{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T13:11:46Z","timestamp":1792674706447,"version":"4.5.0"},"reference-count":68,"publisher":"MDPI AG","issue":"12","license":[{"start":{"date-parts":[[2018,12,18]],"date-time":"2018-12-18T00:00:00Z","timestamp":1545091200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004923","name":"AFM-T\u00e9l\u00e9thon","doi-asserted-by":"publisher","award":["21182"],"award-info":[{"award-number":["21182"]}],"id":[{"id":"10.13039\/501100004923","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Myotonic dystrophy type 1 (DM1), the most common cause of adult-onset muscular dystrophy, is autosomal dominant, multisystemic disease with characteristic symptoms including myotonia, heart defects, cataracts and testicular atrophy. DM1 disease is being successfully modelled in Drosophila allowing to identify and validate new pathogenic mechanisms and potential therapeutic strategies. Here we provide an overview of insights gained from fruit fly DM1 models, either: (i) fundamental with particular focus on newly identified gene deregulations and their link with DM1 symptoms; or (ii) applied via genetic modifiers and drug screens to identify promising therapeutic targets.<\/jats:p>","DOI":"10.3390\/ijms19124104","type":"journal-article","created":{"date-parts":[[2018,12,18]],"date-time":"2018-12-18T05:47:45Z","timestamp":1545112065000},"page":"4104","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":22,"title":["Dissecting Pathogenetic Mechanisms and Therapeutic Strategies in Drosophila Models of Myotonic Dystrophy Type 1"],"prefix":"10.3390","volume":"19","author":[{"given":"Anissa","family":"Souidi","sequence":"first","affiliation":[{"name":"GReD, INSERM U1103, CNRS, UMR6293, University of Clermont Auvergne, 28 Place Henri Dunant, 63000 Clermont-Ferrand, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monika","family":"Zmojdzian","sequence":"additional","affiliation":[{"name":"GReD, INSERM U1103, CNRS, UMR6293, University of Clermont Auvergne, 28 Place Henri Dunant, 63000 Clermont-Ferrand, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Krzysztof","family":"Jagla","sequence":"additional","affiliation":[{"name":"GReD, INSERM U1103, CNRS, UMR6293, University of Clermont Auvergne, 28 Place Henri Dunant, 63000 Clermont-Ferrand, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2018,12,18]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"594","DOI":"10.1016\/j.bbadis.2014.05.019","article-title":"Myotonic dystrophies: An update on clinical aspects, genetic, pathology, and molecular pathomechanisms","volume":"1852","author":"Meola","year":"2015","journal-title":"Biochim. Biophys. Acta BBA\u2014Mol. Basis Dis."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1038\/355547a0","article-title":"Detection of an unstable fragment of DNA specific to individuals with myotonic dystrophy","volume":"355","author":"Buxton","year":"1992","journal-title":"Nature"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1017\/S0012162205000927","article-title":"Myotonic dystrophy: muscle involvement in relation to disease type and size of expanded CTG-repeat sequence","volume":"47","author":"Kroksmark","year":"2005","journal-title":"Dev. Med. Child Neurol."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"505","DOI":"10.1136\/adc.50.7.505","article-title":"Congenital myotonic dystrophy in Britain. I. Clinical aspects","volume":"50","author":"Harper","year":"1975","journal-title":"Arch. Dis. Child."},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"417","DOI":"10.1111\/j.1399-0004.1975.tb00351.x","article-title":"Thin ribs in neonatal myotonic dystrophy","volume":"7","author":"Fried","year":"1975","journal-title":"Clin. Genet."},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"813","DOI":"10.1001\/jama.1995.03530100053034","article-title":"Cardiac involvement in a large kindred with myotonic dystrophy. Quantitative assessment and relation to size of CTG repeat expansion","volume":"274","author":"Tokgozoglu","year":"1995","journal-title":"JAMA"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"292","DOI":"10.1016\/S0002-8703(97)70137-6","article-title":"Cardiac abnormalities and cytosine-thymine-guanine trinucleotide repeats in myotonic dystrophy","volume":"134","author":"Hayashi","year":"1997","journal-title":"Am. Heart J."