{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T14:08:18Z","timestamp":1791986898171,"version":"4.4.0"},"publisher-location":"New York, NY","reference-count":36,"publisher":"Springer US","isbn-type":[{"value":"9781071627716","type":"print"},{"value":"9781071627723","type":"electronic"}],"license":[{"start":{"date-parts":[[2022,11,19]],"date-time":"2022-11-19T00:00:00Z","timestamp":1668816000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2022,11,19]],"date-time":"2022-11-19T00:00:00Z","timestamp":1668816000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2023]]},"DOI":"10.1007\/978-1-0716-2772-3_21","type":"book-chapter","created":{"date-parts":[[2022,11,18]],"date-time":"2022-11-18T18:07:03Z","timestamp":1668794823000},"page":"411-425","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["CRISPR-Cas9 Correction of Duchenne Muscular Dystrophy in Mice by a Self-Complementary AAV Delivery System"],"prefix":"10.1007","author":[{"given":"Yu","family":"Zhang","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rhonda","family":"Bassel-Duby","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric N.","family":"Olson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2022,11,19]]},"reference":[{"issue":"6","key":"21_CR1","doi-asserted-by":"publisher","first-page":"919","DOI":"10.1016\/0092-8674(87)90579-4","volume":"51","author":"EP Hoffman","year":"1987","unstructured":"Hoffman EP, Brown RH Jr, Kunkel LM (1987) Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 51(6):919\u2013928","journal-title":"Cell"},{"issue":"3","key":"21_CR2","doi-asserted-by":"publisher","first-page":"509","DOI":"10.1016\/0092-8674(87)90504-6","volume":"50","author":"M Koenig","year":"1987","unstructured":"Koenig M, Hoffman EP, Bertelson CJ et al (1987) Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals. Cell 50(3):509\u2013517","journal-title":"Cell"},{"issue":"3","key":"21_CR3","doi-asserted-by":"publisher","first-page":"1223","DOI":"10.1002\/cphy.c140048","volume":"5","author":"QQ Gao","year":"2015","unstructured":"Gao QQ, McNally EM (2015) The dystrophin complex: structure, function, and implications for therapy. Compr Physiol 5(3):1223\u20131239. https:\/\/doi.org\/10.1002\/cphy.c140048","journal-title":"Compr Physiol"},{"key":"21_CR4","doi-asserted-by":"publisher","first-page":"281","DOI":"10.1146\/annurev-genom-090314-025003","volume":"16","author":"S Guiraud","year":"2015","unstructured":"Guiraud S, Aartsma-Rus A, Vieira NM et al (2015) The pathogenesis and therapy of muscular dystrophies. Annu Rev Genomics Hum Genet 16:281\u2013308. https:\/\/doi.org\/10.1146\/annurev-genom-090314-025003","journal-title":"Annu Rev Genomics Hum Genet"},{"key":"21_CR5","doi-asserted-by":"publisher","unstructured":"Quattrocelli M, Zelikovich AS, Jiang Z et al (2019) Pulsed glucocorticoids enhance dystrophic muscle performance through epigenetic-metabolic reprogramming. JCI Insight 4(24). https:\/\/doi.org\/10.1172\/jci.insight.132402","DOI":"10.1172\/jci.insight.132402"},{"key":"21_CR6","doi-asserted-by":"publisher","first-page":"533","DOI":"10.2147\/DDDT.S97635","volume":"11","author":"KR Lim","year":"2017","unstructured":"Lim KR, Maruyama R, Yokota T (2017) Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Devel Ther 11:533\u2013545. https:\/\/doi.org\/10.2147\/DDDT.S97635","journal-title":"Drug Des Devel Ther"},{"issue":"10","key":"21_CR7","doi-asserted-by":"publisher","first-page":"2337","DOI":"10.1016\/j.ymthe.2018.07.011","volume":"26","author":"D Duan","year":"2018","unstructured":"Duan D (2018) Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy. Mol Ther 26(10):2337\u20132356. https:\/\/doi.org\/10.1016\/j.ymthe.2018.07.011","journal-title":"Mol Ther"},{"issue":"6121","key":"21_CR8","doi-asserted-by":"publisher","first-page":"819","DOI":"10.1126\/science.1231143","volume":"339","author":"L