nmd.2022.10.007_bib0007","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.nmd.2022.10.007_bib0008","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1002\/mus.20586","article-title":"Den Dunnen JT. Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule","volume":"34","author":"Aartsma-Rus","year":"2006","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.nmd.2022.10.007_bib0009","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1089\/nat.2017.0682","article-title":"Development of exon skipping therapies for duchenne muscular dystrophy: a critical review and a perspective on the outstanding issues","volume":"27","author":"Aartsma-Rus","year":"2017","journal-title":"Nucleic Acid Ther"},{"key":"10.1016\/j.nmd.2022.10.007_bib0010","doi-asserted-by":"crossref","first-page":"371","DOI":"10.1146\/annurev-genom-091212-153527","article-title":"Therapeutics based on stop codon readthrough","volume":"15","author":"Keeling","year":"2014","journal-title":"Annu Rev Genomics Hum Genet"},{"key":"10.1016\/j.nmd.2022.10.007_bib0011","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.nmd.2022.10.007_bib0012","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1146\/annurev-med-081117-010451","article-title":"CRISPR correction of duchenne muscular dystrophy","volume":"70","author":"Min","year":"2019","journal-title":"Annu Rev Med"},{"key":"10.1016\/j.nmd.2022.10.007_bib0013","doi-asserted-by":"crossref","DOI":"10.1242\/dmm.035774","article-title":"Tracking progress: an update on animal models for Duchenne muscular dystrophy","volume":"11","author":"Wells","year":"2018","journal-title":"Dis Model Mech"},{"key":"10.1016\/j.nmd.2022.10.007_bib0014","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1242\/dmm.018424","article-title":"Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy","volume":"8","author":"McGreevy","year":"2015","journal-title":"Dis Model Mech"},{"key":"10.1016\/j.nmd.2022.10.007_bib0015","doi-asserted-by":"crossref","first-page":"1811","DOI":"10.1212\/WNL.0000000000004570","article-title":"A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy","volume":"89","author":"Victor","year":"2017","journal-title":"Neurology"},{"key":"10.1016\/j.nmd.2022.10.007_bib0016","doi-asserted-by":"crossref","first-page":"922","DOI":"10.1002\/cpdd.642","article-title":"A Phase 1b trial to assess the pharmacokinetics of ezutromid in pediatric duchenne muscular dystrophy patients on a balanced diet","volume":"8","author":"Muntoni","year":"2019","journal-title":"Clin Pharmacol Drug Dev"},{"key":"10.1016\/j.nmd.2022.10.007_bib0017","doi-asserted-by":"crossref","first-page":"1215","DOI":"10.1152\/physrev.2000.80.3.1215","article-title":"Calcium ion in skeletal muscle: its crucial role for muscle function, plasticity, and disease","volume":"80","author":"Berchtold","year":"2000","journal-title":"Physiol Rev"},{"key":"10.1016\/j.nmd.2022.10.007_bib0018","series-title":"eLS","first-page":"1","article-title":"Human induced pluripotent stem cells: challenges and opportunities in developing new therapies for muscular dystrophies","author":"Paredes-Redondo","year":"2019"},{"key":"10.1016\/j.nmd.2022.10.007_bib0019","doi-asserted-by":"crossref","first-page":"2104","DOI":"10.1242\/dev.151035","article-title":"Making muscle: skeletal myogenesis in vivo and in vitro","volume":"144","author":"Chal","year":"2017","journal-title":"Development"},{"key":"10.1016\/j.nmd.2022.10.007_bib0020","doi-asserted-by":"crossref","first-page":"393","DOI":"10.1038\/nature24052","article-title":"Establishment of mouse expanded potential stem cells","volume":"550","author":"Yang","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.nmd.2022.10.007_bib0021","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.exphem.2019.07.003","article-title":"Expanded potential stem cell media as a tool to study human developmental hematopoiesis in vitro","volume":"76","author":"Wilkinson","year":"2019","journal-title":"Exp Hematol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0022","doi-asserted-by":"crossref","first-page":"1833","DOI":"10.1038\/nprot.2016.110","article-title":"Generation