{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T13:28:31Z","timestamp":1790774911871,"version":"4.1.0"},"reference-count":200,"publisher":"MDPI AG","issue":"9","license":[{"start":{"date-parts":[[2022,4,21]],"date-time":"2022-04-21T00:00:00Z","timestamp":1650499200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100002426","name":"Telethon Foundation","doi-asserted-by":"publisher","award":["GGP19035"],"award-info":[{"award-number":["GGP19035"]}],"id":[{"id":"10.13039\/501100002426","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007393","name":"French Muscular Dystrophy Association","doi-asserted-by":"publisher","award":["23054"],"award-info":[{"award-number":["23054"]}],"id":[{"id":"10.13039\/100007393","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Italian Ministry of Health","award":["RF-12368521"],"award-info":[{"award-number":["RF-12368521"]}]},{"name":"EU Horizon 2020","award":["101016072"],"award-info":[{"award-number":["101016072"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy affecting many different body tissues, predominantly skeletal and cardiac muscles and the central nervous system. The expansion of CTG repeats in the DM1 protein-kinase (DMPK) gene is the genetic cause of the disease. The pathogenetic mechanisms are mainly mediated by the production of a toxic expanded CUG transcript from the DMPK gene. With the availability of new knowledge, disease models, and technical tools, much progress has been made in the discovery of altered pathways and in the potential of therapeutic intervention, making the path to the clinic a closer reality. In this review, we describe and discuss the molecular therapeutic strategies for DM1, which are designed to directly target the CTG genomic tract, the expanded CUG transcript or downstream signaling molecules.<\/jats:p>","DOI":"10.3390\/ijms23094622","type":"journal-article","created":{"date-parts":[[2022,4,22]],"date-time":"2022-04-22T05:07:22Z","timestamp":1650604042000},"page":"4622","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":37,"title":["Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing"],"prefix":"10.3390","volume":"23","author":[{"given":"Mariapaola","family":"Izzo","sequence":"first","affiliation":[{"name":"Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8309-4775","authenticated-orcid":false,"given":"Jonathan","family":"Battistini","sequence":"additional","affiliation":[{"name":"Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Provenzano","sequence":"additional","affiliation":[{"name":"Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8624-7738","authenticated-orcid":false,"given":"Fabio","family":"Martelli","sequence":"additional","affiliation":[{"name":"Molecular Cardiology Laboratory, IRCCS Policlinico San Donato, San Donato Milanese, 20097 Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3333-3384","authenticated-orcid":false,"given":"Beatrice","family":"Cardinali","sequence":"additional","affiliation":[{"name":"Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3508-7623","authenticated-orcid":false,"given":"Germana","family":"Falcone","sequence":"additional","affiliation":[{"name":"Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2022,4,21]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"e1045","DOI":"10.1212\/WNL.0000000000011425","article-title":"Population-Based Prevalence of Myotonic Dystrophy Type 1 Using Genetic Analysis of Statewide Blood Screening Program","volume":"96","author":"Johnson","year":"2021","journal-title":"Neurology"},{"key":"ref_2","unstructured":"Bird, T.D. 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