{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T02:48:20Z","timestamp":1790131700195,"version":"4.0.1"},"reference-count":31,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2025,4,1]],"date-time":"2025-04-01T00:00:00Z","timestamp":1743465600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2025,4,1]],"date-time":"2025-04-01T00:00:00Z","timestamp":1743465600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Nucleic Acid Therapeutics"],"published-print":{"date-parts":[[2025,4,1]]},"abstract":"<jats:p>\n                    Duchenne muscular dystrophy (DMD) is caused by mutations of the\n                    <jats:italic toggle=\"yes\">DMD<\/jats:italic>\n                    gene that prevent the expression of functional dystrophin protein. BMN 351 is an antisense oligonucleotide (ASO) designed to induce skipping of exon 51 of dystrophin pre-mRNA and production of internally deleted but functional dystrophin. We determined whether extended-term BMN 351 dosing leads to exon skipping, dystrophin production, and improved motor function in hDMDdel52\/\n                    <jats:italic toggle=\"yes\">mdx<\/jats:italic>\n                    mice containing a human exon 52-deleted\n                    <jats:italic toggle=\"yes\">DMD<\/jats:italic>\n                    transgene. Weekly intravenous doses of vehicle, 6 mg\/kg BMN 351, or 18 mg\/kg BMN 351 were administered for 25 weeks, and samples were analyzed 4 and 12 weeks post-dosing. BMN 351 produced dose-dependent exon skipping levels in the heart and quadriceps muscles, accompanied by dose-dependent increases in mean dystrophin levels of 17% to 55% 12 weeks post-dosing. Compared with vehicle-treated hDMDdel52\/\n                    <jats:italic toggle=\"yes\">mdx<\/jats:italic>\n                    mice, BMN 351 ameliorated DMD-related histopathologic changes in the gastrocnemius muscle and heart. Both BMN 351 doses preserved fine motor kinematics, which was worse in vehicle-treated hDMDdel52\/\n                    <jats:italic toggle=\"yes\">mdx<\/jats:italic>\n                    mice compared with wild-type 4 and 12 weeks post-dosing. Liver samples demonstrated findings consistent with ASO accumulation, to which mice are considered especially sensitive compared to humans and other non-clinical species. These results support further non-clinical and clinical development of BMN 351.\n                  <\/jats:p>","DOI":"10.1089\/nat.2024.0050","type":"journal-article","created":{"date-parts":[[2025,2,7]],"date-time":"2025-02-07T03:21:14Z","timestamp":1738898474000},"page":"81-92","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":4,"title":["BMN 351-Induced Exon Skipping and Dystrophin Expression in Skeletal and Cardiac Muscle Lead to Preservation of Motor Function in a Mouse Model of Exon 51 Skip-Amenable Duchenne Muscular Dystrophy"],"prefix":"10.1177","volume":"35","author":[{"given":"Todd","family":"Oppeneer","sequence":"first","affiliation":[{"name":"BioMarin Pharmaceutical Inc., San Rafael, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yulan","family":"Qi","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc., San Rafael, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joshua","family":"Henshaw","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc., San Rafael, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kevin","family":"Larimore","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc., San Rafael, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jukka","family":"Puoliv\u00e4li","sequence":"additional","affiliation":[{"name":"Charles River Discovery Research Services, Kuopio, Finland."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caitlyn","family":"Carter","sequence":"additional","affiliation":[{"name":"Charles River Laboratories, Inc., Mattawan, Michigan, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierluigi","family":"Fant","sequence":"additional","affiliation":[{"name":"Charles River Laboratories France Safety Assessment SAS, Saint-Germain-Nuelles, France."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sebastian","family":"Brennan","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc., San Rafael, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura A.","family":"Wetzel","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc., San Rafael, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monika A.","family":"Sigg","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc., San Rafael, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charles A.","family":"O\u2019neill","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc., San Rafael, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,4,14]]},"reference":[{"key":"e_1_3_6_2_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01430-8"},{"key":"e_1_3_6_3_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-021-00248-3"},{"key":"e_1_3_6_4_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0631-3"},{"key":"e_1_3_6_5_2","doi-asserted-by":"publisher","DOI":"10.3390\/jpm11010046"},{"key":"e_1_3_6_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2011.01.072"},{"key":"e_1_3_6_7_2","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.150.5.1209"},{"key":"e_1_3_6_8_2","first-page":"498","article-title":"The molecular basis for Duchenne versus Becker muscular dystrophy: Correlation of severity with type of deletion","volume":"45","author":"Koenig M","year":"1989","unstructured":"Koenig M, , Beggs AH, , Moyer M, et al. The molecular basis for Duchenne versus Becker muscular dystrophy: Correlation of severity with type of deletion. Am J Hum Genet, 1989; 45:498\u2013506.","journal-title":"Am J Hum Genet"},{"key":"e_1_3_6_9_2","doi-asserted-by":"publisher","DOI":"10.1136\/jmedgenet-2015-103387"},{"key":"e_1_3_6_10_2","doi-asserted-by":"publisher","DOI":"10.1080\/14712598.2017.1271872"},{"key":"e_1_3_6_11_2","doi-asserted-by":"publisher","DOI":"10.1002\/humu.20918"},{"key":"e_1_3_6_12_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-210682"},{"key":"e_1_3_6_13_2","doi-asserted-by":"publisher","DOI":"10.1002\/humu.22758"},{"key":"e_1_3_6_14_2","unstructured":"Package insert for Exondys 51 (eteplirsen). Sarepta Therapeutics Inc. Cambridge MA; 2016. Available from: https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2016\/206488lbl.pdf [Last accessed: October1 2024]."},{"key":"e_1_3_6_15_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00204-021-03184-z"},{"key":"e_1_3_6_16_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2021.117337"},{"key":"e_1_3_6_17_2","doi-asserted-by":"publisher","DOI":"10.1080\/15476286.2021.1874161"},{"key":"e_1_3_6_18_2","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2022.0063"},{"key":"e_1_3_6_19_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0193289"},{"key":"e_1_3_6_20_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0244215"},{"key":"e_1_3_6_21_2","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2024.0049"},{"key":"e_1_3_6_22_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0243052"},{"key":"e_1_3_6_23_2","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2019.0824"},{"key":"e_1_3_6_24_2","doi-asserted-by":"publisher","DOI":"10.1177\/0192623310386499"},{"key":"e_1_3_6_25_2","doi-asserted-by":"publisher","DOI":"10.1038\/s42003-022-03980-y"},{"key":"e_1_3_6_26_2","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddu366"},{"key":"e_1_3_6_27_2","doi-asserted-by":"publisher","DOI":"10.3122\/jabfm.2012.04.110183"},{"key":"e_1_3_6_28_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70272-8"},{"key":"e_1_3_6_29_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ebiom.2019.06.036"},{"key":"e_1_3_6_30_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0195850"},{"key":"e_1_3_6_31_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-023-44270-9"},{"key":"e_1_3_6_32_2","doi-asserted-by":"publisher","DOI":"10.1177\/0192623314551840"}],"container-title":["Nucleic Acid Therapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1089\/nat.2024.0050","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1089\/nat.2024.0050","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1089\/nat.2024.0050","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T21:03:28Z","timestamp":1777669408000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1089\/nat.2024.0050"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,4,1]]},"references-count":31,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2025,4,1]]}},"alternative-id":["10.1089\/nat.2024.0050"],"URL":"https:\/\/doi.org\/10.1089\/nat.2024.0050","relation":{},"ISSN":["2159-3337","2159-3345"],"issn-type":[{"value":"2159-3337","type":"print"},{"value":"2159-3345","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,4,1]]}}}