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"790","DOI":"10.1136\/jnnp.2008.162594","article-title":"Risk of arrhythmia in type I myotonic dystrophy: the role of clinical and genetic variables","volume":"80","author":"Cudia","year":"2009","journal-title":"J. Neurol. Neurosurg. Psychiatry"},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"444","DOI":"10.1046\/j.1540-8167.2002.00444.x","article-title":"Severity of cardiac conduction involvement and arrhythmias in myotonic dystrophy type 1 correlates with age and CTG repeat length","volume":"13","author":"Groh","year":"2002","journal-title":"J. Cardiovasc. Electrophysiol."},{"key":"ref_10","doi-asserted-by":"crossref","unstructured":"Renna, L.V., Bos\u00e8, F., Iachettini, S., Fossati, B., Saraceno, L., Milani, V., Colombo, R., Meola, G., and Cardani, R. (2017). Receptor and post-receptor abnormalities contribute to insulin resistance in myotonic dystrophy type 1 and type 2 skeletal muscle. PLoS ONE, 12.","DOI":"10.1371\/journal.pone.0184987"},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"104","DOI":"10.1002\/ana.410350116","article-title":"Myotonic dystrophy patients have larger CTG expansions in skeletal muscle than in leukocytes","volume":"35","author":"Thornton","year":"1994","journal-title":"Ann. Neurol."},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"3558","DOI":"10.1093\/hmg\/dds185","article-title":"Somatic instability of the expanded CTG triplet repeat in myotonic dystrophy type 1 is a heritable quantitative trait and modifier of disease severity","volume":"21","author":"Morales","year":"2012","journal-title":"Hum. Mol. Genet."},{"key":"ref_13","first-page":"414","article-title":"Characteristics of intergenerational contractions of the CTG repeat in myotonic dystrophy","volume":"54","author":"Ashizawa","year":"1994","journal-title":"Am. J. Hum. Genet."},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1126\/science.8469976","article-title":"Decreased expression of myotonin-protein kinase messenger RNA and protein in adult form of myotonic dystrophy","volume":"260","author":"Fu","year":"1993","journal-title":"Science"},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"316","DOI":"10.1038\/ng0796-316","article-title":"Abnormal myotonic dystrophy protein kinase levels produce only mild myopathy in mice","volume":"13","author":"Jansen","year":"1996","journal-title":"Nat. Genet."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"1421","DOI":"10.1002\/1097-4598(200009)23:9<1421::AID-MUS14>3.0.CO;2-Y","article-title":"Reduced expression of DMAHP\/SIX5 gene in myotonic dystrophy muscle","volume":"23","author":"Inukai","year":"2000","journal-title":"Muscle Nerve"},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"1871","DOI":"10.1093\/nar\/28.9.1871","article-title":"Functional analysis of the homeodomain protein SIX5","volume":"28","author":"Harris","year":"2000","journal-title":"Nucleic Acids Res."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1038\/75490","article-title":"Mice deficient in Six5 develop cataracts: implications for myotonic dystrophy","volume":"25","author":"Klesert","year":"2000","journal-title":"Nat. Genet."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"1769","DOI":"10.1126\/science.289.5485.1769","article-title":"Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat","volume":"289","author":"Mankodi","year":"2000","journal-title":"Science"},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"7388","DOI":"10.1073\/pnas.94.14.7388","article-title":"Expansion of a CUG trinucleotide repeat in the 3\u2019 untranslated region of myotonic dystrophy protein kinase transcripts results in nuclear retention of transcripts","volume":"94","author":"Davis","year":"1997","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"995","DOI":"10.1083\/jcb.128.6.995","article-title":"Foci of trinucleotide repeat transcripts in nuclei of myotonic dystrophy cells and tissues","volume":"128","author":"Taneja","year":"1995","journal-title":"J. Cell Biol."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"2766","DOI":"10.1093\/nar\/29.13.2766","article-title":"In vivo co-localisation of MBNL protein with DMPK expanded-repeat transcripts","volume":"29","author":"Fardaei","year":"2001","journal-title":"Nucleic Acids Res."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"2165","DOI":"10.1093\/hmg\/10.19.2165","article-title":"Muscleblind localizes to nuclear foci of aberrant RNA in myotonic dystrophy types 1 and 2","volume":"10","author":"Mankodi","year":"2001","journal-title":"Hum. Mol. Genet."