{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,29]],"date-time":"2025-11-29T07:39:14Z","timestamp":1764401954897,"version":"build-2065373602"},"reference-count":73,"publisher":"MDPI AG","issue":"12","license":[{"start":{"date-parts":[[2022,12,12]],"date-time":"2022-12-12T00:00:00Z","timestamp":1670803200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Hospital de Cl\u00ednicas de Porto Alegre","award":["2022-0216","403587\/2021-2","312714\/2018-1"],"award-info":[{"award-number":["2022-0216","403587\/2021-2","312714\/2018-1"]}]},{"name":"FIPE","award":["2022-0216","403587\/2021-2","312714\/2018-1"],"award-info":[{"award-number":["2022-0216","403587\/2021-2","312714\/2018-1"]}]},{"name":"Conselho Nacional de Desenvolvimento Cient\u00edfico e Tecnol\u00f3gico\u2014CNPq","award":["2022-0216","403587\/2021-2","312714\/2018-1"],"award-info":[{"award-number":["2022-0216","403587\/2021-2","312714\/2018-1"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Life"],"abstract":"<jats:p>Mucopolysaccharidoses (MPS) are genetic metabolic diseases characterized by defects in the activity of lysosomal hydrolases. In MPS, secondary cell disturbance affects pathways related to cardiovascular disorders. Hence, the study aimed to identify MPS-related drugs targeting cardiovascular disease and select a list of drugs for repositioning. We obtained a list of differentially expressed genes and pathways. To identify drug perturbation-driven gene expression and drug pathways interactions, we used the CMAP and LINCS databases. For molecular docking, we used the DockThor web server. Our results suggest that pirfenidone and colchicine are promising drugs to treat cardiovascular disease in MPS patients. We also provide a brief description of good practices for the repositioning analysis. Furthermore, the list of drugs and related MPS-enriched genes could be helpful to new treatments and considered for pathophysiological studies.<\/jats:p>","DOI":"10.3390\/life12122085","type":"journal-article","created":{"date-parts":[[2022,12,13]],"date-time":"2022-12-13T01:56:06Z","timestamp":1670896566000},"page":"2085","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Drug Repositioning Applied to Cardiovascular Disease in Mucopolysaccharidosis"],"prefix":"10.3390","volume":"12","author":[{"given":"Gerda Cristal","family":"Villalba Silva","sequence":"first","affiliation":[{"name":"Bioinformatics Core, Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, Porto Alegre 2350, RS, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Thiago","family":"Steindorff","sequence":"additional","affiliation":[{"name":"Bioinformatics Core, Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, Porto Alegre 2350, RS, Brazil"},{"name":"Gene Therapy Center, Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, Porto Alegre 2350, RS, Brazil"},{"name":"Biomedical Sciences School, Institute of Health Sciences, UFRGS, Ramiro Barcelos, Porto Alegre 2600, RS, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4189-0436","authenticated-orcid":false,"given":"Roselena","family":"Silvestri Schuh","sequence":"additional","affiliation":[{"name":"Gene Therapy Center, Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, Porto Alegre 2350, RS, Brazil"},{"name":"Pharmaceutical Sciences Graduate Program, UFRGS, Avenida Ipiranga, Porto Alegre 2752, RS, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Natalia","family":"Cardoso Flores","sequence":"additional","affiliation":[{"name":"Bioinformatics Core, Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, Porto Alegre 2350, RS, Brazil"},{"name":"Gene Therapy Center, Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, Porto Alegre 2350, RS, Brazil"},{"name":"Biomedical Sciences School, Institute of Health Sciences, UFRGS, Ramiro Barcelos, Porto Alegre 2600, RS, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4977-6662","authenticated-orcid":false,"given":"Ursula","family":"Matte","sequence":"additional","affiliation":[{"name":"Bioinformatics Core, Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, Porto Alegre 2350, RS, Brazil"},{"name":"Gene Therapy Center, Hospital de Clinicas de Porto Alegre, Ramiro Barcelos, Porto Alegre 2350, RS, Brazil"},{"name":"Genetics and Molecular Biology Graduate Program, UFRGS, Av. Bento Gon\u00e7alves, Porto Alegre 9500, RS, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2022,12,12]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"798262","DOI":"10.3389\/fcell.2021.798262","article-title":"A Compendium of Information on the Lysosome","volume":"9","author":"Bouhamdani","year":"2021","journal-title":"Front. Cell Dev. Biol."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1111\/jnc.14632","article-title":"The role of innate immunity in mucopolysaccharide diseases","volume":"148","author":"Parker","year":"2019","journal-title":"J. Neurochem."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.1007\/s10545-011-9359-8","article-title":"Cardiac disease in patients with mucopolysaccharidosis: Presentation, diagnosis and management","volume":"34","author":"Braunlin","year":"2011","journal-title":"J. Inherit. Metab. 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