{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,2]],"date-time":"2026-11-02T08:38:14Z","timestamp":1793608694034,"version":"4.5.0"},"reference-count":33,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2023,9,1]],"date-time":"2023-09-01T00:00:00Z","timestamp":1693526400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,9,1]],"date-time":"2023-09-01T00:00:00Z","timestamp":1693526400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,6,26]],"date-time":"2023-06-26T00:00:00Z","timestamp":1687737600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Nucleic Acids"],"published-print":{"date-parts":[[2023,9]]},"DOI":"10.1016\/j.omtn.2023.06.022","type":"journal-article","created":{"date-parts":[[2023,6,28]],"date-time":"2023-06-28T21:19:47Z","timestamp":1687987187000},"page":"273-285","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":27,"special_numbering":"C","title":["The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders"],"prefix":"10.1016","volume":"33","author":[{"given":"Xiang","family":"Li","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mahboubeh","family":"Kheirabadi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick G.","family":"Dougherty","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kimberli J.","family":"Kamer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiulong","family":"Shen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nelsa L.","family":"Estrella","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suresh","family":"Peddigari","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anushree","family":"Pathak","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara L.","family":"Blake","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emmanuelle","family":"Sizensky","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmen del","family":"Genio","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arti B.","family":"Gaur","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohanraj","family":"Dhanabal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mahasweta","family":"Girgenrath","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natarajan","family":"Sethuraman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9452-1007","authenticated-orcid":false,"given":"Ziqing","family":"Qian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2023.06.022_bib2","doi-asserted-by":"crossref","first-page":"888","DOI":"10.1124\/dmd.121.000417","article-title":"Absorption, distribution, metabolism, and excretion of US Food and Drug Administration\u2013Approved antisense oligonucleotide drugs","volume":"50","author":"Migliorati","year":"2022","journal-title":"Drug Metab. Dispos."},{"key":"10.1016\/j.omtn.2023.06.022_bib3","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1089\/nat.2017.0682","article-title":"Development of exon skipping therapies for Duchenne muscular dystrophy: a critical review and a perspective on the outstanding issues","volume":"27","author":"Aartsma-Rus","year":"2017","journal-title":"Nucleic Acid Therapeut."},{"key":"10.1016\/j.omtn.2023.06.022_bib4","series-title":"Morpholino Oligomers","author":"Moulton","year":"2017"},{"key":"10.1016\/j.omtn.2023.06.022_bib5","doi-asserted-by":"crossref","first-page":"1405","DOI":"10.1038\/mt.2016.111","article-title":"Morpholino-mediated knockdown of DUX4 toward facioscapulohumeral muscular dystrophy therapeutics","volume":"24","author":"Chen","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2023.06.022_bib6","doi-asserted-by":"crossref","first-page":"1398","DOI":"10.1093\/hmg\/ddab136","article-title":"Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model","volume":"30","author":"Lu-Nguyen","year":"2021","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2023.06.022_bib7","doi-asserted-by":"crossref","first-page":"1210","DOI":"10.1038\/mt.2010.72","article-title":"Preclinical PK and PD studies on 2\u2032-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.2023.06.022_bib8","doi-asserted-by":"crossref","first-page":"361","DOI":"10.1080\/14712598.2021.1823368","article-title":"Status update in the use of cell penetrating peptides for the delivery of macromolecular therapeutics","volume":"21","author":"Kurrikoff","year":"2021","journal-title":"Expet Opin. Biol. Ther."