{"id":1737,"date":"2018-03-04T18:32:55","date_gmt":"2018-03-05T00:32:55","guid":{"rendered":"https:\/\/restruct-lgmd-2021.pantheonsite.io\/?p=1737"},"modified":"2018-03-04T18:32:55","modified_gmt":"2018-03-05T00:32:55","slug":"1737","status":"publish","type":"post","link":"https:\/\/www.lgmd-info.org\/pl\/researcher-interview\/2018\/03\/04\/1737\/","title":{"rendered":"LGMD RESEARCHER: Louise Rodino-Klapac, Ph.D"},"content":{"rendered":"

LGMD \"Wywiad w centrum uwagi na temat bada\u0144\" <\/strong><\/span><\/h2>\n

Badacz LGMD:<\/strong> Louise Rodino-Klapac, Ph.D\"\"<\/a><\/p>\n

Przynale\u017cno\u015b\u0107:\u00a0 <\/strong>Nationwide Children's Hospital i Myonexus Therapeutics, Inc.<\/p>\n

Rola lub stanowisko<\/strong>: Profesor nadzwyczajny (NCH) i dyrektor naukowy (Myonexus)<\/p>\n

 <\/p>\n

 <\/p>\n

Jakie wykszta\u0142cenie i szkolenie zdoby\u0142e\u015b, aby osi\u0105gn\u0105\u0107 obecne stanowisko?<\/strong><\/p>\n

Mam doktorat z genetyki molekularnej badaj\u0105cej rozw\u00f3j nerwowo-mi\u0119\u015bniowy. Nast\u0119pnie odby\u0142em sta\u017c podoktorski w zakresie terapii genowej w krajowym szpitalu dzieci\u0119cym, a moim mentorem by\u0142 Jerry Mendell.<\/p>\n

Co sprawi\u0142o, \u017ce zdecydowa\u0142e\u015b si\u0119 na karier\u0119 naukow\u0105, a w szczeg\u00f3lno\u015bci na badanie dystrofii mi\u0119\u015bniowej?<\/strong><\/p>\n

Od najm\u0142odszych lat wiedzia\u0142em, \u017ce chc\u0119 zosta\u0107 naukowcem. Fascynowa\u0142a mnie genetyka i to, w jaki spos\u00f3b wadliwe geny mog\u0105 powodowa\u0107 choroby. Studiowa\u0142em rozw\u00f3j nerwowo-mi\u0119\u015bniowy na studiach magisterskich i by\u0142em zainteresowany wykorzystaniem tej wiedzy do opracowania metod leczenia chor\u00f3b mi\u0119\u015bni. Na szcz\u0119\u015bcie badania nad dystrofi\u0105 mi\u0119\u015bniow\u0105 s\u0105 obszarem, na kt\u00f3ry k\u0142adzie si\u0119 nacisk w Nationwide Children's, daj\u0105c mi mo\u017cliwo\u015b\u0107 realizacji moich cel\u00f3w.<\/p>\n

Jakie tematy studiujesz?<\/strong><\/p>\n

Zajmuj\u0119 si\u0119 opracowywaniem terapii genowych w leczeniu dystrofii mi\u0119\u015bniowej i dystrofii mi\u0119\u015bniowej Duchenne'a oraz wprowadzaniem tych terapii do bada\u0144 klinicznych. Moje laboratorium bada r\u00f3wnie\u017c mechanizmy chorobowe dystrofii mi\u0119\u015bniowej obr\u0119czy ko\u0144czyny.<\/p>\n

W jaki spos\u00f3b Twoja praca pomo\u017ce pacjentom? Czy ma ona bardziej naukowy charakter, czy te\u017c mo\u017ce sta\u0107 si\u0119 metod\u0105 leczenia LGMD lub og\u00f3lnie lekarzy?<\/strong><\/p>\n

Nasza praca ma bezpo\u015brednie zastosowanie u pacjent\u00f3w. Prowadzimy badania kliniczne dla kilku form LGMD (2B, 2D, 2E) i planujemy badania kliniczne dla innych podtyp\u00f3w. Jeste\u015bmy bardzo optymistyczni, \u017ce zast\u0105pienie gen\u00f3w b\u0119dzie znacz\u0105cym sposobem leczenia chor\u00f3b uk\u0142adu mi\u0119\u015bniowo-szkieletowego.<\/p>\n

Co chcia\u0142by\u015b, aby pacjenci i inne osoby zainteresowane LGMD wiedzia\u0142y o badaniach (Twoich w\u0142asnych projektach i og\u00f3lnie o tej dziedzinie)?<\/strong><\/p>\n

To bardzo ekscytuj\u0105cy czas dla bada\u0144 nad LGMD. Udoskonalenie test\u00f3w genetycznych i sukcesy w terapii genowej innych zaburze\u0144 nerwowo-mi\u0119\u015bniowych przyspieszy\u0142y badania i pr\u00f3by kliniczne nad LGMD. Mo\u017cemy szybko zastosowa\u0107 to, czego nauczyli\u015bmy si\u0119 z bada\u0144 nad jedn\u0105 form\u0105 LGMD, do innej.<\/p>\n

Co inspiruje Ci\u0119 do dalszej pracy w tej dziedzinie? <\/strong><\/p>\n

Pacjenci s\u0105 moj\u0105 najwi\u0119ksz\u0105 inspiracj\u0105. To bardzo satysfakcjonuj\u0105ce wiedzie\u0107, \u017ce ca\u0142a ci\u0119\u017cka praca, kt\u00f3r\u0105 wykonujemy, aby wprowadzi\u0107 leczenie do kliniki, przyniesie pacjentom korzy\u015bci w bardzo znacz\u0105cy spos\u00f3b!<\/p>\n

W jaki spos\u00f3b pacjenci mog\u0105 Ci\u0119 zach\u0119ci\u0107 i pom\u00f3c Ci w pracy?<\/strong><\/p>\n

Wys\u0142uchanie historii pacjent\u00f3w (zar\u00f3wno tych pe\u0142nych nadziei, jak i wyzwa\u0144) jest niezwykle pomocne. Pomaga nam jako badaczom pozna\u0107 wyzwania unikalne dla ka\u017cdej konkretnej choroby, aby w miar\u0119 mo\u017cliwo\u015bci dostosowa\u0107 leczenie. Co wi\u0119cej, jest to sta\u0142e \u017ar\u00f3d\u0142o motywacji do dalszej ci\u0119\u017ckiej pracy, wiedz\u0105c, \u017ce stoi za tob\u0105 ogromny zesp\u00f3\u0142 zwolennik\u00f3w!<\/p>","protected":false},"excerpt":{"rendered":"

LGMD “Spotlight Interview on Research\u201d LGMD Researcher: Louise Rodino-Klapac, Ph.D […]<\/p>","protected":false},"author":1,"featured_media":1735,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[91,92],"tags":[],"class_list":["post-1737","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-researcher-interview","category-researcher"],"acf":[],"yoast_head":"\nLGMD RESEARCHER: Louise Rodino-Klapac, Ph.D - LGMD Awareness Foundation<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"http:\/\/www.lgmd-info.org\/pl\/researcher-interview\/2018\/03\/04\/1737\/\" \/>\n<meta property=\"og:locale\" content=\"pl_PL\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"LGMD RESEARCHER: Louise Rodino-Klapac, Ph.D - 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