of human muscle fibers and satellite-like cells from human pluripotent stem cells in vitro","volume":"11","author":"Chal","year":"2016","journal-title":"Nat Protoc"},{"key":"10.1016\/j.nmd.2022.10.007_bib0023","doi-asserted-by":"crossref","first-page":"e47967","DOI":"10.15252\/embr.201947967","article-title":"A new patient-derived iPSC model for dystroglycanopathies validates a compound that increases glycosylation of \u03b1-dystroglycan","volume":"20","author":"Kim","year":"2019","journal-title":"EMBO Rep"},{"key":"10.1016\/j.nmd.2022.10.007_bib0024","doi-asserted-by":"crossref","first-page":"e30","DOI":"10.5334\/jors.bl","article-title":"PIVlab \u2013 towards user-friendly, affordable and accurate digital particle image velocimetry in MATLAB","volume":"2","author":"Thielicke","year":"2014","journal-title":"J Open Res Softw"},{"key":"10.1016\/j.nmd.2022.10.007_bib0025","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1093\/bioinformatics\/bts635","article-title":"STAR: ultrafast universal RNA-seq aligner","volume":"29","author":"Dobin","year":"2013","journal-title":"Bioinformatics"},{"key":"10.1016\/j.nmd.2022.10.007_bib0026","doi-asserted-by":"crossref","first-page":"417","DOI":"10.1038\/nmeth.4197","article-title":"Salmon provides fast and bias-aware quantification of transcript expression","volume":"14","author":"Patro","year":"2017","journal-title":"Nat Methods"},{"key":"10.1016\/j.nmd.2022.10.007_bib0027","doi-asserted-by":"crossref","first-page":"550","DOI":"10.1186\/s13059-014-0550-8","article-title":"Moderated estimation of fold change and dispersion for RNA-seq data with DESeq2","volume":"15","author":"Love","year":"2014","journal-title":"Genome Biol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0028","first-page":"275","article-title":"False discovery rates: a new deal","volume":"18","author":"Stephens","year":"2017","journal-title":"Biostatistics"},{"key":"10.1016\/j.nmd.2022.10.007_bib0029","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1111\/j.2517-6161.1995.tb02031.x","article-title":"Controlling the false discovery rate: a practical and powerful approach to multiple testing","volume":"57","author":"Benjamini","year":"1995","journal-title":"J R Stat Soc Ser B"},{"key":"10.1016\/j.nmd.2022.10.007_bib0030","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1038\/ng1180","article-title":"PGC-1alpha-responsive genes involved in oxidative phosphorylation are coordinately downregulated in human diabetes","volume":"34","author":"Mootha","year":"2003","journal-title":"Nat Genet"},{"key":"10.1016\/j.nmd.2022.10.007_bib0031","doi-asserted-by":"crossref","first-page":"15545","DOI":"10.1073\/pnas.0506580102","article-title":"Gene set enrichment analysis: A knowledge-based approach for interpreting genome-wide expression profiles","volume":"102","author":"Subramanian","year":"2005","journal-title":"Proc Natl Acad Sci"},{"key":"10.1016\/j.nmd.2022.10.007_bib0032","first-page":"60012","article-title":"Fast gene set enrichment analysis","author":"Korotkevich","year":"2019","journal-title":"BioRxiv"},{"key":"10.1016\/j.nmd.2022.10.007_bib0033","doi-asserted-by":"crossref","first-page":"417","DOI":"10.1016\/j.cels.2015.12.004","article-title":"The Molecular Signatures Database (MSigDB) hallmark gene set collection","volume":"1","author":"Liberzon","year":"2015","journal-title":"Cell Syst"},{"key":"10.1016\/j.nmd.2022.10.007_bib0034","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0211384","article-title":"Identification of qPCR reference genes suitable for normalizing gene expression in the mdx mouse model of Duchenne muscular dystrophy","volume":"14","author":"Hildyard","year":"2019","journal-title":"PLoS One"},{"key":"10.1016\/j.nmd.2022.10.007_bib0035","doi-asserted-by":"crossref","first-page":"2301","DOI":"10.1016\/j.celrep.2016.05.016","article-title":"Concordant but varied phenotypes among duchenne muscular dystrophy patient-specific myoblasts derived using a human iPSC-Based Model","volume":"15","author":"Choi","year":"2016","journal-title":"Cell