{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,18]],"date-time":"2026-10-18T02:54:53Z","timestamp":1792292093914,"version":"4.4.0"},"reference-count":50,"publisher":"Elsevier BV","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":[[2022,8,15]],"date-time":"2022-08-15T00:00:00Z","timestamp":1660521600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100014943","name":"Sarepta Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014943","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100016471","name":"Agios Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100016471","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Nucleic Acids"],"published-print":{"date-parts":[[2022,12]]},"DOI":"10.1016\/j.omtn.2022.08.019","type":"journal-article","created":{"date-parts":[[2022,8,17]],"date-time":"2022-08-17T19:07:16Z","timestamp":1660763236000},"page":"17-27","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":55,"special_numbering":"C","title":["A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice"],"prefix":"10.1016","volume":"30","author":[{"given":"Li","family":"Gan","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leslie C.L.","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6715-0626","authenticated-orcid":false,"given":"Jenna A.","family":"Wood","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monica","family":"Yao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris M.","family":"Treleaven","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nelsa L.","family":"Estrella","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bruce M.","family":"Wentworth","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gunnar J.","family":"Hanson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marco A.","family":"Passini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2022.08.019_bib1","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1152\/physrev.00007.2015","article-title":"Absence of dystrophin disrupts skeletal muscle signaling: roles of Ca2+, 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.omtn.2022.08.019_bib2","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1016\/S1474-4422(09)70271-6","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management","volume":"9","author":"Bushby","year":"2010","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.omtn.2022.08.019_bib3","doi-asserted-by":"crossref","first-page":"104","DOI":"10.1016\/j.addr.2015.05.008","article-title":"Exon skipping therapy for Duchenne muscular dystrophy","volume":"87","author":"Kole","year":"2015","journal-title":"Adv. Drug Deliv. Rev."},{"key":"10.1016\/j.omtn.2022.08.019_bib4","doi-asserted-by":"crossref","first-page":"1795","DOI":"10.2147\/NDT.S93873","article-title":"Current and emerging treatment strategies for Duchenne muscular dystrophy","volume":"12","author":"Mah","year":"2016","journal-title":"Neuropsychiatr. Dis. Treat."},{"key":"10.1016\/j.omtn.2022.08.019_bib5","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.omtn.2022.08.019_bib6","first-page":"935","article-title":"Skeletal muscle dystrophin-glycoprotein complex and muscular dystrophy","volume":"2","author":"Kobayashi","year":"2012"},{"key":"10.1016\/j.omtn.2022.08.019_bib7","doi-asserted-by":"crossref","first-page":"373","DOI":"10.18388\/abp.2004_3576","article-title":"RNA modulation, repair and remodeling by splice switching oligonucleotides","volume":"51","author":"Kole","year":"2004","journal-title":"Acta Biochim. Pol."},{"key":"10.1016\/j.omtn.2022.08.019_bib8","doi-asserted-by":"crossref","first-page":"554","DOI":"10.1038\/mt.2008.287","article-title":"Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene","volume":"17","author":"Popplewell","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib9","doi-asserted-by":"crossref","first-page":"1288","DOI":"10.1038\/sj.mt.6300095","article-title":"Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript","volume":"15","author":"Wilton","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib10","doi-asserted-by":"crossref","first-page":"259","DOI":"10.2174\/156652311796150381","article-title":"RNA splicing manipulation: strategies to modify gene expression for a variety of therapeutic outcomes","volume":"11","author":"Wilton","year":"2011","journal-title":"Curr. Gene Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib11","doi-asserted-by":"crossref","first-page":"330","DOI":"10.1016\/S0960-8966(99)00010-3","article-title":"Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides","volume":"9","author":"Wilton","year":"1999","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.omtn.2022.08.019_bib12","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"},{"key":"10.1016\/j.omtn.2022.08.019_bib13","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1146\/annurev.pharmtox.41.1.403","article-title":"Antisense oligonucleotides: promise and reality","volume":"41","author":"Lebedeva","year":"2001","journal-title":"Annu. Rev. Pharmacol. Toxicol."