Cong","year":"2013","unstructured":"Cong L, Ran FA, Cox D et al (2013) Multiplex genome engineering using CRISPR\/Cas systems. Science 339(6121):819\u2013823. https:\/\/doi.org\/10.1126\/science.1231143","journal-title":"Science"},{"issue":"6096","key":"21_CR9","doi-asserted-by":"publisher","first-page":"816","DOI":"10.1126\/science.1225829","volume":"337","author":"M Jinek","year":"2012","unstructured":"Jinek M, Chylinski K, Fonfara I et al (2012) A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337(6096):816\u2013821. https:\/\/doi.org\/10.1126\/science.1225829","journal-title":"Science"},{"issue":"6121","key":"21_CR10","doi-asserted-by":"publisher","first-page":"823","DOI":"10.1126\/science.1232033","volume":"339","author":"P Mali","year":"2013","unstructured":"Mali P, Yang L, Esvelt KM et al (2013) RNA-guided human genome engineering via Cas9. Science 339(6121):823\u2013826. https:\/\/doi.org\/10.1126\/science.1232033","journal-title":"Science"},{"issue":"7546","key":"21_CR11","doi-asserted-by":"publisher","first-page":"186","DOI":"10.1038\/nature14299","volume":"520","author":"FA Ran","year":"2015","unstructured":"Ran FA, Cong L, Yan WX et al (2015) In vivo genome editing using Staphylococcus aureus Cas9. Nature 520(7546):186\u2013191. https:\/\/doi.org\/10.1038\/nature14299","journal-title":"Nature"},{"issue":"3","key":"21_CR12","doi-asserted-by":"publisher","first-page":"759","DOI":"10.1016\/j.cell.2015.09.038","volume":"163","author":"B Zetsche","year":"2015","unstructured":"Zetsche B, Gootenberg JS, Abudayyeh OO et al (2015) Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system. Cell 163(3):759\u2013771. https:\/\/doi.org\/10.1016\/j.cell.2015.09.038","journal-title":"Cell"},{"issue":"7753","key":"21_CR13","doi-asserted-by":"publisher","first-page":"561","DOI":"10.1038\/s41586-019-1076-8","volume":"568","author":"S Iyer","year":"2019","unstructured":"Iyer S, Suresh S, Guo D et al (2019) Precise therapeutic gene correction by a simple nuclease-induced double-stranded break. Nature 568(7753):561\u2013565. https:\/\/doi.org\/10.1038\/s41586-019-1076-8","journal-title":"Nature"},{"issue":"2","key":"21_CR14","doi-asserted-by":"publisher","first-page":"397","DOI":"10.1021\/acschembio.7b00760","volume":"13","author":"DN Gallagher","year":"2018","unstructured":"Gallagher DN, Haber JE (2018) Repair of a site-specific DNA cleavage: old-school lessons for Cas9-mediated gene editing. ACS Chem Biol 13(2):397\u2013405. https:\/\/doi.org\/10.1021\/acschembio.7b00760","journal-title":"ACS Chem Biol"},{"issue":"6","key":"21_CR15","doi-asserted-by":"publisher","first-page":"2766","DOI":"10.1172\/JCI136873","volume":"130","author":"F Chemello","year":"2020","unstructured":"Chemello F, Bassel-Duby R, Olson EN (2020) Correction of muscular dystrophies by CRISPR gene editing. J Clin Invest 130(6):2766\u20132776. https:\/\/doi.org\/10.1172\/JCI136873","journal-title":"J Clin Invest"},{"key":"21_CR16","doi-asserted-by":"publisher","first-page":"239","DOI":"10.1146\/annurev-med-081117-010451","volume":"70","author":"YL Min","year":"2019","unstructured":"Min YL, Bassel-Duby R, Olson EN (2019) CRISPR correction of Duchenne muscular dystrophy. Annu Rev Med 70:239\u2013255. https:\/\/doi.org\/10.1146\/annurev-med-081117-010451","journal-title":"Annu Rev Med"},{"issue":"3","key":"21_CR17","doi-asserted-by":"publisher","first-page":"1205","DOI":"10.1152\/physrev.00046.2017","volume":"98","author":"Y Zhang","year":"2018","unstructured":"Zhang Y, Long C, Bassel-Duby R et al (2018) Myoediting: toward prevention of muscular dystrophy by therapeutic genome editing. Physiol Rev 98(3):1205\u20131240. https:\/\/doi.org\/10.1152\/physrev.00046.2017","journal-title":"Physiol Rev"},{"issue":"8","key":"21_CR18","doi-asserted-by":"publisher","first-page":"1011","DOI":"10.1212\/wnl.39.8.1011","volume":"39","author":"EP Hoffman","year":"1989","unstructured":"Hoffman