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1017\/S1355838200991544","article-title":"Expanded CUG repeat RNAs form hairpins that activate the double-stranded RNA-dependent protein kinase PKR","volume":"6","author":"Tian","year":"2000","journal-title":"RNA"},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"6477","DOI":"10.1093\/nar\/gkp681","article-title":"MBNL and CELF proteins regulate alternative splicing of the skeletal muscle chloride channel CLCN1","volume":"37","author":"Kino","year":"2009","journal-title":"Nucleic Acids Res."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"20333","DOI":"10.1073\/pnas.0809045105","article-title":"A postnatal switch of CELF and MBNL proteins reprograms alternative splicing in the developing heart","volume":"105","author":"Kalsotra","year":"2008","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"1978","DOI":"10.1126\/science.1088583","article-title":"A Muscleblind Knockout Model for Myotonic Dystrophy","volume":"302","author":"Kanadia","year":"2003","journal-title":"Science"},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"11748","DOI":"10.1073\/pnas.0604970103","article-title":"Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy","volume":"103","author":"Kanadia","year":"2006","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_29","doi-asserted-by":"crossref","unstructured":"Chakraborty, M., Sellier, C., Ney, M., Villa, P., Charlet-Berguerand, N., Artero, R., and Llamusi, B. (2018). Daunorubicin reduces MBNL1 titration by expanded CUG repeat RNA and rescues cardiac dysfunctions in a Drosophila model of myotonic dystrophy. Dis. Model. Mech., dmm032557.","DOI":"10.1242\/dmm.032557"},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1016\/j.molcel.2007.11.024","article-title":"Conserved GU-rich elements mediate mRNA decay by binding to CUG-binding protein 1","volume":"29","author":"Vlasova","year":"2008","journal-title":"Mol. Cell"},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1016\/S0248-4900(03)00010-8","article-title":"A functional deadenylation assay identifies human CUG-BP as a deadenylation factor","volume":"95","author":"Paillard","year":"2003","journal-title":"Biol. Cell"},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1093\/hmg\/ddt419","article-title":"Evaluating the effects of CELF1 deficiency in a mouse model of RNA toxicity","volume":"23","author":"Kim","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"1114","DOI":"10.1101\/gr.169101","article-title":"A systematic analysis of human disease-associated gene sequences in Drosophila melanogaster","volume":"11","author":"Reiter","year":"2001","journal-title":"Genome Res."},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1038\/nrg1503","article-title":"Drosophila, the golden bug, emerges as a tool for human genetics","volume":"6","author":"Bier","year":"2005","journal-title":"Nat. Rev. Genet."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"873","DOI":"10.1093\/hmg\/ddi080","article-title":"Myotonic dystrophy associated expanded CUG repeat muscleblind positive ribonuclear foci are not toxic to Drosophila","volume":"14","author":"Houseley","year":"2005","journal-title":"Hum. Mol. Genet."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"2138","DOI":"10.1093\/hmg\/ddl137","article-title":"MBNL1 and CUGBP1 modify expanded CUG-induced toxicity in a Drosophila model of myotonic dystrophy type 1","volume":"15","author":"Ukani","year":"2006","journal-title":"Hum. Mol. Genet."},{"key":"ref_37","doi-asserted-by":"crossref","unstructured":"Garcia-Lopez, A., Monferrer, L., Garcia-Alcover, I., Vicente-Crespo, M., Alvarez-Abril, M.C., and Artero, R.D. (2008). Genetic and Chemical Modifiers of a CUG Toxicity Model in Drosophila. PLoS ONE, 3.","DOI":"10.1371\/journal.pone.0001595"},{"key":"ref_38","doi-asserted-by":"crossref","unstructured":"Yu, Z., Teng, X., and Bonini, N.M. (2011). Triplet Repeat\u2013Derived siRNAs Enhance RNA\u2013Mediated Toxicity in a Drosophila Model for Myotonic Dystrophy. PLoS Genet., 7.","DOI":"10.1371\/journal.pgen.1001340"},{"key":"ref_39","doi-asserted-by":"crossref","unstructured":"Cammarato, A., Ahrens, C.H., Alayari, N.N., Qeli, E., Rucker, J., Reedy, M.C., Zmasek, C.M., Gucek, M., Cole, R.N., and Eyk, J.E.V. (2011). A Mighty Small Heart: The Cardiac Proteome of Adult Drosophila melanogaster. PLoS ONE, 6.","DOI":"10.1371\/journal.pone.0018497"},{"key":"ref_40","first-page":"719","article-title":"The gene tinman is required for specification of the heart and visceral muscles in Drosophila","volume":"118","author":"Bodmer","year":"1993","journal-title":"Dev. Camb. Engl."