{"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\/tr\/researcher-interview\/2018\/06\/19\/lgmd-researcher-chamberlain\/","title":{"rendered":"LGMD ARA\u015eTIRMACISI: Jeffrey S. Chamberlain, Ph.D."},"content":{"rendered":"<p><strong>\"LGMD ARA\u015eTIRMAYA I\u015eIK TUTUYOR\"<\/strong><\/p>\n<p><strong>LGMD Ara\u015ft\u0131rmac\u0131s\u0131:<\/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>Ba\u011fl\u0131l\u0131k:<\/strong>\u00a0 Washington \u00dcniversitesi T\u0131p Fak\u00fcltesi<\/p>\n<p><strong>Rol veya Pozisyon<\/strong>:  Profes\u00f6r, McCaw Musk\u00fcler Distrofi K\u00fcrs\u00fcs\u00fc Ba\u015fkan\u0131, Wellstone Musk\u00fcler Distrofi Ara\u015ft\u0131rma Merkezi-Seattle Direkt\u00f6r\u00fc<\/p>\n<p><strong>\u015eu anki pozisyonunuza gelmek i\u00e7in hangi e\u011fitim ve \u00f6\u011fretimi ald\u0131n\u0131z?<\/strong><\/p>\n<p>Biyokimya alan\u0131nda lisans ve doktora derecesi ald\u0131m, ard\u0131ndan Molek\u00fcler Genetik alan\u0131nda doktora sonras\u0131 e\u011fitim ald\u0131m.<\/p>\n<p><strong>Sizi ara\u015ft\u0131rma alan\u0131nda kariyer yapmaya ve \u00f6zellikle kas distrofisi \u00fczerine \u00e7al\u0131\u015fmaya y\u00f6nlendiren neydi?<\/strong><\/p>\n<p>Doktoram\u0131 yaparken kas geli\u015fimine ilgi duymaya ve kas hastal\u0131klar\u0131 hakk\u0131nda bilgi edinmeye ba\u015flad\u0131m.  Doktora sonras\u0131 e\u011fitimimi Duchenne kas distrofisi \u00fczerine yapt\u0131m ve gen terapisi kavram\u0131na ilgi duymaya ba\u015flad\u0131m.  Kendi laboratuvar\u0131m\u0131 kurdu\u011fumda, kas distrofileri i\u00e7in gen tedavisinin yan\u0131 s\u0131ra farkl\u0131 distrofilerde kusurlu proteinlerin i\u015flevini incelemeye odaklanmaya karar verdim.<\/p>\n<p><strong>Hangi konular \u00fczerinde \u00e7al\u0131\u015f\u0131yorsunuz?<\/strong><\/p>\n<p>\u015eu anda, genleri kas dokular\u0131na iletmek i\u00e7in vekt\u00f6rler, genleri farkl\u0131 dokularda a\u00e7\u0131p kapatmak i\u00e7in gen d\u00fczenleyici kasetler ve distrofilerde ve gen terapisi ba\u011flam\u0131nda ba\u011f\u0131\u015f\u0131kl\u0131k sisteminin rol\u00fc dahil olmak \u00fczere kas bozukluklar\u0131n\u0131n gen tedavisi i\u00e7in y\u00f6ntemler \u00fczerinde \u00e7al\u0131\u015f\u0131yorum.  Ayr\u0131ca distrofin ve FKRP gibi kas i\u015flevine ve hastal\u0131\u011f\u0131na katk\u0131da bulunan farkl\u0131 proteinlerin i\u015flevini ve etkile\u015fimlerini de inceliyorum.<\/p>\n<p><strong>\u00c7al\u0131\u015fman\u0131z hastalara nas\u0131l yard\u0131mc\u0131 olacak? Do\u011fas\u0131 gere\u011fi daha bilimsel mi yoksa LGMD'ler veya genel olarak MD'ler i\u00e7in bir tedavi olabilir mi?<\/strong><\/p>\n<p>\u00c7al\u0131\u015fmalar\u0131m\u0131z hem bilimsel hem de tedavi odakl\u0131d\u0131r.  Etkili tedaviler geli\u015ftirmenin en iyi yolunun, \u00e7e\u015fitli kas bozukluklar\u0131n\u0131n arkas\u0131ndaki temel biyolojiyi ayr\u0131nt\u0131l\u0131 bir \u015fekilde anlamak oldu\u011funa inan\u0131yoruz.  Bu temel ara\u015ft\u0131rmay\u0131, g\u00fcvenli ve etkili gen tedavisi i\u00e7in gereken genlerin ve da\u011f\u0131t\u0131m ara\u00e7lar\u0131n\u0131n geli\u015ftirilmesine rehberlik etmek i\u00e7in kullan\u0131yoruz.<\/p>\n<p><strong>Hastalar\u0131n ve LGMD ile ilgilenen di\u011fer ki\u015filerin ara\u015ft\u0131rma hakk\u0131nda (kendi projeleriniz ve genel olarak alan hakk\u0131nda) ne bilmelerini istersiniz?