EP, Kunkel LM, Angelini C et al (1989) Improved diagnosis of Becker muscular dystrophy by dystrophin testing. Neurology 39(8):1011\u20131017. https:\/\/doi.org\/10.1212\/wnl.39.8.1011","journal-title":"Neurology"},{"issue":"6410","key":"21_CR19","doi-asserted-by":"publisher","first-page":"86","DOI":"10.1126\/science.aau1549","volume":"362","author":"L Amoasii","year":"2018","unstructured":"Amoasii L, Hildyard JCW, Li H et al (2018) Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy. Science 362(6410):86\u201391. https:\/\/doi.org\/10.1126\/science.aau1549","journal-title":"Science"},{"issue":"1","key":"21_CR20","doi-asserted-by":"publisher","first-page":"4537","DOI":"10.1038\/s41467-019-12335-x","volume":"10","author":"L Amoasii","year":"2019","unstructured":"Amoasii L, Li H, Zhang Y et al (2019) In vivo non-invasive monitoring of dystrophin correction in a new Duchenne muscular dystrophy reporter mouse. Nat Commun 10(1):4537. https:\/\/doi.org\/10.1038\/s41467-019-12335-x","journal-title":"Nat Commun"},{"key":"21_CR21","doi-asserted-by":"publisher","unstructured":"Amoasii L, Long C, Li H et al (2017) Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy. Sci Transl Med 9(418). https:\/\/doi.org\/10.1126\/scitranslmed.aan8081","DOI":"10.1126\/scitranslmed.aan8081"},{"key":"21_CR22","doi-asserted-by":"publisher","first-page":"14454","DOI":"10.1038\/ncomms14454","volume":"8","author":"NE Bengtsson","year":"2017","unstructured":"Bengtsson NE, Hall JK, Odom GL et al (2017) Muscle-specific CRISPR\/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy. Nat Commun 8:14454. https:\/\/doi.org\/10.1038\/ncomms14454","journal-title":"Nat Commun"},{"key":"21_CR23","doi-asserted-by":"publisher","unstructured":"Hakim CH, Wasala NB, Nelson CE et al (2018) AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice. JCI Insight 3(23). https:\/\/doi.org\/10.1172\/jci.insight.124297","DOI":"10.1172\/jci.insight.124297"},{"issue":"6271","key":"21_CR24","doi-asserted-by":"publisher","first-page":"400","DOI":"10.1126\/science.aad5725","volume":"351","author":"C Long","year":"2016","unstructured":"Long C, Amoasii L, Mireault AA et al (2016) Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science 351(6271):400\u2013403. https:\/\/doi.org\/10.1126\/science.aad5725","journal-title":"Science"},{"issue":"9","key":"21_CR25","doi-asserted-by":"publisher","first-page":"2044","DOI":"10.1016\/j.ymthe.2020.05.024","volume":"28","author":"YL Min","year":"2020","unstructured":"Min YL, Chemello F, Li H et al (2020) Correction of three prominent mutations in mouse and human models of Duchenne muscular dystrophy by single-cut genome editing. Mol Ther 28(9):2044\u20132055. https:\/\/doi.org\/10.1016\/j.ymthe.2020.05.024","journal-title":"Mol Ther"},{"issue":"3","key":"21_CR26","doi-asserted-by":"publisher","first-page":"eaav4324","DOI":"10.1126\/sciadv.aav4324","volume":"5","author":"YL Min","year":"2019","unstructured":"Min YL, Li H, Rodriguez-Caycedo C et al (2019) CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells. Sci Adv 5(3):eaav4324. https:\/\/doi.org\/10.1126\/sciadv.aav4324","journal-title":"Sci Adv"},{"issue":"6271","key":"21_CR27","doi-asserted-by":"publisher","first-page":"403","DOI":"10.1126\/science.aad5143","volume":"351","author":"CE Nelson","year":"2016","unstructured":"Nelson CE, Hakim CH, Ousterout DG et al (2016) In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science 351(6271):403\u2013407. https:\/\/doi.org\/10.1126\/science.aad5143","journal-title":"Science"},{"issue":"3","key":"21_CR28","doi-asserted-by":"publisher","first-page":"427","DOI":"10.1038\/s41591-019-0344-3","volume":"25","author":"CE Nelson","year":"2019","unstructured":"Nelson CE, Wu Y, Gemberling MP et al (2019) Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy. Nat Med 25(3):427\u2013432. https:\/\/doi.org\/10.1038\/s41591-019-0344-3","journal-title":"Nat