{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,17]],"date-time":"2026-09-17T03:57:33Z","timestamp":1789617453670,"version":"build-2803163510"},"reference-count":34,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,1,3]],"date-time":"2025-01-03T00:00:00Z","timestamp":1735862400000},"content-version":"vor","delay-in-days":368,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Global Medical Genetics"],"published-print":{"date-parts":[[2024]]},"DOI":"10.1055\/s-0044-1791803","type":"journal-article","created":{"date-parts":[[2024,10,18]],"date-time":"2024-10-18T21:28:22Z","timestamp":1729286902000},"page":"349-357","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":10,"title":["The Role of CRISPR\/Cas9 in Revolutionizing Duchenne's Muscular Dystrophy Treatment: Opportunities and Obstacles"],"prefix":"10.1016","volume":"11","author":[{"given":"Ahsan","family":"Ali","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Md Yakeen","family":"Rahman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Danish","family":"Sheikh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"05","key":"10.1055\/s-0044-1791803_bib2","doi-asserted-by":"crossref","first-page":"1125","DOI":"10.1016\/j.ymthe.2017.02.019","article-title":"Progress toward gene therapy for Duchenne muscular dystrophy","volume":"25","author":"Chamberlain","year":"2017","journal-title":"Mol Ther"},{"issue":"06","key":"10.1055\/s-0044-1791803_bib3","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1016\/0092-8674(87)90579-4","article-title":"Dystrophin: the protein product of the Duchenne muscular dystrophy locus","volume":"51","author":"Hoffman","year":"1987","journal-title":"Cell"},{"issue":"01","key":"10.1055\/s-0044-1791803_bib4","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"},{"issue":"02","key":"10.1055\/s-0044-1791803_bib5","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1089\/humc.2017.075","article-title":"A new kid on the playground of CRISPR DMD therapy","volume":"28","author":"Duan","year":"2017","journal-title":"Hum Gene Ther Clin Dev"},{"issue":"01","key":"10.1055\/s-0044-1791803_bib6","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0228072","article-title":"Challenges associated with homologous directed repair using CRISPR-Cas9 and TALEN to edit the DMD genetic mutation in canine Duchenne muscular dystrophy","volume":"15","author":"Mata L\u00f3pez","year":"2020","journal-title":"PLoS One"},{"issue":"01","key":"10.1055\/s-0044-1791803_bib7","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-018-04252-2","article-title":"The CRISPR tool kit for genome editing and beyond","volume":"9","author":"Adli","year":"2018","journal-title":"Nat Commun"},{"issue":"11","key":"10.1055\/s-0044-1791803_bib8","doi-asserted-by":"crossref","first-page":"777","DOI":"10.1161\/CIRCULATIONAHA.116.024007","article-title":"CRISPR (clustered regularly interspaced palindromic repeat)\/Cas9 system: a revolutionary disease-modifying technology","volume":"134","author":"Munshi","year":"2016","journal-title":"Circulation"},{"issue":"6268","key":"10.1055\/s-0044-1791803_bib9","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1126\/science.aad5227","article-title":"Rationally engineered Cas9 nucleases with improved specificity","volume":"351","author":"Slaymaker","year":"2016","journal-title":"Science"},{"issue":"03","key":"10.1055\/s-0044-1791803_bib10","doi-asserted-by":"crossref","first-page":"759","DOI":"10.1016\/j.cell.2015.09.038","article-title":"Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system","volume":"163","author":"Zetsche","year":"2015","journal-title":"Cell"},{"issue":"02","key":"10.1055\/s-0044-1791803_bib11","doi-asserted-by":"crossref","first-page":"184","DOI":"10.1038\/nbt.3437","article-title":"Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9","volume":"34","author":"Doench","year":"2016","journal-title":"Nat Biotechnol"},{"issue":"12","key":"10.1055\/s-0044-1791803_bib12","doi-asserted-by":"crossref","first-page":"770","DOI":"10.1038\/s41576-018-0059-1","article-title":"Base editing: precision chemistry on the genome and transcriptome of living cells","volume":"19","author":"Rees","year":"2018","journal-title":"Nat Rev Genet"},{"issue":"7785","key":"10.1055\/s-0044-1791803_bib13","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"issue":"06","key":"10.1055\/s-0044-1791803_bib14","doi-asserted-by":"crossref","first-page":"536","DOI":"10.1038\/nbt.4148","article-title":"Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy","volume":"36","author":"Ryu","year":"2018","journal-title":"Nat Biotechnol"},{"issue":"01","key":"10.1055\/s-0044-1791803_bib15","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1038\/s41587-020-0609-x","article-title":"CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells","volume":"39","author":"Kurt","year":"2021","journal-title":"Nat Biotechnol"},{"issue":"7587","key":"10.1055\/s-0044-1791803_bib16","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1038\/nature16526","article-title":"High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects","volume":"529","author":"Kleinstiver","year":"2016","journal-title":"Nature"},{"issue":"6201","key":"10.1055\/s-0044-1791803_bib17","doi-asserted-by":"crossref","first-page":"1184","DOI":"10.1126\/science.1254445","article-title":"Prevention of muscular dystrophy in mice by CRISPR\/Cas9-mediated editing of germline DNA","volume":"345","author":"Long","year":"2014","journal-title":"Science"},{"issue":"6410","key":"10.1055\/s-0044-1791803_bib18","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"},{"issue":"02","key":"10.1055\/s-0044-1791803_bib19","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1038\/s41591-019-0738-2","article-title":"Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy","volume":"26","author":"Moretti","year":"2020","journal-title":"Nat