{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T18:02:59Z","timestamp":1786989779129,"version":"build-2736575974"},"reference-count":70,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Gene"],"published-print":{"date-parts":[[2023,5]]},"DOI":"10.1016\/j.gene.2023.147358","type":"journal-article","created":{"date-parts":[[2023,3,11]],"date-time":"2023-03-11T16:17:37Z","timestamp":1678551457000},"page":"147358","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":10,"special_numbering":"C","title":["New advancements in CRISPR based gene therapy of Duchenne muscular dystrophy"],"prefix":"10.1016","volume":"867","author":[{"given":"Atieh","family":"Eslahi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Farzaneh","family":"Alizadeh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amir","family":"Avan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gordon A","family":"Ferns","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Meysam","family":"Moghbeli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohammad","family":"Reza Abbaszadegan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Majid","family":"Mojarrad","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0005","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1089\/nat.2014.1500","article-title":"Antisense-mediated exon skipping: networking to meet opportunities and to overcome challenges","volume":"24","author":"Aartsma-Rus","year":"2014","journal-title":"Nucleic Acid Ther."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0010","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1089\/nat.2016.0657","article-title":"FDA approves eteplirsen for duchenne muscular dystrophy: the next chapter in the eteplirsen saga","volume":"27","author":"Aartsma-Rus","year":"2017","journal-title":"Nucleic Acid Ther."},{"issue":"Pt 12","key":"10.1016\/j.gene.2023.147358_b0015","doi-asserted-by":"crossref","first-page":"3547","DOI":"10.1093\/brain\/awr291","article-title":"Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials","volume":"134","author":"Anthony","year":"2011","journal-title":"Brain"},{"issue":"7785","key":"10.1016\/j.gene.2023.147358_b0020","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":"5","key":"10.1016\/j.gene.2023.147358_b0025","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1038\/s41587-021-01133-w","article-title":"Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing","volume":"40","author":"Anzalone","year":"2022","journal-title":"Nat. Biotechnol."},{"issue":"3","key":"10.1016\/j.gene.2023.147358_b0030","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/0960-8966(94)00048-E","article-title":"Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy","volume":"5","author":"Backman","year":"1995","journal-title":"Neuromuscul. Disord."},{"issue":"2","key":"10.1016\/j.gene.2023.147358_b0035","doi-asserted-by":"crossref","first-page":"227","DOI":"10.4103\/0976-3147.112777","article-title":"Deletion pattern in the dystrophin gene in Duchenne muscular dystrophy patients in northeast India","volume":"4","author":"Basumatary","year":"2013","journal-title":"J. Neurosci. Rural Pract."},{"issue":"4","key":"10.1016\/j.gene.2023.147358_b0040","doi-asserted-by":"crossref","first-page":"463","DOI":"10.3233\/JND-210776","article-title":"Comparing deflazacort and prednisone in Duchenne muscular dystrophy","volume":"9","author":"Biggar","year":"2022","journal-title":"J. Neuromuscul. Dis."},{"issue":"2","key":"10.1016\/j.gene.2023.147358_b0045","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1152\/physrev.00028.2001","article-title":"Function and genetics of dystrophin and dystrophin-related proteins in muscle","volume":"82","author":"Blake","year":"2002","journal-title":"Physiol. Rev."},{"issue":"23","key":"10.1016\/j.gene.2023.147358_b0050","doi-asserted-by":"crossref","first-page":"e2304","DOI":"10.1212\/WNL.0000000000012910","article-title":"Life expectancy in Duchenne muscular dystrophy: reproduced individual patient data meta-analysis","volume":"97","author":"Broomfield","year":"2021","journal-title":"Neurology"},{"issue":"5","key":"10.1016\/j.gene.2023.147358_b0055","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":"18","key":"10.1016\/j.gene.2023.147358_b0060","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.abg4910","article-title":"Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing","volume":"7","author":"Chemello","year":"2021","journal-title":"Sci. Adv."},{"issue":"12","key":"10.1016\/j.gene.2023.147358_b0065","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1038\/nrneurol.2016.180","article-title":"Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line","volume":"12","author":"Dowling","year":"2016","journal-title":"Nat. Rev. Neurol."