},{"key":"10.1016\/j.omtn.2022.08.019_bib14","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1007\/978-1-4939-7374-3_9","article-title":"Exon skipping therapy using phosphorodiamidate morpholino oligomers in the mdx52 mouse model of Duchenne muscular dystrophy","volume":"1687","author":"Miyatake","year":"2018","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.omtn.2022.08.019_bib15","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1038\/gt.2009.120","article-title":"Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino","volume":"17","author":"Wu","year":"2010","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib16","doi-asserted-by":"crossref","first-page":"667","DOI":"10.1002\/ana.21627","article-title":"Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs","volume":"65","author":"Yokota","year":"2009","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtn.2022.08.019_bib17","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1038\/sj.mt.6300329","article-title":"Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice","volume":"16","author":"Yin","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib18","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1073\/pnas.0406700102","article-title":"Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles","volume":"102","author":"Lu","year":"2005","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2022.08.019_bib19","unstructured":"Exondys 51 (eteplirsen) injection, for intravenous use [Prescribing Information]. 2020. Cambridge, MA: Sarepta Therapeutics, Inc."},{"key":"10.1016\/j.omtn.2022.08.019_bib20","unstructured":"Vyondys 53 (golodirsen) injection, for intravenous use [Prescribing Information]. 2020. Cambridge, MA: Sarepta Therapeutics, Inc."},{"key":"10.1016\/j.omtn.2022.08.019_bib21","unstructured":"Viltepso (viltolarsen) injection, for intravenous use [Prescribing Information]. 2020. Paramus, NJ: NS Pharma, Inc."},{"key":"10.1016\/j.omtn.2022.08.019_bib22","unstructured":"Amondys 45 (casimersen) injection, for intravenous use [Prescribing Information]. 2021. Cambridge, MA: Sarepta Therapeutics, Inc."},{"key":"10.1016\/j.omtn.2022.08.019_bib23","doi-asserted-by":"crossref","first-page":"47","DOI":"10.3233\/JND-170272","article-title":"Long-term pulmonary function in Duchenne muscular dystrophy: comparison of eteplirsen-treated patients to natural history","volume":"5","author":"Kinane","year":"2018","journal-title":"J.\u00a0Neuromuscul. Dis."},{"key":"10.1016\/j.omtn.2022.08.019_bib24","doi-asserted-by":"crossref","first-page":"989","DOI":"10.3233\/JND-210643","article-title":"Open-label evaluation of eteplirsen in patients with Duchenne muscular dystrophy amenable to exon 51 skipping: PROMOVI trial","volume":"8","author":"McDonald","year":"2021","journal-title":"J.\u00a0Neuromuscul. Dis."},{"key":"10.1016\/j.omtn.2022.08.019_bib25","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1002\/ana.24555","article-title":"Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy","volume":"79","author":"Mendell","year":"2016","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtn.2022.08.019_bib26","doi-asserted-by":"crossref","first-page":"469","DOI":"10.3233\/JND-200548","article-title":"Comparison of long-term ambulatory function in patients with Duchenne muscular dystrophy treated with eteplirsen and matched natural history controls","volume":"8","author":"Mendell","year":"2021","journal-title":"J.\u00a0Neuromuscul. Dis."},{"key":"10.1016\/j.omtn.2022.08.019_bib27","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1089\/nat.2021.0043","article-title":"Long-term safety and efficacy data of golodirsen in ambulatory patients with Duchenne muscular dystrophy amenable to exon 53 skipping: a first-in-human, multicenter, two-part, open-label Phase 1\/2 trial","volume":"32","author":"Servais","year":"2021","journal-title":"Nucleic Acid Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib28","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.202013243","article-title":"Delivery of oligonucleotide-based therapeutics: challenges and opportunities","volume":"13","author":"Hammond","year":"2021","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtn.2022.08.019_bib29","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1038\/mt.2010.261","article-title":"Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice","volume":"19","author":"Malerba","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib30","doi-asserted-by":"crossref","first-page":"576","DOI":"10.1038\/mt.2010.288","article-title":"One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice","volume":"19","author":"Wu","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib31","doi-asserted-by":"crossref","first-page":"343","DOI":"10.1080\/14712598.2021.1832462","article-title":"Developments in reading frame restoring therapy approaches for Duchenne muscular dystrophy","volume":"21","author":"Schneider","year":"2021","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib32","doi-asserted-by":"crossref","first-page":"R163","DOI":"10.1093\/hmg\/ddy171","article-title":"Exon-skipping advances for Duchenne muscular dystrophy","volume":"27","author":"Echevarr\u00eda","year":"2018","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2022.08.019_bib33","doi-asserted-by":"crossref","first-page":"304","DOI":"10.1016\/j.jconrel.2006.09.011","article-title":"Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents","volume":"116","author":"Abes","year":"2006","journal-title":"J.