},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"1922","DOI":"10.1126\/science.1132292","article-title":"Gene Regulatory Networks in the Evolution and Development of the Heart","volume":"313","author":"Olson","year":"2006","journal-title":"Science"},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1016\/1050-1738(94)00032-Q","article-title":"Heart development in Drosophila and its relationship to vertebrates","volume":"5","author":"Bodmer","year":"1995","journal-title":"Trends Cardiovasc. Med."},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"662","DOI":"10.1016\/0735-1097(88)91547-1","article-title":"Pathology of the cardiac conduction system in myotonic dystrophy: A study of 12 cases","volume":"11","author":"Nguyen","year":"1988","journal-title":"J. Am. Coll. Cardiol."},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"305","DOI":"10.1111\/jce.12604","article-title":"Long-term arrhythmia follow-up of patients with myotonic dystrophy","volume":"26","author":"Benhayon","year":"2015","journal-title":"J. Cardiovasc. Electrophysiol."},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"2688","DOI":"10.1056\/NEJMoa062800","article-title":"Electrocardiographic abnormalities and sudden death in myotonic dystrophy type 1","volume":"358","author":"Groh","year":"2008","journal-title":"N. Engl. J. Med."},{"key":"ref_46","first-page":"1569","article-title":"Pentamidine rescues contractility and rhythmicity in a Drosophila model of myotonic dystrophy heart dysfunction","volume":"8","author":"Chakraborty","year":"2015","journal-title":"Dis. Model. Mech."},{"key":"ref_47","doi-asserted-by":"crossref","unstructured":"Plantie, E., Nakamori, M., Renaud, Y., Huguet, A., Choquet, C., Dondi, C., Miquerol, L., Takahashi, M., Gourdon, G., and Junion, G. (2018). Straightjacket\/\u03b1 2\u03b4 3 deregulation is associated with cardiac conduction defects in Myotonic Dystrophy type 1. bioRxiv, 431569.","DOI":"10.1101\/431569"},{"key":"ref_48","first-page":"993","article-title":"(CCUG)n RNA toxicity in a Drosophila model of myotonic dystrophy type 2 (DM2) activates apoptosis","volume":"10","author":"Yenigun","year":"2017","journal-title":"Dis. Model. Mech."},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"1025","DOI":"10.1136\/jcp.2003.015420","article-title":"Cardiac troponins","volume":"57","author":"Sharma","year":"2004","journal-title":"J. Clin. Pathol."},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"737","DOI":"10.1126\/science.280.5364.737","article-title":"Disruption of splicing regulated by a CUG-binding protein in myotonic dystrophy","volume":"280","author":"Philips","year":"1998","journal-title":"Science"},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1006\/dbio.1997.8833","article-title":"The muscleblind gene participates in the organization of Z-bands and epidermal attachments of Drosophila muscles and is regulated by Dmef2","volume":"195","author":"Artero","year":"1998","journal-title":"Dev. Biol."},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"2795","DOI":"10.1093\/hmg\/ddt127","article-title":"Novel Drosophila model of myotonic dystrophy type 1: phenotypic characterization and genome-wide view of altered gene expression","volume":"22","author":"Picchio","year":"2013","journal-title":"Hum. Mol. Genet."},{"key":"ref_53","doi-asserted-by":"crossref","first-page":"704","DOI":"10.1093\/hmg\/dds478","article-title":"Expanded CTG repeats trigger miRNA alterations in Drosophila that are conserved in myotonic dystrophy type 1 patients","volume":"22","author":"Mata","year":"2013","journal-title":"Hum. Mol. Genet."},{"key":"ref_54","doi-asserted-by":"crossref","first-page":"840","DOI":"10.1038\/nsmb.2067","article-title":"Misregulation of miR-1 processing is associated with heart defects in myotonic dystrophy","volume":"18","author":"Rau","year":"2011","journal-title":"Nat. Struct. Mol. Biol."},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1016\/j.cell.2004.09.011","article-title":"Ca(V)1.2 calcium channel dysfunction causes a multisystem disorder including arrhythmia and autism","volume":"119","author":"Splawski","year":"2004","journal-title":"Cell"},{"key":"ref_56","doi-asserted-by":"crossref","first-page":"27487","DOI":"10.1074\/jbc.M109.036541","article-title":"Unique MicroRNA Profile in End-stage Heart Failure Indicates Alterations in Specific Cardiovascular Signaling Networks","volume":"284","author":"Duan","year":"2009","journal-title":"J. Biol. Chem."