},{"issue":"s2","key":"10.1016\/j.gene.2023.147358_b0070","doi-asserted-by":"crossref","first-page":"S303","DOI":"10.3233\/JND-210678","article-title":"Gene therapy for Duchenne muscular dystrophy","volume":"8","author":"Elangkovan","year":"2021","journal-title":"J. Neuromuscul. Dis."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0075","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1186\/s40246-018-0152-8","article-title":"Molecular characterization of exonic rearrangements and frame shifts in the dystrophin gene in Duchenne muscular dystrophy patients in a Saudi community","volume":"12","author":"Elhawary","year":"2018","journal-title":"Hum. Genomics"},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0080","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.bbrc.2012.01.029","article-title":"Restoration of muscle fibers and satellite cells after isogenic MSC transplantation with microdystrophin gene delivery","volume":"419","author":"Feng","year":"2012","journal-title":"Biochem. Biophys. Res. Commun."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0085","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1186\/s13059-018-1482-5","article-title":"CRISPR-SKIP: programmable gene splicing with single base editors","volume":"19","author":"Gapinske","year":"2018","journal-title":"Genome Biol."},{"issue":"8","key":"10.1016\/j.gene.2023.147358_b0090","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1038\/jhg.2015.43","article-title":"DMD mutation spectrum analysis in 613 Chinese patients with dystrophinopathy","volume":"60","author":"Guo","year":"2015","journal-title":"J. Hum. Genet."},{"issue":"18","key":"10.1016\/j.gene.2023.147358_b0095","doi-asserted-by":"crossref","DOI":"10.3390\/ijms20184638","article-title":"miRNA profiling for early detection and treatment of Duchenne muscular dystrophy","volume":"20","author":"Hrach","year":"2019","journal-title":"Int. J. Mol. Sci."},{"issue":"8","key":"10.1016\/j.gene.2023.147358_b0100","doi-asserted-by":"crossref","first-page":"611","DOI":"10.1016\/j.nmd.2013.05.003","article-title":"Scoliosis in Duchenne muscular dystrophy (DMD)","volume":"23","author":"Hsu","year":"2013","journal-title":"Neuromuscul. Disord."},{"issue":"3","key":"10.1016\/j.gene.2023.147358_b0105","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1038\/nbt.2508","article-title":"RNA-guided editing of bacterial genomes using CRISPR-Cas systems","volume":"31","author":"Jiang","year":"2013","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.gene.2023.147358_b0110","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1016\/j.csbj.2020.03.012","article-title":"Effective restoration of dystrophin expression in iPSC (Mdx)-derived muscle progenitor cells using the CRISPR\/Cas9 system and homology-directed repair technology","volume":"18","author":"Jin","year":"2020","journal-title":"Comput. Struct. Biotechnol. J."},{"key":"10.1016\/j.gene.2023.147358_b0115","doi-asserted-by":"crossref","first-page":"e00471","DOI":"10.7554\/eLife.00471","article-title":"RNA-programmed genome editing in human cells","volume":"2","author":"Jinek","year":"2013","journal-title":"Elife"},{"issue":"4","key":"10.1016\/j.gene.2023.147358_b0120","doi-asserted-by":"crossref","first-page":"335","DOI":"10.1038\/s41587-019-0103-5","article-title":"Duchenne muscular dystrophy awaits gene therapy","volume":"37","author":"Jones","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.gene.2023.147358_b0125","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1186\/1755-8794-4-36","article-title":"Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy","volume":"4","author":"Kemaladewi","year":"2011","journal-title":"BMC Med. Genomics"},{"issue":"10","key":"10.1016\/j.gene.2023.147358_b0130","doi-asserted-by":"crossref","first-page":"1985","DOI":"10.1016\/j.jid.2020.02.012","article-title":"Predictable CRISPR\/Cas9-mediated COL7A1 reframing for Dystrophic epidermolysis bullosa","volume":"140","author":"Kocher","year":"2020","journal-title":"J, Invest. Dermatol."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0135","doi-asserted-by":"crossref","first-page":"139","DOI":"10.1186\/s12881-019-0873-0","article-title":"Genetic analysis of 1051 Chinese families with Duchenne\/Becker Muscular Dystrophy","volume":"20","author":"Kong","year":"2019","journal-title":"BMC Med. Genet."},{"issue":"9","key":"10.1016\/j.gene.2023.147358_b0140","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1002\/jgm.1602","article-title":"Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog","volume":"13","author":"Koo","year":"2011","journal-title":"J. Gene Med."