{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,15]],"date-time":"2026-06-15T06:54:21Z","timestamp":1781506461284,"version":"3.54.1"},"publisher-location":"New York, NY","reference-count":19,"publisher":"Springer US","isbn-type":[{"value":"9781071648100","type":"print"},{"value":"9781071648117","type":"electronic"}],"license":[{"start":{"date-parts":[[2025,10,1]],"date-time":"2025-10-01T00:00:00Z","timestamp":1759276800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2025,10,1]],"date-time":"2025-10-01T00:00:00Z","timestamp":1759276800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026]]},"DOI":"10.1007\/978-1-0716-4811-7_12","type":"book-chapter","created":{"date-parts":[[2025,10,1]],"date-time":"2025-10-01T00:03:15Z","timestamp":1759276995000},"page":"173-189","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Exploring Therapies for Duchenne Muscular Dystrophy Using Transdifferentiated Patient Fibroblasts"],"prefix":"10.1007","author":[{"given":"Camila F.","family":"Almeida","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolas","family":"Wein","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,10,1]]},"reference":[{"key":"12_CR1","doi-asserted-by":"publisher","first-page":"1957","DOI":"10.2147\/DDDT.S129447","volume":"11","author":"M Szabo","year":"2017","unstructured":"Szabo M, Akusj\u00e4rvi SS, Saxena A et al (2017) Cell and small animal models for phenotypic drug discovery. Drug Des Devel Ther 11:1957\u20131967. https:\/\/doi.org\/10.2147\/DDDT.S129447","journal-title":"Drug Des Devel Ther"},{"key":"12_CR2","doi-asserted-by":"publisher","first-page":"e91","DOI":"10.1016\/J.DDTEC.2012.03.001","volume":"10","author":"G Kawahara","year":"2013","unstructured":"Kawahara G, Kunkel LM (2013) Zebrafish based small molecule screens for novel DMD drugs. Drug Discov Today Technol 10:e91\u2013e96. https:\/\/doi.org\/10.1016\/J.DDTEC.2012.03.001","journal-title":"Drug Discov Today Technol"},{"key":"12_CR3","doi-asserted-by":"publisher","DOI":"10.1002\/jgm.3082","volume":"21","author":"A Zarei","year":"2019","unstructured":"Zarei A, Razban V, Hosseini SE, Tabei SMB (2019) Creating cell and animal models of human disease by genome editing using CRISPR\/Cas9. J Gene Med 21:e3082. https:\/\/doi.org\/10.1002\/jgm.3082","journal-title":"J Gene Med"},{"key":"12_CR4","doi-asserted-by":"publisher","first-page":"189","DOI":"10.1042\/bc20070085","volume":"100","author":"A Bigot","year":"2008","unstructured":"Bigot A, Jacquemin V, Debacq-Chainiaux F et al (2008) Replicative aging down-regulates the myogenic regulatory factors in human myoblasts. Biol Cell 100:189\u2013199. https:\/\/doi.org\/10.1042\/bc20070085","journal-title":"Biol Cell"},{"key":"12_CR5","doi-asserted-by":"publisher","unstructured":"Mamchaoui K, Trollet C, Bigot A et al (2011) Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders. Skelet Muscle 1. https:\/\/doi.org\/10.1186\/2044-5040-1-34","DOI":"10.1186\/2044-5040-1-34"},{"key":"12_CR6","doi-asserted-by":"publisher","first-page":"39","DOI":"10.1016\/j.yexcr.2016.02.013","volume":"342","author":"B Pantic","year":"2016","unstructured":"Pantic B, Borgia D, Giunco S et al (2016) Reliable and versatile immortal muscle cell models from healthy and myotonic dystrophy type 1 primary human myoblasts. Exp Cell Res 342:39\u201351. https:\/\/doi.org\/10.1016\/j.yexcr.2016.02.013","journal-title":"Exp Cell Res"},{"key":"12_CR7","doi-asserted-by":"publisher","first-page":"43","DOI":"10.1186\/s13395-016-0115-5","volume":"6","author":"M Thorley","year":"2016","unstructured":"Thorley M, Duguez S, Mazza EMC et al (2016) Skeletal muscle characteristics are preserved in hTERT\/cdk4 human myogenic cell lines. Skelet Muscle 6:43. https:\/\/doi.org\/10.1186\/s13395-016-0115-5","journal-title":"Skelet Muscle"},{"key":"12_CR8","doi-asserted-by":"publisher","first-page":"784","DOI":"10.1089\/hum.2008.163","volume":"20","author":"S Chaouch","year":"2009","unstructured":"Chaouch S, Mouly V, Goyenvalle A et al (2009) Immortalized skin fibroblasts expressing conditional MyoD as a renewable and reliable source of converted human muscle cells to assess therapeutic strategies for muscular dystrophies: validation of an exon-skipping approach to restore dystrophin in duchen. Hum Gene Ther 20:784\u2013790. https:\/\/doi.org\/10.1089\/hum.2008.163","journal-title":"Hum Gene Ther"},{"key":"12_CR9","doi-asserted-by":"publisher","first-page":"R163","DOI":"10.1093\/hmg\/ddy171","volume":"27","author":"L Echevarr\u00eda","year":"2018","unstructured":"Echevarr\u00eda L, Aupy P, Goyenvalle A (2018) Exon-skipping advances for Duchenne muscular dystrophy. Hum Mol Genet 27:R163\u2013R172","journal-title":"Hum Mol Genet"},{"key":"12_CR10","doi-asserted-by":"publisher","DOI":"10.1017\/erm.2019.5","volume":"21","author":"J Hwang","year":"2019","unstructured":"Hwang J, Yokota T (2019) Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies. Expert Rev Mol Med 21:e5","journal-title":"Expert Rev Mol Med"},{"key":"12_CR11","doi-asserted-by":"publisher","first-page":"199","DOI":"10.3233\/JND-170233","volume":"4","author":"N Wein","year":"2017","unstructured":"Wein N, Vulin A, Findlay AR et al (2017) Efficient skipping of single exon duplications in DMD patient-derived cell lines using an antisense oligonucleotide approach. J Neuromuscul Dis 4:199\u2013207. https:\/\/doi.org\/10.3233\/JND-170233","journal-title":"J Neuromuscul Dis"},{"key":"12_CR12","doi-asserted-by":"publisher","first-page":"9456","DOI":"10.1073\/pnas.142302299","volume":"99","author":"FG De Angelis","year":"2002","unstructured":"De Angelis FG, Sthandier O, Berarducci B et al (2002) Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in \u039448-50 DMD cells. Proc Natl Acad Sci USA 99:9456\u20139461. https:\/\/doi.org\/10.1073\/pnas.142302299","journal-title":"Proc Natl Acad Sci USA"},{"key":"12_CR13","doi-asserted-by":"publisher","first-page":"4929","DOI":"10.1073\/pnas.95.9.4929","volume":"95","author":"L Gorman","year":"1998","unstructured":"Gorman L, Suter D, Emerick V et al (1998) Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs. Proc Natl Acad Sci USA 95:4929\u20134934. https:\/\/doi.org\/10.1073\/pnas.95.9.4929","journal-title":"Proc Natl Acad Sci USA"},{"key":"12_CR14","doi-asserted-by":"publisher","first-page":"992","DOI":"10.1038\/nm.3628","volume":"20","author":"N Wein","year":"2014","unstructured":"Wein N, Vulin A, Falzarano MS et al (2014) Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice. Nat Med 20:992\u20131000. https:\/\/doi.org\/10.1038\/nm.3628","journal-title":"Nat Med"},{"key":"12_CR15","doi-asserted-by":"publisher","unstructured":"Findlay AR, Wein N, Kaminoh Y et al (2015) Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45. Ann Neurol 77. https:\/\/doi.org\/10.1002\/ana.24365","DOI":"10.1002\/ana.24365"},{"key":"12_CR16","doi-asserted-by":"publisher","first-page":"136","DOI":"10.1002\/humu.21160","volume":"31","author":"N Wein","year":"2010","unstructured":"Wein N, Avril A, Bartoli M et al (2010) Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping. Hum Mutat 31:136\u2013142","journal-title":"Hum Mutat"},{"key":"12_CR17","doi-asserted-by":"publisher","first-page":"368","DOI":"10.1016\/j.nmd.2007.01.019","volume":"17","author":"K Aur\u00e9","year":"2007","unstructured":"Aur\u00e9 K, Mamchaoui K, Frachon P et al (2007) Impact on oxidative phosphorylation of immortalization with the telomerase gene. Neuromuscul Disord 17:368\u2013375. https:\/\/doi.org\/10.1016\/j.nmd.2007.01.019","journal-title":"Neuromuscul Disord"},{"key":"12_CR18","doi-asserted-by":"publisher","first-page":"382","DOI":"10.1016\/j.ymthe.2005.09.012","volume":"13","author":"I Barde","year":"2006","unstructured":"Barde I, Zanta-Boussif MA, Paisant S et al (2006) Efficient control of gene expression in the hematopoietic system using a single Tet-on inducible lentiviral vector. Mol Ther 13:382\u2013390. https:\/\/doi.org\/10.1016\/j.ymthe.2005.09.012","journal-title":"Mol Ther"},{"key":"12_CR19","doi-asserted-by":"publisher","unstructured":"Almeida CF, Frair EC, Huang N et al (2021) Direct reprogramming of human fibroblasts into myoblasts to investigate therapies for neuromuscular disorders. J Vis Exp 2021. https:\/\/doi.org\/10.3791\/61991","DOI":"10.3791\/61991"}],"container-title":["Methods in Molecular Biology","Duchenne Muscular Dystrophy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/978-1-0716-4811-7_12","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,6,15]],"date-time":"2026-06-15T06:32:16Z","timestamp":1781505136000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/978-1-0716-4811-7_12"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,10,1]]},"ISBN":["9781071648100","9781071648117"],"references-count":19,"URL":"https:\/\/doi.org\/10.1007\/978-1-0716-4811-7_12","relation":{},"ISSN":["1064-3745","1940-6029"],"issn-type":[{"value":"1064-3745","type":"print"},{"value":"1940-6029","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,10,1]]},"assertion":[{"value":"1 October 2025","order":1,"name":"first_online","label":"First Online","group":{"name":"ChapterHistory","label":"Chapter History"}}]}}