},{"key":"ref_57","doi-asserted-by":"crossref","unstructured":"Li, B., Li, R., Zhang, C., Bian, H., Wang, F., Xiao, J., Liu, S., Yi, W., Zhang, M., and Wang, S. (2014). MicroRNA-7a\/b Protects against Cardiac Myocyte Injury in Ischemia\/Reperfusion by Targeting Poly(ADP-Ribose) Polymerase. PLoS ONE, 9.","DOI":"10.1371\/journal.pone.0090096"},{"key":"ref_58","doi-asserted-by":"crossref","unstructured":"Geng, H.-H., Li, R., Su, Y.-M., Xiao, J., Pan, M., Cai, X.-X., and Ji, X.-P. (2016). The Circular RNA Cdr1as Promotes Myocardial Infarction by Mediating the Regulation of miR-7a on Its Target Genes Expression. PLoS ONE, 11.","DOI":"10.1371\/journal.pone.0151753"},{"key":"ref_59","doi-asserted-by":"crossref","unstructured":"Picchio, L., Legagneux, V., Deschamps, S., Renaud, Y., Chauveau, S., Paillard, L., and Jagla, K. (2018). Bruno-3 regulates sarcomere component expression and contributes to muscle phenotypes of myotonic dystrophy type 1. Dis. Model. Mech., 11.","DOI":"10.1242\/dmm.031849"},{"key":"ref_60","doi-asserted-by":"crossref","unstructured":"de Haro, M., Al-Ramahi, I., Jones, K.R., Holth, J.K., Timchenko, L.T., and Botas, J. (2013). Smaug\/SAMD4A Restores Translational Activity of CUGBP1 and Suppresses CUG-Induced Myopathy. PLoS Genet., 9.","DOI":"10.1371\/journal.pgen.1003445"},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"679","DOI":"10.1242\/dmm.018127","article-title":"Increased autophagy and apoptosis contribute to muscle atrophy in a myotonic dystrophy type 1 Drosophila model","volume":"8","author":"Bargiela","year":"2015","journal-title":"Dis. Model. Mech."},{"key":"ref_62","doi-asserted-by":"crossref","first-page":"2843","DOI":"10.1038\/s41598-017-02829-3","article-title":"Expanded CCUG repeat RNA expression in Drosophila heart and muscle trigger Myotonic Dystrophy type 1-like phenotypes and activate autophagocytosis genes","volume":"7","author":"Chakraborty","year":"2017","journal-title":"Sci. Rep."},{"key":"ref_63","doi-asserted-by":"crossref","unstructured":"Cerro-Herreros, E., Fernandez-Costa, J.M., Sabater-Arcis, M., Llamusi, B., and Artero, R. (2016). Derepressing muscleblind expression by miRNA sponges ameliorates myotonic dystrophy-like phenotypes in Drosophila. Sci. Rep., 6.","DOI":"10.1038\/srep36230"},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"5792","DOI":"10.1016\/j.bmcl.2016.10.037","article-title":"Development of pharmacophore models for small molecules targeting RNA: Application to the RNA repeat expansion in myotonic dystrophy type 1","volume":"26","author":"Angelbello","year":"2016","journal-title":"Bioorg. Med. Chem. Lett."},{"key":"ref_65","doi-asserted-by":"crossref","first-page":"349","DOI":"10.3389\/fneur.2018.00349","article-title":"Small Molecules Which Improve Pathogenesis of Myotonic Dystrophy Type 1","volume":"9","author":"Brook","year":"2018","journal-title":"Front. Neurol."},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"11866","DOI":"10.1073\/pnas.1018213108","article-title":"In vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy models","volume":"108","author":"Artero","year":"2011","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_67","doi-asserted-by":"crossref","first-page":"6355","DOI":"10.1021\/ja5012146","article-title":"Targeting toxic RNAs that cause myotonic dystrophy type 1 (DM1) with a bisamidinium inhibitor","volume":"136","author":"Wong","year":"2014","journal-title":"J. Am. Chem. Soc."},{"key":"ref_68","doi-asserted-by":"crossref","first-page":"1428","DOI":"10.1002\/cmdc.201600081","article-title":"A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in a Drosophila Model of Myotonic Dystrophy","volume":"11","author":"Luu","year":"2016","journal-title":"ChemMedChem"}],"container-title":["International Journal of Molecular Sciences"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1422-0067\/19\/12\/4104\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,11]],"date-time":"2025-10-11T15:34:42Z","timestamp":1760196882000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1422-0067\/19\/12\/4104"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,12,18]]},"references-count":68,"journal-issue":{"issue":"12","published-online":{"date-parts":[[2018,12]]}},"alternative-id":["ijms19124104"],"URL":"https:\/\/doi.org\/10.3390\/ijms19124104","relation":{},"ISSN":["1422-0067"],"issn-type":[{"value":"1422-0067","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,12,18]]}}}