},{"issue":"11","key":"10.1016\/j.gene.2023.147358_b0145","doi-asserted-by":"crossref","first-page":"1355","DOI":"10.1038\/gt.2010.84","article-title":"Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene","volume":"17","author":"Koppanati","year":"2010","journal-title":"Gene Ther."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0150","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/j.stemcr.2014.10.013","article-title":"Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9","volume":"4","author":"Li","year":"2015","journal-title":"Stem Cell Rep."},{"issue":"14","key":"10.1016\/j.gene.2023.147358_b0155","doi-asserted-by":"crossref","first-page":"1099","DOI":"10.1038\/sj.gt.3302505","article-title":"Stable transduction of myogenic cells with lentiviral vectors expressing a minidystrophin","volume":"12","author":"Li","year":"2005","journal-title":"Gene Ther."},{"issue":"22","key":"10.1016\/j.gene.2023.147358_b0160","doi-asserted-by":"crossref","first-page":"1760","DOI":"10.1161\/CIRCULATIONAHA.121.054628","article-title":"Therapeutic exon skipping through a CRISPR-guided cytidine deaminase rescues dystrophic cardiomyopathy in vivo","volume":"144","author":"Li","year":"2021","journal-title":"Circulation"},{"issue":"6201","key":"10.1016\/j.gene.2023.147358_b0165","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"},{"key":"10.1016\/j.gene.2023.147358_b0170","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1007\/978-1-0716-2010-6_14","article-title":"Evaluation of exon skipping and dystrophin restoration in in vitro models of Duchenne muscular dystrophy","volume":"2434","author":"Lopez-Martinez","year":"2022","journal-title":"Methods Mol. Biol."},{"issue":"8","key":"10.1016\/j.gene.2023.147358_b0175","doi-asserted-by":"crossref","DOI":"10.3390\/ijms23084270","article-title":"Antisense oligonucleotides conjugated with lipophilic compounds: synthesis and in vitro evaluation of exon skipping in Duchenne muscular dystrophy","volume":"23","author":"Marchesi","year":"2022","journal-title":"Int. J. Mol. Sci."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0180","doi-asserted-by":"crossref","first-page":"e0228072","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 Lopez","year":"2020","journal-title":"PLoS One"},{"issue":"3","key":"10.1016\/j.gene.2023.147358_b0185","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1016\/0387-7604(96)00007-1","article-title":"Duchenne\/Becker muscular dystrophy: from molecular diagnosis to gene therapy","volume":"18","author":"Matsuo","year":"1996","journal-title":"Brain and Development"},{"issue":"11","key":"10.1016\/j.gene.2023.147358_b0190","doi-asserted-by":"crossref","first-page":"3140","DOI":"10.1016\/j.ymthe.2021.09.026","article-title":"Genome editing in large animal models","volume":"29","author":"Maynard","year":"2021","journal-title":"Mol. Ther."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0195","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/0888-7543(88)90113-9","article-title":"An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus","volume":"2","author":"Monaco","year":"1988","journal-title":"Genomics"},{"issue":"5","key":"10.1016\/j.gene.2023.147358_b0200","doi-asserted-by":"crossref","first-page":"1267","DOI":"10.1093\/hmg\/ddu537","article-title":"Becker muscular dystrophy severity is linked to the structure of dystrophin","volume":"24","author":"Nicolas","year":"2015","journal-title":"Hum. Mol. Genet."},{"issue":"9","key":"10.1016\/j.gene.2023.147358_b0205","doi-asserted-by":"crossref","first-page":"1718","DOI":"10.1038\/mt.2013.111","article-title":"Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients","volume":"21","author":"Ousterout","year":"2013","journal-title":"Mol. Ther."},{"issue":"3","key":"10.1016\/j.gene.2023.147358_b0210","doi-asserted-by":"crossref","first-page":"523","DOI":"10.1038\/mt.2014.234","article-title":"Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases","volume":"23","author":"Ousterout","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.gene.2023.147358_b0215","doi-asserted-by":"crossref","first-page":"6244","DOI":"10.1038\/ncomms7244","article-title":"Multiplex CRISPR\/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy","volume":"6","author":"Ousterout","year":"2015","journal-title":"Nat. Commun."},{"issue":"11","key":"10.1016\/j.gene.2023.147358_b0220","doi-asserted-by":"crossref","first-page":"3243","DOI":"10.1016\/j.ymthe.2021.09.003","article-title":"Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy","volume":"29","author":"Pickar-Oliver","year":"2021","journal-title":"Mol. Ther."