\u00a0Control. Release"},{"key":"10.1016\/j.omtn.2022.08.019_bib34","doi-asserted-by":"crossref","first-page":"1325","DOI":"10.1021\/bc070060v","article-title":"Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate","volume":"18","author":"Amantana","year":"2007","journal-title":"Bioconjug. Chem."},{"key":"10.1016\/j.omtn.2022.08.019_bib35","doi-asserted-by":"crossref","first-page":"e38","DOI":"10.1038\/mtna.2012.30","article-title":"Pip6-PMO, a new generation of peptide-oligonucleotide conjugates with improved cardiac exon skipping activity for DMD treatment","volume":"1","author":"Betts","year":"2012","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2022.08.019_bib36","doi-asserted-by":"crossref","first-page":"1587","DOI":"10.1038\/sj.mt.6300245","article-title":"Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse","volume":"15","author":"Fletcher","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib37","doi-asserted-by":"crossref","first-page":"1624","DOI":"10.1038\/mt.2008.120","article-title":"Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice","volume":"16","author":"Jearawiriyapaisarn","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib38","doi-asserted-by":"crossref","first-page":"3909","DOI":"10.1093\/hmg\/ddn293","article-title":"Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function","volume":"17","author":"Yin","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2022.08.019_bib39","series-title":"Peptide Oligonucleotide Conjugates","author":"Hanson","year":"2015"},{"key":"10.1016\/j.omtn.2022.08.019_bib40","author":"Schnell"},{"key":"10.1016\/j.omtn.2022.08.019_bib41","doi-asserted-by":"crossref","first-page":"e148","DOI":"10.1038\/mtna.2014.1","article-title":"The dynamics of compound, transcript, and protein effects after treatment with 2OMePS antisense oligonucleotides in mdx mice","volume":"3","author":"Verhaart","year":"2014","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2022.08.019_bib42","doi-asserted-by":"crossref","first-page":"392","DOI":"10.1016\/j.ajpath.2012.04.006","article-title":"Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino","volume":"181","author":"Wu","year":"2012","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.omtn.2022.08.019_bib43","series-title":"Presented at: Oligonucleotide Therapeutics Society (OTS) annual Meeting","author":"Shahid","year":"2020"},{"key":"10.1016\/j.omtn.2022.08.019_bib44","series-title":"Presented at: Oligonucleotide Therapeutics Society (OTS) Annual Meeting","author":"Wu","year":"2020"},{"key":"10.1016\/j.omtn.2022.08.019_bib45","series-title":"Presented at: Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference","author":"Hadcock","year":"2021"},{"key":"10.1016\/j.omtn.2022.08.019_bib46","doi-asserted-by":"crossref","first-page":"1210","DOI":"10.1038\/mt.2010.72","article-title":"Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model","volume":"18","author":"Heemskerk","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2022.08.019_bib47","doi-asserted-by":"crossref","first-page":"2484","DOI":"10.1096\/fj.12-224170","article-title":"Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin\/utrophin double-knockout mice","volume":"27","author":"van Putten","year":"2013","journal-title":"FASEB J"},{"key":"10.1016\/j.omtn.2022.08.019_bib48","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1016\/j.yjmcc.2014.01.009","article-title":"Low dystrophin levels in heart can delay heart failure in mdx mice","volume":"69","author":"van Putten","year":"2014","journal-title":"J.\u00a0Mol. Cell. Cardiol."},{"key":"10.1016\/j.omtn.2022.08.019_bib49","doi-asserted-by":"crossref","first-page":"280","DOI":"10.1002\/ana.25951","article-title":"Very low residual dystrophin quantity is associated with milder dystrophinopathy","volume":"89","author":"de Feraudy","year":"2021","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtn.2022.08.019_bib50","author":"DeLuca"}],"container-title":["Molecular Therapy - Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253122002189?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253122002189?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,31]],"date-time":"2025-10-31T01:36:38Z","timestamp":1761874598000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253122002189"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,12]]},"references-count":50,"alternative-id":["S2162253122002189"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2022.08.019","relation":{},"ISSN":["2162-2531"],"issn-type":[{"value":"2162-2531","type":"print"}],"subject":[],"published":{"date-parts":[[2022,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2022.08.019","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 The Authors.","name":"copyright","label":"Copyright"}]}}