Rep"},{"key":"10.1016\/j.nmd.2022.10.007_bib0036","doi-asserted-by":"crossref","first-page":"eabi8787","DOI":"10.1126\/sciadv.abi8787","article-title":"Optogenetic modeling of human neuromuscular circuits in Duchenne muscular dystrophy with CRISPR and pharmacological corrections","volume":"7","author":"Paredes-Redondo","year":"2021","journal-title":"Sci Adv"},{"key":"10.1016\/j.nmd.2022.10.007_bib0037","doi-asserted-by":"crossref","first-page":"19233","DOI":"10.1074\/jbc.M115.666719","article-title":"Absence of the regulator of G-protein signaling, RGS4, predisposes to atrial fibrillation and is associated with abnormal calcium handling","volume":"290","author":"Opel","year":"2015","journal-title":"J Biol Chem"},{"key":"10.1016\/j.nmd.2022.10.007_bib0038","doi-asserted-by":"crossref","first-page":"140","DOI":"10.1016\/j.scr.2016.06.003","article-title":"Differentiation of control and ALS mutant human iPSCs into functional skeletal muscle cells, a tool for the study of neuromuscolar diseases","volume":"17","author":"Lenzi","year":"2016","journal-title":"Stem Cell Res"},{"key":"10.1016\/j.nmd.2022.10.007_bib0039","doi-asserted-by":"crossref","first-page":"152","DOI":"10.1186\/1471-2288-13-152","article-title":"Restricted mean survival time: an alternative to the hazard ratio for the design and analysis of randomized trials with a time-to-event outcome","volume":"13","author":"Royston","year":"2013","journal-title":"BMC Med Res Methodol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0040","doi-asserted-by":"crossref","first-page":"475","DOI":"10.1038\/s41467-017-00629-x","article-title":"The Ca2+ influx through the mammalian skeletal muscle dihydropyridine receptor is irrelevant for muscle performance","volume":"8","author":"Dayal","year":"2017","journal-title":"Nat Commun"},{"key":"10.1016\/j.nmd.2022.10.007_bib0041","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1038\/368065a0","article-title":"Ca2+-induced tropomyosin movement in Limulus thin filaments revealed by three-dimensional reconstruction","volume":"368","author":"Lehman","year":"1994","journal-title":"Nature"},{"key":"10.1016\/j.nmd.2022.10.007_bib0042","doi-asserted-by":"crossref","first-page":"2363","DOI":"10.1126\/science.289.5488.2363","article-title":"NF-kappaB-induced loss of MyoD messenger RNA: possible role in muscle decay and cachexia","volume":"289","author":"Guttridge","year":"2000","journal-title":"Science"},{"key":"10.1016\/j.nmd.2022.10.007_bib0043","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1016\/j.cell.2004.09.027","article-title":"IKK\u03b2\/NF-\u03baB activation causes severe muscle wasting in mice","volume":"119","author":"Cai","year":"2004","journal-title":"Cell"},{"key":"10.1016\/j.nmd.2022.10.007_bib0044","doi-asserted-by":"crossref","first-page":"889","DOI":"10.1172\/JCI30556","article-title":"Interplay of IKK\/NF-kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophy","volume":"117","author":"Acharyya","year":"2007","journal-title":"J Clin Invest"},{"key":"10.1016\/j.nmd.2022.10.007_bib0045","doi-asserted-by":"crossref","first-page":"1174","DOI":"10.1038\/nm.3655","article-title":"Inhibition of JAK-STAT signaling stimulates adult satellite cell function","volume":"20","author":"Price","year":"2014","journal-title":"Nat Med"},{"key":"10.1016\/j.nmd.2022.10.007_bib0046","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1016\/j.ebiom.2015.02.014","article-title":"Functional and Morphological Improvement of Dystrophic Muscle by Interleukin 6 Receptor Blockade","volume":"2","author":"Pelosi","year":"2015","journal-title":"EBioMedicine"},{"key":"10.1016\/j.nmd.2022.10.007_bib0047","doi-asserted-by":"crossref","first-page":"1137","DOI":"10.1172\/JCI118101","article-title":"Expression of transforming growth factor-beta 1 in dystrophic patient muscles correlates with fibrosis. Pathogenetic role of a fibrogenic cytokine","volume":"96","author":"Bernasconi","year":"1995","journal-title":"J Clin Invest"},{"key":"10.1016\/j.nmd.2022.10.007_bib0048","doi-asserted-by":"crossref","first-page":"209","DOI":"10.1002\/jcsm.12665","article-title":"Myogenesis