},{"issue":"6413","key":"10.1016\/j.gene.2023.147358_b0225","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1038\/361647a0","article-title":"Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice","volume":"361","author":"Ragot","year":"1993","journal-title":"Nature"},{"issue":"11","key":"10.1016\/j.gene.2023.147358_b0230","doi-asserted-by":"crossref","first-page":"2281","DOI":"10.1038\/nprot.2013.143","article-title":"Genome engineering using the CRISPR-Cas9 system","volume":"8","author":"Ran","year":"2013","journal-title":"Nat. Protoc."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0235","doi-asserted-by":"crossref","first-page":"3390","DOI":"10.1038\/s41598-022-07158-8","article-title":"Transposase-CRISPR mediated targeted integration (TransCRISTI) in the human genome","volume":"12","author":"Rezazade Bazaz","year":"2022","journal-title":"Sci. Rep."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0240","doi-asserted-by":"crossref","first-page":"96","DOI":"10.1186\/s13018-022-02996-8","article-title":"Global prevalence of Duchenne and Becker muscular dystrophy: a systematic review and meta-analysis","volume":"17","author":"Salari","year":"2022","journal-title":"J. Orthop. Surg. Res."},{"issue":"3","key":"10.1016\/j.gene.2023.147358_b0245","first-page":"158","article-title":"Analysis of dystrophin gene deletions by multiplex PCR in Moroccan patients","volume":"2","author":"Sbiti","year":"2002","journal-title":"J. Biomed. Biotechnol."},{"issue":"8","key":"10.1016\/j.gene.2023.147358_b0250","doi-asserted-by":"crossref","first-page":"e464","DOI":"10.1097\/BPO.0000000000000717","article-title":"Surgical correction of scoliosis in patients with Duchenne muscular dystrophy: 30-year experience","volume":"37","author":"Scannell","year":"2017","journal-title":"J. Pediatr. Orthop."},{"issue":"6","key":"10.1016\/j.gene.2023.147358_b0255","doi-asserted-by":"crossref","first-page":"566","DOI":"10.1089\/hum.2011.017","article-title":"Long-term preservation of cardiac structure and function after adeno-associated virus serotype 9-mediated microdystrophin gene transfer in mdx mice","volume":"23","author":"Schinkel","year":"2012","journal-title":"Hum. Gene Ther."},{"issue":"18","key":"10.1016\/j.gene.2023.147358_b0260","doi-asserted-by":"crossref","first-page":"1337","DOI":"10.2217\/cer-2021-0018","article-title":"Meta-analyses of deflazacort versus prednisone\/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy","volume":"10","author":"Shieh","year":"2021","journal-title":"J. Comp. Eff. Res."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0265","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1007\/BF02913084","article-title":"Multiplex PCR for rapid detection of exonal deletions in patients of duchenne muscular dystrophy. Indian","volume":"21","author":"Singh","year":"2006","journal-title":"J. Clin. Biochem."},{"issue":"11","key":"10.1016\/j.gene.2023.147358_b0270","doi-asserted-by":"crossref","first-page":"1884","DOI":"10.1038\/mt.2016.188","article-title":"Eteplirsen approved for duchenne muscular dystrophy: the FDA faces a difficult choice","volume":"24","author":"Stein","year":"2016","journal-title":"Mol. Ther."},{"issue":"6448","key":"10.1016\/j.gene.2023.147358_b0275","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1126\/science.aax9181","article-title":"RNA-guided DNA insertion with CRISPR-associated transposases","volume":"365","author":"Strecker","year":"2019","journal-title":"Science"},{"issue":"4","key":"10.1016\/j.gene.2023.147358_b0280","doi-asserted-by":"crossref","first-page":"e01307","DOI":"10.1128\/JCM.01307-18","article-title":"CRISPR-Cas biology and its application to infectious diseases","volume":"57","author":"Strich","year":"2019","journal-title":"J. Clin. Microbiol."},{"issue":"7631","key":"10.1016\/j.gene.2023.147358_b0285","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1038\/nature20565","article-title":"In vivo genome editing via CRISPR\/Cas9 mediated homology-independent targeted integration","volume":"540","author":"Suzuki","year":"2016","journal-title":"Nature"},{"issue":"6271","key":"10.1016\/j.gene.2023.147358_b0290","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"},{"issue":"19","key":"10.1016\/j.gene.2023.147358_b0295","doi-asserted-by":"crossref","first-page":"4919","DOI":"10.1016\/j.cell.2021.08.028","article-title":"Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species","volume":"184","author":"Tabebordbar","year":"2021","journal-title":"Cell"},{"issue":"8","key":"10.1016\/j.gene.2023.147358_b0300","doi-asserted-by":"crossref","first-page":"1711","DOI":"10.1016\/j.jid.2019.02.015","article-title":"Efficient gene reframing therapy for recessive dystrophic epidermolysis bullosa with CRISPR\/Cas9","volume":"139","author":"Takashima","year":"2019","journal-title":"J, Invest. Dermatol."