Med"},{"issue":"6271","key":"21_CR29","doi-asserted-by":"publisher","first-page":"407","DOI":"10.1126\/science.aad5177","volume":"351","author":"M Tabebordbar","year":"2016","unstructured":"Tabebordbar M, Zhu K, Cheng JKW et al (2016) In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 351(6271):407\u2013411. https:\/\/doi.org\/10.1126\/science.aad5177","journal-title":"Science"},{"issue":"2","key":"21_CR30","doi-asserted-by":"publisher","first-page":"207","DOI":"10.1038\/s41591-019-0738-2","volume":"26","author":"A Moretti","year":"2020","unstructured":"Moretti A, Fonteyne L, Giesert F et al (2020) Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy. Nat Med 26(2):207\u2013214. https:\/\/doi.org\/10.1038\/s41591-019-0738-2","journal-title":"Nat Med"},{"issue":"8","key":"21_CR31","doi-asserted-by":"publisher","first-page":"eaay6812","DOI":"10.1126\/sciadv.aay6812","volume":"6","author":"Y Zhang","year":"2020","unstructured":"Zhang Y, Li H, Min YL et al (2020) Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system. Sci Adv 6(8):eaay6812. https:\/\/doi.org\/10.1126\/sciadv.aay6812","journal-title":"Sci Adv"},{"issue":"26","key":"21_CR32","doi-asserted-by":"publisher","first-page":"2112","DOI":"10.1038\/sj.gt.3302134","volume":"10","author":"DM McCarty","year":"2003","unstructured":"McCarty DM, Fu H, Monahan PE et al (2003) Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther 10(26):2112\u20132118. https:\/\/doi.org\/10.1038\/sj.gt.3302134","journal-title":"Gene Ther"},{"issue":"5","key":"21_CR33","doi-asserted-by":"publisher","first-page":"3227","DOI":"10.1128\/jvi.70.5.3227-3234.1996","volume":"70","author":"FK Ferrari","year":"1996","unstructured":"Ferrari FK, Samulski T, Shenk T et al (1996) Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol 70(5):3227\u20133234","journal-title":"J Virol"},{"issue":"1","key":"21_CR34","doi-asserted-by":"publisher","first-page":"520","DOI":"10.1128\/jvi.70.1.520-532.1996","volume":"70","author":"KJ Fisher","year":"1996","unstructured":"Fisher KJ, Gao GP, Weitzman MD et al (1996) Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol 70(1):520\u2013532","journal-title":"J Virol"},{"issue":"9","key":"21_CR35","doi-asserted-by":"publisher","first-page":"1047","DOI":"10.1089\/hum.2005.16.1047","volume":"16","author":"C Ren","year":"2005","unstructured":"Ren C, Kumar S, Shaw DR et al (2005) Genomic stability of self-complementary adeno-associated virus 2 during early stages of transduction in mouse muscle in vivo. Hum Gene Ther 16(9):1047\u20131057. https:\/\/doi.org\/10.1089\/hum.2005.16.1047","journal-title":"Hum Gene Ther"},{"issue":"10","key":"21_CR36","doi-asserted-by":"publisher","first-page":"1648","DOI":"10.1038\/mt.2008.171","volume":"16","author":"DM McCarty","year":"2008","unstructured":"McCarty DM (2008) Self-complementary AAV vectors; advances and applications. Mol Ther 16(10):1648\u20131656. https:\/\/doi.org\/10.1038\/mt.2008.171","journal-title":"Mol Ther"}],"container-title":["Methods in Molecular Biology","Muscular Dystrophy Therapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/978-1-0716-2772-3_21","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,3,1]],"date-time":"2023-03-01T07:08:15Z","timestamp":1677654495000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/978-1-0716-2772-3_21"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,11,19]]},"ISBN":["9781071627716","9781071627723"],"references-count":36,"URL":"https:\/\/doi.org\/10.1007\/978-1-0716-2772-3_21","relation":{},"ISSN":["1064-3745","1940-6029"],"issn-type":[{"value":"1064-3745","type":"print"},{"value":"1940-6029","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,11,19]]},"assertion":[{"value":"19 November 2022","order":1,"name":"first_online","label":"First Online","group":{"name":"ChapterHistory","label":"Chapter History"}}]}}