{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,6]],"date-time":"2026-06-06T12:59:27Z","timestamp":1780750767548,"version":"3.54.1"},"publisher-location":"New York, NY","reference-count":18,"publisher":"Springer US","isbn-type":[{"value":"9781071647295","type":"print"},{"value":"9781071647301","type":"electronic"}],"license":[{"start":{"date-parts":[[2025,1,1]],"date-time":"2025-01-01T00:00:00Z","timestamp":1735689600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2025,1,1]],"date-time":"2025-01-01T00:00:00Z","timestamp":1735689600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025]]},"DOI":"10.1007\/978-1-0716-4730-1_23","type":"book-chapter","created":{"date-parts":[[2025,7,28]],"date-time":"2025-07-28T12:59:28Z","timestamp":1753707568000},"page":"345-359","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Use of Tricyclo-DNA Antisense Oligonucleotides for Exon-Skipping"],"prefix":"10.1007","author":[{"given":"Aurelie","family":"Goyenvalle","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,7,29]]},"reference":[{"key":"23_CR1","doi-asserted-by":"publisher","first-page":"270","DOI":"10.1038\/nm.3765","volume":"21","author":"A Goyenvalle","year":"2015","unstructured":"Goyenvalle A, Griffith G, Babbs A et al (2015) Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers. Nat Med 21:270\u2013275","journal-title":"Nat Med"},{"key":"23_CR2","doi-asserted-by":"publisher","first-page":"157","DOI":"10.3233\/JND-160146","volume":"3","author":"A Goyenvalle","year":"2016","unstructured":"Goyenvalle A, Leumann C, Garcia L (2016) Therapeutic potential of Tricyclo-DNA antisense oligonucleotides. J Neuromuscul Dis 3:157\u2013167","journal-title":"J Neuromuscul Dis"},{"key":"23_CR3","doi-asserted-by":"publisher","first-page":"144","DOI":"10.1016\/j.omtn.2017.06.013","volume":"8","author":"K Relizani","year":"2017","unstructured":"Relizani K, Griffith G, Echevarr\u00eda L et al (2017) Efficacy and safety profile of Tricyclo-DNA antisense oligonucleotides in Duchenne muscular dystrophy mouse model. Mol Ther Nucleic Acids 8:144\u2013157","journal-title":"Mol Ther Nucleic Acids"},{"key":"23_CR4","doi-asserted-by":"publisher","first-page":"81","DOI":"10.1016\/j.omtn.2017.02.009","volume":"7","author":"V Robin","year":"2017","unstructured":"Robin V, Griffith G, Carter J-PL et al (2017) Efficient SMN rescue following subcutaneous Tricyclo-DNA antisense oligonucleotide treatment. Mol Ther Nucleic Acids 7:81\u201389","journal-title":"Mol Ther Nucleic Acids"},{"key":"23_CR5","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1093\/nar\/gkab1199","volume":"50","author":"K Relizani","year":"2022","unstructured":"Relizani K, Echevarr\u00eda L, Zarrouki F et al (2022) Palmitic acid conjugation enhances potency of tricyclo-DNA splice switching oligonucleotides. Nucleic Acids Res 50:17\u201334","journal-title":"Nucleic Acids Res"},{"key":"23_CR6","doi-asserted-by":"publisher","first-page":"917","DOI":"10.1038\/s41573-023-00775-6","volume":"22","author":"TC Roberts","year":"2023","unstructured":"Roberts TC, Wood MJA, Davies KE (2023) Therapeutic approaches for Duchenne muscular dystrophy. Nat Rev Drug Discov 22:917\u2013934","journal-title":"Nat Rev Drug Discov"},{"key":"23_CR7","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2014.6","volume":"3","author":"Q-L Lu","year":"2014","unstructured":"Lu Q-L, Cirak S, Partridge T (2014) What can we learn from clinical trials of exon skipping for DMD? Mol Ther Nucleic Acids 3:e152","journal-title":"Mol Ther Nucleic Acids"},{"key":"23_CR8","doi-asserted-by":"publisher","first-page":"882","DOI":"10.1016\/S1474-4422(16)30035-7","volume":"15","author":"V Straub","year":"2016","unstructured":"Straub V, Balabanov P, Bushby K et al (2016) Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy. Lancet Neurol 15:882\u2013890","journal-title":"Lancet Neurol"},{"key":"23_CR9","doi-asserted-by":"publisher","first-page":"1884","DOI":"10.1038\/mt.2016.188","volume":"24","author":"CA Stein","year":"2016","unstructured":"Stein