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We have developed an in vitro screening platform based on ddPCR and in-cell western to quantify these mRNAs and proteins and characterized &amp;gt;20 cell models to define DM1 biomarkers that could be useful for drug screening. DMPK protein levels were reduced in DM1-immortalized myoblasts and myotubes, but not in fibroblasts, while MBNL1 protein was consistently lower in all DM1 myogenic cultures, whether primary or immortalized. Myogenic differentiation of cultures led to an increase in DMPK mRNA expression, which was translated into increased MBNL1 sequestration in foci. We further corroborated the platform\u2019s ability to assess therapeutic outcomes, evaluating the effect of a DMPK gapmer ASO and one siRNA: while the gapmer increased MBNL1 protein levels, the siRNA had no significant effect on MBNL1 release. Our platform and the in-depth characterization of some of the most used models would be of use to the DM1 research community.<\/jats:p>","DOI":"10.1093\/narmme\/ugaf023","type":"journal-article","created":{"date-parts":[[2025,6,20]],"date-time":"2025-06-20T08:01:02Z","timestamp":1750406462000},"source":"Crossref","is-referenced-by-count":1,"title":["Characterization of\n                    <i>DMPK<\/i>\n                    and MBNL1 expression in cell models of myotonic dystrophy: a\u00a0platform for drug screening"],"prefix":"10.1093","volume":"2","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4711-7495","authenticated-orcid":false,"given":"Andrea","family":"L\u00f3pez-Mart\u00ednez","sequence":"first","affiliation":[{"name":"Nucleic Acid Therapeutics for Rare Disorders (NAT-RD), Biobizkaia Health Research Institute , 48903\u00a0 Barakaldo ,","place":["Spain"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sergio","family":"Mart\u00edn-Gonz\u00e1lez","sequence":"additional","affiliation":[{"name":"Nucleic Acid Therapeutics for Rare Disorders (NAT-RD), Biobizkaia Health Research Institute , 48903\u00a0 Barakaldo ,","place":["Spain"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Noemi","family":"Torres-Conde","sequence":"additional","affiliation":[{"name":"Nucleic Acid Therapeutics for Rare Disorders (NAT-RD), Biobizkaia Health Research Institute , 48903\u00a0 Barakaldo ,","place":["Spain"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nahia","family":"Alcal\u00e1-Manso","sequence":"additional","affiliation":[{"name":"Nucleic Acid Therapeutics for Rare Disorders (NAT-RD), Biobizkaia Health Research Institute , 48903\u00a0 Barakaldo ,","place":["Spain"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4567-4837","authenticated-orcid":false,"given":"Abdullah","family":"Al-Ani","sequence":"additional","affiliation":[{"name":"Nucleic Acid Therapeutics for Rare Disorders (NAT-RD), Biobizkaia Health Research Institute , 48903\u00a0 Barakaldo ,","place":["Spain"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Adolfo","family":"L\u00f3pez\u00a0de\u00a0Munain","sequence":"additional","affiliation":[{"name":"Group of Neurosciences , Department of Pediatrics and Neuroscience, Faculty of Medicine and Nursing, University of Basque Country (UPV\/EHU), 20014 Donostia -San Sebastian,","place":["Spain"]},{"name":"Groups of Sensorial Neurodegeneration and Neuromuscular Diseases, Neuroscience Area, BioGipuzkoa-BioDonostia Health Research Institute (IIS Biodonostia) , 20014 Donostia -San Sebastian,","place":["Spain"]},{"name":"CIBERNED, ISCIII (CIBER, Carlos III Institute, Spanish Ministry of Sciences and Innovation) , 28031 Madrid ,","place":["Spain"]},{"name":"Hospital Universitario Donostia. OSAKIDETZA Department of Neurology, , 20014 Donostia -San Sebasti\u00e1n,","place":["Spain"]},{"name":"School of Medicine, University of Deusto Department of Medicine, , 48007\u00a0 Bilbao ,","place":["Spain"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anne","family":"Bigot","sequence":"additional","affiliation":[{"name":"MyoLine, Sorbonne Universit\u00e9, Inserm, Institut de Myologie , Centre de Recherche en Myologie, F- 75013 Paris ,","place":["France"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kamel","family":"Mamchaoui","sequence":"additional","affiliation":[{"name":"MyoLine, Sorbonne Universit\u00e9, Inserm, Institut de Myologie , Centre de Recherche en Myologie, F- 75013 Paris ,","place":["France"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7414-212X","authenticated-orcid":false,"given":"Gisela","family":"Nogales-Gadea","sequence":"additional","affiliation":[{"name":"Grup de REcerca Neuromuscular de BAdalona (GRENBA), Institut d\u2019Investigaci\u00f3 en Ci\u00e8ncies de la Salut Germans Trias i Pujol (IGTP), Campus Can Ruti, Universitat Aut\u00f2noma de Barcelona , 08916 Badalona ,","place":["Spain"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7703-3255","authenticated-orcid":false,"given":"Virginia","family":"Arechavala-Gomeza","sequence":"additional","affiliation":[{"name":"Nucleic Acid Therapeutics for Rare Disorders (NAT-RD), Biobizkaia Health Research Institute , 48903\u00a0 Barakaldo ,","place":["Spain"]},{"name":"Ikerbasque, Basque Foundation for Science , 48009\u00a0 Bilbao ,","place":["Spain"]}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"286","published-online":{"date-parts":[[2025,6,20]]},"reference":[{"key":"2025072000520895000_B1","doi-asserted-by":"publisher","first-page":"507","DOI":"10.1212\/CPJ.000000000000053","article-title":"Consensus-based care recommendations for adults 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