{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T19:46:03Z","timestamp":1777923963113,"version":"3.51.4"},"reference-count":49,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2021,3,25]],"date-time":"2021-03-25T00:00:00Z","timestamp":1616630400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2021,3,25]],"date-time":"2021-03-25T00:00:00Z","timestamp":1616630400000},"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":[[2021,4,1]]},"abstract":"<jats:p>\n                    Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease caused by frameshift or nonsense mutations in the\n                    <jats:italic toggle=\"yes\">DMD<\/jats:italic>\n                    gene, resulting in the loss of dystrophin from muscle membranes. Exon skipping using splice-switching oligonucleotides (SSOs) restores the reading frame of\n                    <jats:italic toggle=\"yes\">DMD<\/jats:italic>\n                    pre-mRNA by generating internally truncated but functional dystrophin protein. To potentiate effective tissue-specific targeting by functional SSOs, it is essential to perform accelerated and reliable\n                    <jats:italic toggle=\"yes\">in vitro<\/jats:italic>\n                    screening-based assessment of novel oligonucleotides and drug delivery technologies, such as cell-penetrating peptides, before their\n                    <jats:italic toggle=\"yes\">in vivo<\/jats:italic>\n                    pharmacokinetic and toxicity evaluation. We have established novel canine immortalized myoblast lines by transducing murine cyclin-dependent kinase-4 and human telomerase reverse transcriptase genes into myoblasts isolated from beagle-based wild-type or canine X-linked muscular dystrophy in Japan (CXMD\n                    <jats:sub>J<\/jats:sub>\n                    ) dogs. These myoblast lines exhibited improved myogenic differentiation and increased proliferation rates compared with passage-15 primary parental myoblasts, and their potential to differentiate into myotubes was maintained in later passages. Using these dystrophin-deficient immortalized myoblast lines, we demonstrate that a novel cell-penetrating peptide (Pip8b2)-conjugated SSO markedly improved multiexon skipping activity compared with the respective naked phosphorodiamidate morpholino oligomers.\n                    <jats:italic toggle=\"yes\">In vitro<\/jats:italic>\n                    screening using immortalized canine cell lines will provide a basis for further pharmacological studies on drug delivery tools.\n                  <\/jats:p>","DOI":"10.1089\/nat.2020.0907","type":"journal-article","created":{"date-parts":[[2021,2,10]],"date-time":"2021-02-10T17:37:41Z","timestamp":1612978661000},"page":"172-181","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":14,"title":["Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides"],"prefix":"10.1177","volume":"31","author":[{"given":"Yuichiro","family":"Tone","sequence":"first","affiliation":[{"name":"Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Japan."},{"name":"Discovery Research Laboratories in Tsukuba, Nippon Shinyaku Co., Ltd., Tsukuba, Japan."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kamel","family":"Mamchaoui","sequence":"additional","affiliation":[{"name":"Center of Research in Myology, Sorbonne University, INSERM, Institute of Myology, Paris, France."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria K.","family":"Tsoumpra","sequence":"additional","affiliation":[{"name":"Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Japan."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yasumasa","family":"Hashimoto","sequence":"additional","affiliation":[{"name":"Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Japan."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Reiko","family":"Terada","sequence":"additional","affiliation":[{"name":"Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Japan."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rika","family":"Maruyama","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, Canada."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael J.","family":"Gait","sequence":"additional","affiliation":[{"name":"Medical Research Council, Laboratory of Molecular Biology, 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Kingdom."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vincent","family":"Mouly","sequence":"additional","affiliation":[{"name":"Center of Research in Myology, Sorbonne University, INSERM, Institute of Myology, Paris, France."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yoshitsugu","family":"Aoki","sequence":"additional","affiliation":[{"name":"Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, 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