},{"key":"10.1016\/j.omtn.2023.06.022_bib9","doi-asserted-by":"crossref","first-page":"273","DOI":"10.1021\/acs.bioconjchem.8b00778","article-title":"Overcoming endosomal entrapment in drug delivery","volume":"30","author":"Pei","year":"2019","journal-title":"Bioconjugate Chem."},{"key":"10.1016\/j.omtn.2023.06.022_bib10","doi-asserted-by":"crossref","first-page":"309","DOI":"10.1021\/acs.accounts.1c00560","article-title":"How do biomolecules cross the cell membrane","volume":"55","author":"Pei","year":"2022","journal-title":"Acc. Chem. Res."},{"key":"10.1016\/j.omtn.2023.06.022_bib11","doi-asserted-by":"crossref","first-page":"423","DOI":"10.1021\/cb3005275","article-title":"Efficient delivery of cyclic peptides into mammalian cells with short sequence motifs","volume":"8","author":"Qian","year":"2013","journal-title":"ACS Chem. Biol."},{"key":"10.1016\/j.omtn.2023.06.022_bib12","doi-asserted-by":"crossref","first-page":"4034","DOI":"10.1021\/bi5004102","article-title":"Early endosomal escape of a cyclic cell-penetrating peptide allows effective cytosolic cargo delivery","volume":"53","author":"Qian","year":"2014","journal-title":"Biochemistry"},{"key":"10.1016\/j.omtn.2023.06.022_bib13","doi-asserted-by":"crossref","first-page":"2601","DOI":"10.1021\/acs.biochem.6b00226","article-title":"Discovery and mechanism of highly efficient cyclic cell-penetrating peptides","volume":"55","author":"Qian","year":"2016","journal-title":"Biochemistry"},{"key":"10.1016\/j.omtn.2023.06.022_bib14","doi-asserted-by":"crossref","first-page":"2568","DOI":"10.1021\/acschembio.0c00593","article-title":"Engineering cell-permeable proteins through insertion of cell-penetrating motifs into surface loops","volume":"15","author":"Chen","year":"2020","journal-title":"ACS Chem. Biol."},{"key":"10.1016\/j.omtn.2023.06.022_bib15","doi-asserted-by":"crossref","first-page":"12853","DOI":"10.1021\/acs.jmedchem.0c01236","article-title":"A Peptidyl inhibitor that blocks calcineurin\u2013NFAT interaction and prevents acute lung injury","volume":"63","author":"Dougherty","year":"2020","journal-title":"J.\u00a0Med. Chem."},{"key":"10.1016\/j.omtn.2023.06.022_bib16","doi-asserted-by":"crossref","first-page":"1228","DOI":"10.1038\/nbt759","article-title":"Systemically delivered antisense oligomers upregulate gene expression in mouse tissues","volume":"20","author":"Sazani","year":"2002","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2023.06.022_bib17","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1073\/pnas.96.1.91","article-title":"Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus","volume":"96","author":"Zanta","year":"1999","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2023.06.022_bib18","doi-asserted-by":"crossref","first-page":"1223","DOI":"10.1002\/j.2040-4603.2015.tb00638.x","article-title":"The dystrophin complex: Structure, function and implications for therapy","volume":"5","author":"Gao","year":"2015","journal-title":"Compr. Physiol."},{"key":"10.1016\/j.omtn.2023.06.022_bib19","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1038\/ng.3858","article-title":"DUX-family transcription factors regulate zygotic genome activation in placental mammals","volume":"49","author":"De Iaco","year":"2017","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omtn.2023.06.022_bib20","doi-asserted-by":"crossref","first-page":"168","DOI":"10.5582\/irdr.2016.01056","article-title":"Targeting mRNA for the treatmenet of facioscapulohumeral muscular dystrophy","volume":"5","author":"Bao","year":"2016","journal-title":"Intractable Rare Dis. Res."},{"key":"10.1016\/j.omtn.2023.06.022_bib21","doi-asserted-by":"crossref","first-page":"865","DOI":"10.3390\/jpm12060865","article-title":"FSHD therapeutic strategies: what will it take to get to clinic?","volume":"12","author":"Himeda","year":"2022","journal-title":"J.