modelled by human pluripotent stem cells: a multi-omic study of Duchenne myopathy early onset","volume":"12","author":"Mournetas","year":"2021","journal-title":"J Cachexia Sarcopenia Muscle"},{"key":"10.1016\/j.nmd.2022.10.007_bib0049","doi-asserted-by":"crossref","first-page":"1145","DOI":"10.5966\/sctm.2015-0224","article-title":"A human pluripotent stem cell model of facioscapulohumeral muscular dystrophy-affected skeletal muscles","volume":"5","author":"Caron","year":"2016","journal-title":"Stem Cells Transl Med"},{"key":"10.1016\/j.nmd.2022.10.007_bib0050","doi-asserted-by":"crossref","first-page":"3943","DOI":"10.1111\/j.1432-1033.2004.04332.x","article-title":"Subproteomics analysis of Ca+-binding proteins demonstrates decreased calsequestrin expression in dystrophic mouse skeletal muscle","volume":"271","author":"Doran","year":"2004","journal-title":"Eur J Biochem"},{"key":"10.1016\/j.nmd.2022.10.007_bib0051","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1085\/jgp.201010592","article-title":"Measurement of RyR permeability reveals a role of calsequestrin in termination of SR Ca(2+) release in skeletal muscle","volume":"138","author":"Sztretye","year":"2011","journal-title":"J Gen Physiol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0052","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1038\/s41419-020-2484-2","article-title":"CACNA1H downregulation induces skeletal muscle atrophy involving endoplasmic reticulum stress activation and autophagy flux blockade","volume":"11","author":"Li","year":"2020","journal-title":"Cell Death Dis"},{"key":"10.1016\/j.nmd.2022.10.007_bib0053","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1113\/jphysiol.2004.061291","article-title":"The action potential-evoked sarcoplasmic reticulum calcium release is impaired in mdx mouse muscle fibres","volume":"557","author":"Woods","year":"2004","journal-title":"J Physiol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0054","doi-asserted-by":"crossref","first-page":"C1077","DOI":"10.1152\/ajpcell.00428.2009","article-title":"Excitation-contraction coupling alterations in mdx and utrophin\/dystrophin double knockout mice: a comparative study","volume":"298","author":"Capote","year":"2010","journal-title":"Am J Physiol Cell Physiol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0055","doi-asserted-by":"crossref","first-page":"1047","DOI":"10.1038\/sj.bjp.0703907","article-title":"Alteration of excitation-contraction coupling mechanism in extensor digitorum longus muscle fibres of dystrophic mdx mouse and potential efficacy of taurine","volume":"132","author":"De Luca","year":"2001","journal-title":"Br J Pharmacol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0056","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1186\/s13395-016-0087-5","article-title":"Characterization of a multiprotein complex involved in excitation-transcription coupling of skeletal muscle","volume":"6","author":"Arias-Calder\u00f3n","year":"2016","journal-title":"Skelet Muscle"},{"key":"10.1016\/j.nmd.2022.10.007_bib0057","doi-asserted-by":"crossref","first-page":"1763","DOI":"10.1093\/hmg\/ddr059","article-title":"Zebrafish Fukutin family proteins link the unfolded protein response with dystroglycanopathies","volume":"20","author":"Lin","year":"2011","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.nmd.2022.10.007_bib0058","doi-asserted-by":"crossref","first-page":"5325","DOI":"10.1093\/hmg\/ddu249","article-title":"Caspase-12 ablation preserves muscle function in the mdx mouse","volume":"23","author":"Moorwood","year":"2014","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.nmd.2022.10.007_bib0059","doi-asserted-by":"crossref","first-page":"394","DOI":"10.1038\/nature10980","article-title":"Hsp72 preserves muscle function and slows progression of severe muscular dystrophy","volume":"484","author":"Gehrig","year":"2012","journal-title":"Nature"},{"key":"10.1016\/j.nmd.2022.10.007_bib0060","doi-asserted-by":"crossref","first-page":"690","DOI":"10.3389\/fphys.2020.00690","article-title":"Mitochondrial