{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,14]],"date-time":"2026-06-14T21:51:07Z","timestamp":1781473867910,"version":"3.54.1"},"reference-count":36,"publisher":"MDPI AG","issue":"21","license":[{"start":{"date-parts":[[2025,11,5]],"date-time":"2025-11-05T00:00:00Z","timestamp":1762300800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Cincinnati Children\u2019s Hospital Research Development Funds","award":["5RO1NS115662"],"award-info":[{"award-number":["5RO1NS115662"]}]},{"name":"Cincinnati Children\u2019s Hospital Research Development Funds","award":["5RO1AR073379"],"award-info":[{"award-number":["5RO1AR073379"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Myotonic Dystrophy type 1 (DM1) is a complex disease affecting multiple tissues, including skeletal and cardiac muscles, the brain and the eyes. DM1 results from an expansion of CTG repeats in the 3\u2032 UTR of the DMPK gene. Previously, we described that the small-molecule inhibitor of GSK3\u03b2, tideglusib (TG), reduces DM1 pathology in DM1 cell and mouse models by correcting the GSK3\u03b2-CUGBP1 pathway, decreasing the mutant CUG-containing RNA. Respectively, clinical trials using TG showed promising results for patients with congenital DM1 (CDM1). The drug development in DM1 human studies needs specific and noninvasive biomarkers. We examined the blood levels of active GSK3\u03b2 in different clinical forms of DM1 and found an increase in active GSK3\u03b2 in the peripheral blood mononuclear cells (PBMCs) in patients with CDM1, juvenile DM1 and adult-onset DM1 vs. unaffected patients. The blood levels of active GSK3\u03b2 correlate with the length of CTG repeats and severity of muscle weakness. Thrombospondin and TGF\u03b2, linked to the TG-GSK3\u03b2 pathway in DM1, are also elevated in the DM1 patients\u2019 blood. These findings show that the blood levels of active GSK3\u03b2 might be developed as a potential noninvasive biomarker of muscle weakness in DM1.<\/jats:p>","DOI":"10.3390\/ijms262110760","type":"journal-article","created":{"date-parts":[[2025,11,5]],"date-time":"2025-11-05T13:19:29Z","timestamp":1762348769000},"page":"10760","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Elevated Levels of Active GSK3\u03b2 in the Blood of Patients with Myotonic Dystrophy Type 1 Correlate with Muscle Weakness"],"prefix":"10.3390","volume":"26","author":[{"given":"Katherine","family":"Jennings","sequence":"first","affiliation":[{"name":"Division of Neurology, Cincinnati Children\u2019s Hospital, 3333 Burnet Ave., Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cuixia","family":"Tian","sequence":"additional","affiliation":[{"name":"Division of Neurology, Cincinnati Children\u2019s Hospital, 3333 Burnet Ave., Cincinnati, OH 45229, USA"},{"name":"Department of Pediatrics, University of Cincinnati, 2600 Clifton Ave., Cincinnati, OH 45221, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1834-4808","authenticated-orcid":false,"given":"Rebeccah L.","family":"Brown","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Cincinnati, 2600 Clifton Ave., Cincinnati, OH 45221, USA"},{"name":"Department of Surgery, Cincinnati Children\u2019s Hospital, 3333 Burnet Ave., Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4990-5754","authenticated-orcid":false,"given":"Paul S.","family":"Horn","sequence":"additional","affiliation":[{"name":"Division of Neurology, Cincinnati Children\u2019s Hospital, 3333 Burnet Ave., Cincinnati, OH 45229, USA"},{"name":"Department of Pediatrics, University of Cincinnati, 2600 Clifton Ave., Cincinnati, OH 45221, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2757-8131","authenticated-orcid":false,"given":"Benedikt","family":"Schoser","sequence":"additional","affiliation":[{"name":"Department of Neurology, Friedrich-Baur-Institute, LMU Clinics, Ludwig-Maximilians-University, Ziemssenstr., 80336 Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3786-1133","authenticated-orcid":false,"given":"Hani","family":"Kushlaf","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Cincinnati, 2600 Clifton Ave., Cincinnati, OH 45221, USA"},{"name":"Department of Neurology & Rehabilitation Medicine, University of Cincinnati, 260 Stetson St., Cincinnati, OH 45219, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nikolai A.","family":"Timchenko","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Cincinnati, 2600 Clifton Ave., Cincinnati, OH 45221, USA"},{"name":"Department of Surgery, Cincinnati Children\u2019s Hospital, 3333 Burnet Ave., Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lubov","family":"Timchenko","sequence":"additional","affiliation":[{"name":"Division of Neurology, Cincinnati Children\u2019s Hospital, 3333 Burnet Ave., Cincinnati, OH 45229, USA"},{"name":"Department of Pediatrics, University of Cincinnati, 2600 Clifton Ave., Cincinnati, OH 45221, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,11,5]]},"reference":[{"key":"ref_1","unstructured":"Harper, P.S. 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