Med"},{"issue":"6271","key":"10.1055\/s-0044-1791803_bib20","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1126\/science.aad5143","article-title":"In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy","volume":"351","author":"Nelson","year":"2016","journal-title":"Science"},{"issue":"03","key":"10.1055\/s-0044-1791803_bib21","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1056\/NEJMoa2031054","article-title":"CRISPR-Cas9 gene editing for sickle cell disease and \u03b2-thalassemia","volume":"384","author":"Frangoul","year":"2021","journal-title":"N Engl J Med"},{"issue":"03","key":"10.1055\/s-0044-1791803_bib22","doi-asserted-by":"crossref","first-page":"205","DOI":"10.1056\/NEJMoa2029392","article-title":"Post-transcriptional genetic silencing of BCL11A to treat sickle cell disease","volume":"384","author":"Esrick","year":"2021","journal-title":"N Engl J Med"},{"issue":"01","key":"10.1055\/s-0044-1791803_bib23","doi-asserted-by":"crossref","DOI":"10.1038\/srep37051","article-title":"Adenoviral vectors encoding CRISPR\/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells","volume":"6","author":"Maggio","year":"2016","journal-title":"Sci Rep"},{"issue":"04","key":"10.1055\/s-0044-1791803_bib24","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.1602814","article-title":"CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice","volume":"3","author":"Zhang","year":"2017","journal-title":"Sci Adv"},{"issue":"04","key":"10.1055\/s-0044-1791803_bib25","doi-asserted-by":"crossref","first-page":"41","DOI":"10.3390\/jpm8040041","article-title":"Multiple exon skipping in the Duchenne muscular dystrophy hot spots: prospects and challenges","volume":"8","author":"Echigoya","year":"2018","journal-title":"J Pers Med"},{"issue":"01","key":"10.1055\/s-0044-1791803_bib26","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-021-83259-0","article-title":"Construction of adenovirus vectors simultaneously expressing four multiplex, double-nicking guide RNAs of CRISPR\/Cas9 and in vivo genome editing","volume":"11","author":"Nakanishi","year":"2021","journal-title":"Sci Rep"},{"key":"10.1055\/s-0044-1791803_bib27","doi-asserted-by":"crossref","DOI":"10.1016\/j.dmpk.2022.100450","article-title":"Recent advances in lipid nanoparticles for delivery of nucleic acid, mRNA, and gene editing-based therapeutics","volume":"44","author":"Mukai","year":"2022","journal-title":"Drug Metab Pharmacokinet"},{"issue":"09","key":"10.1055\/s-0044-1791803_bib28","doi-asserted-by":"crossref","first-page":"812","DOI":"10.1182\/blood.2022019333","article-title":"Lipid nanoparticles allow efficient and harmless ex vivo gene editing of human hematopoietic cells","volume":"142","author":"Vavassori","year":"2023","journal-title":"Blood"},{"issue":"05","key":"10.1055\/s-0044-1791803_bib29","doi-asserted-by":"crossref","first-page":"626","DOI":"10.3390\/ijms17050626","article-title":"In vivo delivery systems for therapeutic genome editing","volume":"17","author":"Wang","year":"2016","journal-title":"Int J Mol Sci"},{"issue":"03","key":"10.1055\/s-0044-1791803_bib30","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1038\/s41591-019-0344-3","article-title":"Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy","volume":"25","author":"Nelson","year":"2019","journal-title":"Nat Med"},{"issue":"11","key":"10.1055\/s-0044-1791803_bib31","doi-asserted-by":"crossref","first-page":"972","DOI":"10.3390\/cells13110972","article-title":"A novel CRISPR-Cas9 strategy to target dystrophin mutations downstream of exon 44 in patient-specific DMD iPSCs","volume":"13","author":"Dhoke","year":"2024","journal-title":"Cells"},{"issue":"04","key":"10.1055\/s-0044-1791803_bib32","doi-asserted-by":"crossref","first-page":"395","DOI":"10.1002\/humu.22758","article-title":"The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations","volume":"36","author":"Bladen","year":"2015","journal-title":"Hum Mutat"},{"issue":"01","key":"10.1055\/s-0044-1791803_bib33","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1016\/j.jmb.2018.05.044","article-title":"CRISPR ethics: Moral considerations for applications of a powerful tool","volume":"431","author":"Brokowski","year":"2019","journal-title":"J Mol Biol"},{"issue":"08","key":"10.1055\/s-0044-1791803_bib34","doi-asserted-by":"crossref","first-page":"1651","DOI":"10.1111\/pbi.13383","article-title":"Genome editing with the CRISPR-Cas system: an art, ethics and global regulatory perspective","volume":"18","author":"Zhang","year":"2020","journal-title":"Plant Biotechnol J"},{"issue":"6271","key":"10.1055\/s-0044-1791803_bib35","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"}],"container-title":["Global Medical Genetics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2699940425000359?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2699940425000359?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/www.thieme-connect.de\/products\/ejournals\/pdf\/10.1055\/s-0044-1791803.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,23]],"date-time":"2025-10-23T07:37:17Z","timestamp":1761205037000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2699940425000359"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024]]},"references-count":34,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2024]]}},"alternative-id":["S2699940425000359"],"URL":"https:\/\/doi.org\/10.1055\/s-0044-1791803","relation":{},"ISSN":["2699-9404"],"issn-type":[{"value":"2699-9404","type":"print"}],"subject":[],"published":{"date-parts":[[2024]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"The Role of CRISPR\/Cas9 in Revolutionizing Duchenne's Muscular Dystrophy Treatment: Opportunities and Obstacles","name":"articletitle","label":"Article Title"},{"value":"Global Medical Genetics","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1055\/s-0044-1791803","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2024 International Knowledge Direct Medical Associations. Publishing services by Elsevier B.V. on behalf of KeAi Communications Co. Ltd. Published by Elsevier B.V.","name":"copyright","label":"Copyright"}]}}