{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,26]],"date-time":"2026-08-26T05:20:36Z","timestamp":1787721636870,"version":"build-2784847793"},"reference-count":29,"publisher":"SAGE Publications","issue":"5","license":[{"start":{"date-parts":[[2025,8,29]],"date-time":"2025-08-29T00:00:00Z","timestamp":1756425600000},"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":[[2025,10,1]]},"abstract":"<jats:p>\n                    The severe X-linked degenerative neuromuscular disease Duchenne muscular dystrophy (DMD) is caused by the loss of dystrophin through reading frame disruptive mutations in the DMD gene. Dystrophin protein is crucial for the stability of the muscle. Targeting specific exons with antisense oligonucleotides (ASO) will prevent inclusion of the exon during pre-mRNA splicing, which can restore the reading frame, facilitating the production of partially functional dystrophin proteins. For DMD, four ASOs of the phosphorodiamidate morpholino oligomer (PMOs) chemistry are FDA approved. It is anticipated that improved delivery to skeletal muscle and heart will lead to larger therapeutic results. With our research, we sought to identify muscle-homing peptides that can achieve increased delivery of ASOs to muscle or heart when conjugated to PMOs. We applied\n                    <jats:italic toggle=\"yes\">in\u00a0vivo<\/jats:italic>\n                    phage display biopanning mouse models for DMD to identify muscle-homing peptides while simultaneously negatively selecting peptides that home to unwanted organs, such as the kidney and liver. After confirmation of the muscle homing ability\n                    <jats:italic toggle=\"yes\">in vitro<\/jats:italic>\n                    , we conjugated selected candidate peptides to PMOs to be tested\n                    <jats:italic toggle=\"yes\">in\u00a0vivo<\/jats:italic>\n                    , where we found that conjugation of one specific muscle homing peptide led to significantly improved delivery to muscle, with a small improvement in exon skipping and dystrophin restoration.\n                  <\/jats:p>","DOI":"10.1177\/21593337251371708","type":"journal-article","created":{"date-parts":[[2025,10,8]],"date-time":"2025-10-08T17:15:51Z","timestamp":1759943751000},"page":"220-233","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["<i>In Vivo<\/i>\n                    Phage Display for the Identification of Muscle Homing Peptides to Improve the Delivery of Phosphorodiamidate Morpholino Oligomers for Duchenne Muscular Dystrophy Therapy"],"prefix":"10.1177","volume":"35","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1870-2962","authenticated-orcid":false,"given":"Anne-Fleur E.","family":"Schneider","sequence":"first","affiliation":[{"name":"Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christa L. Tanganyika-de","family":"Winter","sequence":"additional","affiliation":[{"name":"Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hailiang","family":"Mei","sequence":"additional","affiliation":[{"name":"Department of Molecular Epidemiology, Leiden University Medical Center, Leiden, The Netherlands."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silvana M.G.","family":"Jirka","sequence":"additional","affiliation":[{"name":"Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xuyu","family":"Tan","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emily G.","family":"Thompson","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristin","family":"Ha","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anindita","family":"Mitra","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephanie","family":"Garcia","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marleen","family":"Luimes","sequence":"additional","affiliation":[{"name":"Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ryan","family":"Oliver","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathy Y.","family":"Morgan","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vincent","family":"Guerlavais","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1565-654X","authenticated-orcid":false,"given":"Annemieke","family":"Aartsma-Rus","sequence":"additional","affiliation":[{"name":"Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,8,29]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1038\/nrneurol.2017.148"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkad067"},{"key":"e_1_3_5_4_2","volume-title":"Muscular Dystrophy. StatPearls","author":"LaPelusa A","year":"2025","unstructured":"LaPelusa A, , Asuncion RMD, , Kentris M. Muscular Dystrophy. StatPearls. StatPearls Publishing LLC: Treasure Island, FL; 2025."},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.5535\/arm.2017.41.2.306"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.880100513"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1155\/2015\/543282"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1111\/jpc.12868"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.rehab.2013.06.002"},{"key":"e_1_3_5_10_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(19)32910-1"},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM198805263182104"},{"key":"e_1_3_5_12_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-021-00248-3"},{"key":"e_1_3_5_13_2","first-page":"184","article-title":"The role of fibrosis in Duchenne muscular dystrophy","volume":"31","author":"Klingler W","year":"2012","unstructured":"Klingler W, , Jurkat-Rott K, , Lehmann-Horn F, et al. The role of fibrosis in Duchenne muscular dystrophy. Acta Myol, 2012; 31:184\u2013195.","journal-title":"Acta Myol"},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.02.009"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1261\/rna.653607"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1016\/0888-7543(88)90113-9"},{"key":"e_1_3_5_17_2","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2016.16437"},{"key":"e_1_3_5_18_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25951"},{"key":"e_1_3_5_19_2","doi-asserted-by":"publisher","DOI":"10.15252\/emmm.202013243"},{"key":"e_1_3_5_20_2","doi-asserted-by":"publisher","DOI":"10.1002\/jmr.630"},{"key":"e_1_3_5_21_2","doi-asserted-by":"publisher","DOI":"10.3390\/biomedicines6020051"},{"key":"e_1_3_5_22_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.crtox.2024.100182"},{"key":"e_1_3_5_23_2","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2013.0448"},{"key":"e_1_3_5_24_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2017.10.004"},{"key":"e_1_3_5_25_2","doi-asserted-by":"publisher","DOI":"10.3390\/pharmaceutics13122065"},{"key":"e_1_3_5_26_2","doi-asserted-by":"publisher","DOI":"10.3390\/ijms241814035"},{"key":"e_1_3_5_27_2","doi-asserted-by":"publisher","DOI":"10.1124\/dmd.123.001425"},{"key":"e_1_3_5_28_2","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2022.0054"},{"key":"e_1_3_5_29_2","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkz354"},{"key":"e_1_3_5_30_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2022.069"}],"container-title":["Nucleic Acid Therapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/21593337251371708","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/21593337251371708","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/21593337251371708","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,26]],"date-time":"2026-08-26T04:40:38Z","timestamp":1787719238000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/21593337251371708"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,8,29]]},"references-count":29,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2025,10,1]]}},"alternative-id":["10.1177\/21593337251371708"],"URL":"https:\/\/doi.org\/10.1177\/21593337251371708","relation":{},"ISSN":["2159-3337","2159-3345"],"issn-type":[{"value":"2159-3337","type":"print"},{"value":"2159-3345","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,8,29]]}}}