{"id":1988,"date":"2018-06-19T12:29:12","date_gmt":"2018-06-19T17:29:12","guid":{"rendered":"https:\/\/restruct-lgmd-2021.pantheonsite.io\/?p=1988"},"modified":"2018-06-19T12:29:12","modified_gmt":"2018-06-19T17:29:12","slug":"lgmd-researcher-chamberlain","status":"publish","type":"post","link":"https:\/\/www.lgmd-info.org\/pl\/researcher-interview\/2018\/06\/19\/lgmd-researcher-chamberlain\/","title":{"rendered":"BADACZ LGMD: Jeffrey S. Chamberlain, Ph.D."},"content":{"rendered":"<p><strong>\"LGMD SPOTLIGHT ON RESEARCH\"<\/strong><\/p>\n<p><strong>Badacz LGMD:<\/strong> Jeffrey S. Chamberlain, Ph.D.<a href=\"https:\/\/www.lgmd-info.org\/wp-content\/uploads\/2018\/06\/Chamberlain-Jeff.png\"><img decoding=\"async\" class=\"lazyload size-medium wp-image-1987 alignright\" src=\"data:image\/svg+xml,%3Csvg%20xmlns%3D%27http%3A%2F%2Fwww.w3.org%2F2000%2Fsvg%27%20width%3D%27300%27%20height%3D%27225%27%20viewBox%3D%270%200%20300%20225%27%3E%3Crect%20width%3D%27300%27%20height%3D%27225%27%20fill-opacity%3D%220%22%2F%3E%3C%2Fsvg%3E\" data-orig-src=\"https:\/\/www.lgmd-info.org\/wp-content\/uploads\/2018\/06\/Chamberlain-Jeff-300x225.png\" alt=\"\" width=\"300\" height=\"225\" \/><\/a><\/p>\n<p><strong>Przynale\u017cno\u015b\u0107:<\/strong>\u00a0 University of Washington School of Medicine<\/p>\n<p><strong>Rola lub stanowisko<\/strong>:  Profesor, Katedra Dystrofii Mi\u0119\u015bniowej McCaw, Dyrektor, Wellstone Muscular Dystrophy Research Center-Seattle<\/p>\n<p><strong>Jakie wykszta\u0142cenie i szkolenie zdoby\u0142e\u015b, aby osi\u0105gn\u0105\u0107 obecne stanowisko?<\/strong><\/p>\n<p>Uzyska\u0142em tytu\u0142 licencjata i doktora biochemii, a nast\u0119pnie odby\u0142em sta\u017c podoktorski z genetyki molekularnej<\/p>\n<p><strong>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<p>Podczas pracy nad doktoratem zainteresowa\u0142em si\u0119 rozwojem mi\u0119\u015bni i zacz\u0105\u0142em uczy\u0107 si\u0119 o chorobach mi\u0119\u015bni.  Odby\u0142em sta\u017c podoktorski badaj\u0105c dystrofi\u0119 mi\u0119\u015bniow\u0105 Duchenne'a i zainteresowa\u0142em si\u0119 koncepcj\u0105 terapii genowej.  Kiedy za\u0142o\u017cy\u0142em w\u0142asne laboratorium, postanowi\u0142em skupi\u0107 si\u0119 na terapii genowej dystrofii mi\u0119\u015bniowych, a tak\u017ce na badaniu funkcji bia\u0142ek wadliwych w r\u00f3\u017cnych dystrofiach.<\/p>\n<p><strong>Jakie tematy studiujesz?<\/strong><\/p>\n<p>Obecnie badam metody terapii genowej zaburze\u0144 mi\u0119\u015bniowych, w tym wektory do dostarczania gen\u00f3w do tkanek mi\u0119\u015bniowych, kasety regulacyjne gen\u00f3w do w\u0142\u0105czania i wy\u0142\u0105czania gen\u00f3w w r\u00f3\u017cnych tkankach oraz rol\u0119 uk\u0142adu odporno\u015bciowego w dystrofiach i w kontek\u015bcie terapii genowej.  Badam r\u00f3wnie\u017c funkcje i interakcje r\u00f3\u017cnych bia\u0142ek, kt\u00f3re przyczyniaj\u0105 si\u0119 do funkcjonowania mi\u0119\u015bni i chor\u00f3b, takich jak dystrofina i FKRP.<\/p>\n<p><strong>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<p>Nasza praca jest zar\u00f3wno naukowa, jak i ukierunkowana na leczenie.  Wierzymy, \u017ce najlepszym sposobem na opracowanie skutecznych metod leczenia jest szczeg\u00f3\u0142owe zrozumienie podstawowej biologii stoj\u0105cej za r\u00f3\u017cnymi zaburzeniami mi\u0119\u015bni.  Wykorzystujemy te podstawowe badania, aby kierowa\u0107 rozwojem gen\u00f3w i no\u015bnik\u00f3w potrzebnych do bezpiecznej i skutecznej terapii genowej.