CA (2016) Eteplirsen approved for Duchenne muscular dystrophy: the FDA faces a difficult choice. Mol Ther J Am Soc Gene Ther 24:1884\u20131885","journal-title":"Mol Ther J Am Soc Gene Ther"},{"key":"23_CR10","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1089\/nat.2016.0657","volume":"27","author":"A Aartsma-Rus","year":"2017","unstructured":"Aartsma-Rus A, Krieg AM (2017) FDA approves Eteplirsen for Duchenne muscular dystrophy: the next chapter in the Eteplirsen saga. Nucleic Acid Ther 27:1\u20133","journal-title":"Nucleic Acid Ther"},{"key":"23_CR11","doi-asserted-by":"crossref","unstructured":"Godfrey C, Desviat LR, Smedsr\u00f8d B et al (2017) Delivery is key: lessons learnt from developing splice-switching antisense therapies. EMBO Mol Med","DOI":"10.15252\/emmm.201607199"},{"key":"23_CR12","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1089\/nat.2022.0061","volume":"33","author":"A Goyenvalle","year":"2023","unstructured":"Goyenvalle A, Jimenez-Mallebrera C, van Roon W et al (2023) Considerations in the preclinical assessment of the safety of antisense oligonucleotides. Nucleic Acid Ther 33:1\u201316","journal-title":"Nucleic Acid Ther"},{"key":"23_CR13","doi-asserted-by":"publisher","first-page":"34","DOI":"10.1186\/2044-5040-1-34","volume":"1","author":"K Mamchaoui","year":"2011","unstructured":"Mamchaoui K, Trollet C, Bigot A et al (2011) Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders. Skelet Muscle 1:34","journal-title":"Skelet Muscle"},{"key":"23_CR14","doi-asserted-by":"crossref","unstructured":"Bremmer-Bout M, Aartsma-Rus A, Meijer EJ de, et al (2004) Targeted exon skipping in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotides. Mol Ther J Am Soc Gene Ther 10:232\u2013240","DOI":"10.1016\/j.ymthe.2004.05.031"},{"key":"23_CR15","doi-asserted-by":"publisher","first-page":"492","DOI":"10.1006\/bbrc.1997.7328","volume":"238","author":"E Araki","year":"1997","unstructured":"Araki E, Nakamura K, Nakao K et al (1997) Targeted disruption of exon 52 in the mouse dystrophin gene induced muscle degeneration similar to that observed in Duchenne muscular dystrophy. Biochem Biophys Res Commun 238:492\u2013497","journal-title":"Biochem Biophys Res Commun"},{"key":"23_CR16","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkp841","volume":"38","author":"CA Stein","year":"2010","unstructured":"Stein CA, Hansen JB, Lai J et al (2010) Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents. Nucleic Acids Res 38:e3","journal-title":"Nucleic Acids Res"},{"key":"23_CR17","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0244215","volume":"15","author":"A Yavas","year":"2020","unstructured":"Yavas A, Weij R, van Putten M et al (2020) Detailed genetic and functional analysis of the hDMDdel52\/mdx mouse model. PLoS One 15:e0244215","journal-title":"PLoS One"},{"key":"23_CR18","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/29.9.e45","volume":"29","author":"MW Pfaffl","year":"2001","unstructured":"Pfaffl MW (2001) A new mathematical model for relative quantification in real-time RT-PCR. Nucleic Acids Res 29:e45","journal-title":"Nucleic Acids Res"}],"container-title":["Methods in Molecular Biology","Exon Skipping and Inclusion Therapies"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/978-1-0716-4730-1_23","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,6,6]],"date-time":"2026-06-06T12:08:07Z","timestamp":1780747687000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/978-1-0716-4730-1_23"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025]]},"ISBN":["9781071647295","9781071647301"],"references-count":18,"URL":"https:\/\/doi.org\/10.1007\/978-1-0716-4730-1_23","relation":{},"ISSN":["1064-3745","1940-6029"],"issn-type":[{"value":"1064-3745","type":"print"},{"value":"1940-6029","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025]]},"assertion":[{"value":"29 July 2025","order":1,"name":"first_online","label":"First Online","group":{"name":"ChapterHistory","label":"Chapter History"}}]}}