dysfunction is an early consequence of partial or complete dystrophin loss in mdx mice","volume":"11","author":"Moore","year":"2020","journal-title":"Front Physiol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0061","doi-asserted-by":"crossref","first-page":"1044","DOI":"10.1172\/JCI43844","article-title":"Mitigation of muscular dystrophy in mice by SERCA overexpression in skeletal muscle","volume":"121","author":"Goonasekera","year":"2011","journal-title":"J Clin Invest"},{"key":"10.1016\/j.nmd.2022.10.007_bib0062","doi-asserted-by":"crossref","first-page":"C699","DOI":"10.1152\/ajpcell.00341.2014","article-title":"SERCA1 overexpression minimizes skeletal muscle damage in dystrophic mouse models","volume":"308","author":"M\u00e1zala","year":"2015","journal-title":"Am J Physiol Cell Physiol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0063","doi-asserted-by":"crossref","first-page":"845","DOI":"10.1016\/j.ymthe.2019.12.011","article-title":"Single SERCA2a therapy ameliorated dilated cardiomyopathy for 18 months in a mouse model of duchenne muscular dystrophy","volume":"28","author":"Wasala","year":"2020","journal-title":"Mol Ther"},{"key":"10.1016\/j.nmd.2022.10.007_bib0064","doi-asserted-by":"crossref","first-page":"1068","DOI":"10.1038\/s41467-017-01146-7","article-title":"Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice","volume":"8","author":"Voit","year":"2017","journal-title":"Nat Commun"},{"key":"10.1016\/j.nmd.2022.10.007_bib0065","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1146\/annurev-pharmtox-010919-023309","article-title":"Unlocking personalized biomedicine and drug discovery with human induced pluripotent stem cell-derived cardiomyocytes: fit for purpose or forever elusive?","volume":"60","author":"de Korte","year":"2020","journal-title":"Annu Rev Pharmacol Toxicol"},{"key":"10.1016\/j.nmd.2022.10.007_bib0066","doi-asserted-by":"crossref","first-page":"e95918","DOI":"10.1172\/jci.insight.95918","article-title":"Functional correction of dystrophin actin binding domain mutations by genome editing","volume":"2","author":"Kyrychenko","year":"2017","journal-title":"JCI Insight"},{"key":"10.1016\/j.nmd.2022.10.007_bib0067","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1212\/WNL.0000000000000669","article-title":"The burden of Duchenne muscular dystrophy: an international, cross-sectional study","volume":"83","author":"Landfeldt","year":"2014","journal-title":"Neurology"},{"key":"10.1016\/j.nmd.2022.10.007_bib0068","doi-asserted-by":"crossref","first-page":"2123","DOI":"10.1212\/WNL.0000000000003217","article-title":"Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy","volume":"87","author":"Griggs","year":"2016","journal-title":"Neurology"}],"container-title":["Neuromuscular Disorders"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0960896622007027?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0960896622007027?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,18]],"date-time":"2025-10-18T08:13:10Z","timestamp":1760775190000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0960896622007027"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,12]]},"references-count":68,"journal-issue":{"issue":"11-12","published-print":{"date-parts":[[2022,12]]}},"alternative-id":["S0960896622007027"],"URL":"https:\/\/doi.org\/10.1016\/j.nmd.2022.10.007","relation":{"has-preprint":[{"id-type":"doi","id":"10.1101\/2022.02.17.480850","asserted-by":"object"}]},"ISSN":["0960-8966"],"issn-type":[{"value":"0960-8966","type":"print"}],"subject":[],"published":{"date-parts":[[2022,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"CRISPR-mediated correction of skeletal muscle Ca2+ handling in a novel DMD patient-derived pluripotent stem cell model","name":"articletitle","label":"Article Title"},{"value":"Neuromuscular Disorders","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.nmd.2022.10.007","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 The Author(s). Published by Elsevier B.V.","name":"copyright","label":"Copyright"}]}}