<\/p>\n<p><strong>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<p>Terapia genowa przesz\u0142a od futurystycznej koncepcji do obiecuj\u0105cego podej\u015bcia do leczenia zaburze\u0144 dziedzicznych.  Kilka terapii genowych zosta\u0142o ju\u017c zatwierdzonych do u\u017cytku komercyjnego i mo\u017ce by\u0107 przepisywanych przez lekarzy.  Uwa\u017cam, \u017ce terapie genowe s\u0105 najbardziej obiecuj\u0105cym podej\u015bciem do leczenia wi\u0119kszo\u015bci r\u00f3\u017cnych rodzaj\u00f3w dystrofii mi\u0119\u015bniowej.  Wa\u017cne jest r\u00f3wnie\u017c, aby zda\u0107 sobie spraw\u0119, \u017ce wyzwania ograniczaj\u0105ce rozw\u00f3j terapii genowej dla wielu dystrofii s\u0105 takie same dla r\u00f3\u017cnych zaburze\u0144, a post\u0119p w jednej z nich pomaga przybli\u017cy\u0107 leczenie wszystkich dystrofii.  Trwaj\u0105 pr\u00f3by terapii genowej dla kilku dystrofii mi\u0119\u015bniowych, a wiele innych jest bliskich przetestowania.  Jeste\u015bmy u progu rewolucji w rozwoju i stosowaniu nowych terapii genetycznych i my\u015bl\u0119, \u017ce to niesamowity czas, kiedy te nowe technologie wreszcie zaczynaj\u0105 pomaga\u0107 pacjentom i ich rodzinom.<\/p>\n<p><strong>Co inspiruje Ci\u0119 do dalszej pracy w tej dziedzinie? <\/strong><\/p>\n<p>Pracuj\u0119 nad dystrofi\u0105 mi\u0119\u015bniow\u0105 od 30 lat i w ko\u0144cu jestem \u015bwiadkiem, jak nowe terapie s\u0105 stosowane i dzia\u0142aj\u0105 w klinice.  To najbardziej ekscytuj\u0105cy czas w mojej karierze i ciesz\u0119 si\u0119, \u017ce mog\u0119 przyczyni\u0107 si\u0119 do rozwoju nowych metod leczenia r\u00f3\u017cnych rodzaj\u00f3w zaburze\u0144 mi\u0119\u015bniowych.<\/p>\n<p><strong>W jaki spos\u00f3b pacjenci mog\u0105 Ci\u0119 zach\u0119ci\u0107 i pom\u00f3c Ci w pracy?<\/strong><\/p>\n<p>Finansowanie bada\u0144 naukowych spada w ca\u0142ym kraju, a w wielu kr\u0119gach wydaje si\u0119, \u017ce toczy si\u0119 niemal wojna z nauczanymi i badanymi ideami i zasadami naukowymi.  Najwa\u017cniejsz\u0105 rzecz\u0105, jak\u0105 mog\u0105 zrobi\u0107 pacjenci i cz\u0142onkowie ich rodzin, jest zach\u0119canie cz\u0142onk\u00f3w w\u0142adz lokalnych i krajowych do wspierania bada\u0144 naukowych na uniwersytetach w ca\u0142ym kraju.  Prawie wszystkie innowacyjne i nowe podej\u015bcia do terapii, takie jak terapie genowe i kom\u00f3rkowe, s\u0105 opracowywane wy\u0142\u0105cznie na uniwersytetach, zanim zostan\u0105 ostatecznie przej\u0119te przez biotechnologi\u0119 i du\u017ce firmy farmaceutyczne.<\/p>\n<p>&nbsp;<\/p>\n<p>* * * Prosimy o LIKE, COMMENT i SHARE tego postu, aby pom\u00f3c zwi\u0119kszy\u0107 \u015bwiadomo\u015b\u0107 na temat LGMD!<\/p>\n<p>* * * Aby dowiedzie\u0107 si\u0119 wi\u0119cej o Dystrofii Mi\u0119\u015bniowej Ko\u0144czyn (LGMD) lub przeczyta\u0107 wi\u0119cej \"Wywiad\u00f3w w centrum uwagi\", odwied\u017a nasz\u0105 stron\u0119 internetow\u0105 pod adresem <a href=\"https:\/\/www.lgmd-info.org\/pl\/\">https:\/\/lgmd-info.org<\/a><\/p>\n<p>#CureLGMD<\/p>\n<p>#LgmdDay2018<\/p>","protected":false},"excerpt":{"rendered":"<p>&#8220;LGMD SPOTLIGHT ON RESEARCH&#8221; LGMD Researcher: Jeffrey S. Chamberlain, Ph.D. [&hellip;]<\/p>","protected":false},"author":1,"featured_media":1987,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[91,92],"tags":[],"class_list":["post-1988","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-researcher-interview","category-researcher"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v23.9 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>LGMD RESEARCHER: Jeffrey S. 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