{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2022,5,29]],"date-time":"2022-05-29T19:11:16Z","timestamp":1653851476848},"reference-count":0,"publisher":"Polskie Towarzystwo Biochemiczne (Polish Biochemical Society)","license":[{"start":{"date-parts":[[2022,5,24]],"date-time":"2022-05-24T00:00:00Z","timestamp":1653350400000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-sa\/4.0"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Postepy Biochem"],"abstract":"<jats:p>Dystrofia mi\u0119\u015bniowa Duchenne\u2019a (DMD) to choroba genetyczna sprz\u0119\u017cona z chromosomem X, dotykaj\u0105ca w przybli\u017ceniu 1 na 5000 urodzonych ch\u0142opc\u00f3w. Jest spowodowana mutacjami w genie DMD koduj\u0105cym dystrofin\u0119, kt\u00f3ra odpowiada za stabilno\u015b\u0107 mechaniczn\u0105 mi\u0119\u015bni podczas skurczu. Jej brak prowadzi do post\u0119puj\u0105cego os\u0142abienia mi\u0119\u015bni i przedwczesnej \u015bmierci chorych w wyniku niewydolno\u015bci sercowo-oddechowej. W ostatnich latach opracowano wiele eksperymentalnych terapii, kt\u00f3rych celem jest przywr\u00f3cenie funkcjonalnej dystrofiny lub przeciwdzia\u0142anie procesom przyczyniaj\u0105cym si\u0119 do post\u0119pu choroby, takim jak zapalenie czy zw\u0142\u00f3knienie. Pomimo tego DMD wci\u0105\u017c pozostaje chorob\u0105 nieuleczaln\u0105, a glikokortykoidy, wykazuj\u0105ce wiele dzia\u0142a\u0144 niepo\u017c\u0105danych, nadal stanowi\u0105 \u201ez\u0142oty standard\u201d leczenia. Aktualne jest zatem opracowywanie innowacyjnych mo\u017cliwo\u015bci terapeutycznych, kt\u00f3re przynajmniej z\u0142agodz\u0105 objawy DMD. W\u015br\u00f3d nich na uwag\u0119 zas\u0142uguje celowanie w okre\u015blone mikroRNA (miR), np. miR-378a, przywr\u00f3cenie prawid\u0142owej angiogenezy oraz wykorzystanie cytoprotekcyjnych czynnik\u00f3w takich jak oksygenaza hemowa-1 (HO-1) czy siarkowod\u00f3r (H2S). W niniejszej pracy om\u00f3wiono zar\u00f3wno patologi\u0119 choroby jak i wspomniane, nowe mo\u017cliwo\u015bci terapeutyczne w DMD.<\/jats:p>","DOI":"10.18388\/pb.2021_428","type":"journal-article","created":{"date-parts":[[2022,5,29]],"date-time":"2022-05-29T18:55:51Z","timestamp":1653850551000},"source":"Crossref","is-referenced-by-count":0,"title":["Molekularne mechanizmy dystrofii mi\u0119\u015bniowej Duchenne\u2019a  i nowe mo\u017cliwo\u015bci terapeutyczne"],"prefix":"10.18388","author":[{"given":"Paulina","family":"Podkalicka","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ma\u0142gorzata","family":"Myszka","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J\u00f3zef","family":"Dulak","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Agnieszka","family":"\u0141oboda","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"7609","published-online":{"date-parts":[[2022,5,24]]},"container-title":["Post\u0119py Biochemii"],"original-title":[],"link":[{"URL":"https:\/\/postepybiochemii.ptbioch.edu.pl\/index.php\/PB\/article\/download\/428\/1018","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/postepybiochemii.ptbioch.edu.pl\/index.php\/PB\/article\/download\/428\/1018","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,5,29]],"date-time":"2022-05-29T18:55:51Z","timestamp":1653850551000},"score":1,"resource":{"primary":{"URL":"https:\/\/postepybiochemii.ptbioch.edu.pl\/index.php\/PB\/article\/view\/428"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,5,24]]},"references-count":0,"URL":"https:\/\/doi.org\/10.18388\/pb.2021_428","relation":{},"ISSN":["0032-5422"],"issn-type":[{"value":"0032-5422","type":"print"}],"subject":[],"published":{"date-parts":[[2022,5,24]]}}}