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0305","doi-asserted-by":"crossref","first-page":"162","DOI":"10.1002\/ana.24842","article-title":"Regarding \u201cEteplirsen for the treatment of Duchenne muscular dystrophy\u201d","volume":"81","author":"Unger","year":"2017","journal-title":"Ann. Neurol."},{"issue":"1","key":"10.1016\/j.gene.2023.147358_b0310","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1016\/j.yexcr.2014.01.026","article-title":"Antisense-mediated exon skipping: taking advantage of a trick from Mother Nature to treat rare genetic diseases","volume":"325","author":"Veltrop","year":"2014","journal-title":"Exp. Cell Res."},{"key":"10.1016\/j.gene.2023.147358_b0315","doi-asserted-by":"crossref","unstructured":"Verma, A.K., Chettri, D., Verma, A.K., 2022. Potential of CRISPR\/Cas9-Based Genome Editing in the Fields of Industrial Biotechnology: Strategies, Challenges, and Applications. Industrial Microbiology and Biotechnology. Springer, pp. 667\u2013690.","DOI":"10.1007\/978-981-16-5214-1_23"},{"issue":"5","key":"10.1016\/j.gene.2023.147358_b0320","doi-asserted-by":"crossref","first-page":"722","DOI":"10.1097\/WCO.0000000000000739","article-title":"Update in Duchenne and Becker muscular dystrophy","volume":"32","author":"Waldrop","year":"2019","journal-title":"Curr. Opin. Neurol."},{"key":"10.1016\/j.gene.2023.147358_b0325","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1016\/j.omtn.2018.02.002","article-title":"Repurposing dantrolene for long-term combination therapy to potentiate antisense-mediated DMD exon skipping in the mdx Mouse","volume":"11","author":"Wang","year":"2018","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.gene.2023.147358_b0330","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1016\/j.omtn.2021.03.005","article-title":"Efficient correction of Duchenne muscular dystrophy mutations by SpCas9 and dual gRNAs","volume":"24","author":"Xiang","year":"2021","journal-title":"Mol. Ther. Nucleic Acids"},{"issue":"9","key":"10.1016\/j.gene.2023.147358_b0335","doi-asserted-by":"crossref","first-page":"896","DOI":"10.1089\/hum.2004.15.896","article-title":"Trans-splicing adeno-associated viral vector-mediated gene therapy is limited by the accumulation of spliced mRNA but not by dual vector coinfection efficiency","volume":"15","author":"Xu","year":"2004","journal-title":"Hum. Gene Ther."},{"issue":"5","key":"10.1016\/j.gene.2023.147358_b0340","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1038\/s41571-019-0166-8","article-title":"CRISPR\u2013Cas: a tool for cancer research and therapeutics","volume":"16","author":"Yin","year":"2019","journal-title":"Nat. Rev. Clin. Oncol."},{"issue":"6","key":"10.1016\/j.gene.2023.147358_b0345","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1517\/14712598.7.6.831","article-title":"Potential of oligonucleotide-mediated exon-skipping therapy for Duchenne muscular dystrophy","volume":"7","author":"Yokota","year":"2007","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.gene.2023.147358_b0350","doi-asserted-by":"crossref","unstructured":"Zhang, Y., Li, H., Min, Y.L., Sanchez-Ortiz, E., Huang, J., Mireault, A.A., 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.","DOI":"10.1126\/sciadv.aay6812"}],"container-title":["Gene"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0378111923001993?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0378111923001993?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,7]],"date-time":"2025-10-07T17:03:01Z","timestamp":1759856581000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0378111923001993"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,5]]},"references-count":70,"alternative-id":["S0378111923001993"],"URL":"https:\/\/doi.org\/10.1016\/j.gene.2023.147358","relation":{},"ISSN":["0378-1119"],"issn-type":[{"value":"0378-1119","type":"print"}],"subject":[],"published":{"date-parts":[[2023,5]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"New advancements in CRISPR based gene therapy of Duchenne muscular dystrophy","name":"articletitle","label":"Article Title"},{"value":"Gene","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.gene.2023.147358","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 Published by Elsevier B.V.","name":"copyright","label":"Copyright"}],"article-number":"147358"}}