\u00a0Personalized Med."},{"key":"10.1016\/j.omtn.2023.06.022_bib22","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1038\/s41582-022-00762-2","article-title":"Facioscapulohumeral muscular dystrophy: the road to targeted therapies","volume":"19","author":"Tihaya","year":"2023","journal-title":"Nat. Rev. Neurol."},{"key":"10.1016\/j.omtn.2023.06.022_bib23","doi-asserted-by":"crossref","first-page":"e13243","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.2023.06.022_bib24","doi-asserted-by":"crossref","first-page":"673","DOI":"10.1038\/s41573-020-0075-7","article-title":"Advances in oligonucleotide drug delivery","volume":"19","author":"Roberts","year":"2020","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omtn.2023.06.022_bib25","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.jconrel.2016.06.036","article-title":"Development of antibody-siRNA conjugate targeted to cardiac and skeletal muscles","volume":"237","author":"Sugo","year":"2016","journal-title":"J.\u00a0Contr. Release"},{"key":"10.1016\/j.omtn.2023.06.022_bib26","unstructured":"(2016). EXONDYS 51 (eteplirsen) [prescribing information]. (Sarepta Therapeutics, Inc). www.exondys51.com\/modules\/exondys\/files\/EXONDYS51PI.pdf"},{"key":"10.1016\/j.omtn.2023.06.022_bib27","unstructured":"(2019). VYONDYS 53 (golodirsen) [prescribing information]. (Sarepta Therapeutics, Inc). https:\/\/www.vyondys53.com\/assets\/Vyondys53_Prescribing_Information.pdf"},{"key":"10.1016\/j.omtn.2023.06.022_bib28","unstructured":"(2021). AMONDYS 45 (casimersen) [prescribing information]. (Sarepta Therapeutics, Inc). https:\/\/www.amondys45.com\/Amondys45_(casimersen)_Prescribing_Information.pdf"},{"key":"10.1016\/j.omtn.2023.06.022_bib29","unstructured":"(2020). VILTEPSO (Viltolarsen) [prescribing Information]. (NS Pharma, Inc). https:\/\/www.viltepso.com\/prescribing-information"},{"key":"10.1016\/j.omtn.2023.06.022_bib30","doi-asserted-by":"crossref","first-page":"389","DOI":"10.1007\/s40259-021-00486-7","article-title":"Restoring protein expression in neuromuscular conditions: A review assessing the current state of exon skipping\/inclusion and gene therapies for Duchenne muscular dystrophy and spinal muscular atrophy","volume":"35","author":"Sheikh","year":"2021","journal-title":"BioDrugs"},{"key":"10.1016\/j.omtn.2023.06.022_bib31","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.2023.06.022_bib32","doi-asserted-by":"crossref","first-page":"729","DOI":"10.3390\/ijms21030729","article-title":"DUX4 signalling in the pathogenesis of facioscapulohumeral muscular dystrophy","volume":"21","author":"Lim","year":"2020","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.omtn.2023.06.022_bib33","doi-asserted-by":"crossref","first-page":"3965","DOI":"10.1093\/nar\/29.19.3965","article-title":"Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs","volume":"29","author":"Sazani","year":"2001","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2023.06.022_bib34","doi-asserted-by":"crossref","first-page":"1578","DOI":"10.1126\/science.2662404","article-title":"The molecular basis of muscular dystrophy in the mdx mouse: a point mutation","volume":"244","author":"Sicinski","year":"1989","journal-title":"Science"}],"container-title":["Molecular Therapy - Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253123001725?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253123001725?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,31]],"date-time":"2025-10-31T01:35:53Z","timestamp":1761874553000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253123001725"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,9]]},"references-count":33,"alternative-id":["S2162253123001725"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2023.06.022","relation":{},"ISSN":["2162-2531"],"issn-type":[{"value":"2162-2531","type":"print"}],"subject":[],"published":{"date-parts":[[2023,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2023.06.022","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 The Author(s).","name":"copyright","label":"Copyright"}]}}