<\/strong><\/p>\n<p>Gen terapisi f\u00fct\u00fcristik bir kavram olmaktan \u00e7\u0131k\u0131p kal\u0131tsal hastal\u0131klar\u0131n tedavisinde umut verici bir yakla\u015f\u0131m haline gelmi\u015ftir.  Bir\u00e7ok gen terapisi art\u0131k ticari kullan\u0131m i\u00e7in onaylanm\u0131\u015ft\u0131r ve doktorlar taraf\u0131ndan re\u00e7ete edilebilir.  Gen terapilerinin, farkl\u0131 kas distrofisi t\u00fcrlerinin \u00e7o\u011funun tedavisinde en umut verici yakla\u015f\u0131m oldu\u011funa inan\u0131yorum.  Bir\u00e7ok distrofide gen terapisinin geli\u015ftirilmesini s\u0131n\u0131rlayan zorluklar\u0131n \u00e7e\u015fitli bozukluklar i\u00e7in ayn\u0131 oldu\u011funu ve birindeki ilerlemenin t\u00fcm distrofiler i\u00e7in tedavileri klini\u011fe yakla\u015ft\u0131rmaya yard\u0131mc\u0131 oldu\u011funu anlamak da \u00f6nemlidir.  Bir\u00e7ok kas distrofisi i\u00e7in gen terapisi denemeleri devam etmektedir ve daha bir\u00e7oklar\u0131 da test edilmeye yak\u0131nd\u0131r.  Yeni genetik tedavilerin geli\u015ftirilmesi ve uygulanmas\u0131nda bir devrimin e\u015fi\u011findeyiz ve bu yeni teknolojilerin nihayet hastalara ve ailelerine yard\u0131mc\u0131 olmaya ba\u015flad\u0131\u011f\u0131n\u0131 g\u00f6rmek i\u00e7in harika bir zaman oldu\u011funu d\u00fc\u015f\u00fcn\u00fcyorum.<\/p>\n<p><strong>Bu alanda \u00e7al\u0131\u015fmaya devam etmek i\u00e7in size ilham veren nedir? <\/strong><\/p>\n<p>Kas distrofisi \u00fczerine 30 y\u0131ld\u0131r \u00e7al\u0131\u015f\u0131yorum ve nihayet yeni tedavilerin klinikte uyguland\u0131\u011f\u0131na ve i\u015fe yarad\u0131\u011f\u0131na tan\u0131k oluyorum.  Kariyerimin en heyecan verici d\u00f6nemini ya\u015f\u0131yorum ve farkl\u0131 kas hastal\u0131klar\u0131n\u0131n tedavisine y\u00f6nelik yeni yakla\u015f\u0131mlar\u0131n geli\u015ftirilmesine katk\u0131da bulunabilece\u011fim i\u00e7in \u00e7ok heyecanl\u0131y\u0131m.<\/p>\n<p><strong>Hastalar sizi nas\u0131l te\u015fvik edebilir ve \u00e7al\u0131\u015fmalar\u0131n\u0131za nas\u0131l yard\u0131mc\u0131 olabilir?<\/strong><\/p>\n<p>Bilimsel ara\u015ft\u0131rmalara ayr\u0131lan fonlar \u00fclke \u00e7ap\u0131nda azal\u0131yor ve bir\u00e7ok \u00e7evrede bilimsel fikirlerin ve ilkelerin \u00f6\u011fretilmesine ve ara\u015ft\u0131r\u0131lmas\u0131na kar\u015f\u0131 neredeyse bir sava\u015f varm\u0131\u015f gibi g\u00f6r\u00fcn\u00fcyor.  Hastalar\u0131n ve aile \u00fcyelerinin yapabilece\u011fi en \u00f6nemli \u015fey, yerel ve ulusal h\u00fck\u00fcmet \u00fcyelerini \u00fclke \u00e7ap\u0131ndaki \u00fcniversitelerde bilimsel ara\u015ft\u0131rmalar\u0131 desteklemeye te\u015fvik etmektir.  Gen ve h\u00fccre tedavileri gibi yenilik\u00e7i ve yeni tedavi yakla\u015f\u0131mlar\u0131n\u0131n neredeyse tamam\u0131, biyoteknoloji ve b\u00fcy\u00fck ila\u00e7 firmalar\u0131 taraf\u0131ndan benimsenmeden \u00f6nce sadece \u00fcniversitelerde geli\u015ftirilmektedir.<\/p>\n<p>&nbsp;<\/p>\n<p>* * * LGMD konusunda fark\u0131ndal\u0131k yaratmaya yard\u0131mc\u0131 olmak i\u00e7in l\u00fctfen bu g\u00f6nderiyi BE\u011eEN\u0130N, YORUM YAPIN ve PAYLA\u015eIN!<\/p>\n<p>* Limb Girdle Muscular Dystrophy (LGMD) hakk\u0131nda daha fazla bilgi edinmek veya daha fazla \"Spotlight Interviews\" okumak i\u00e7in l\u00fctfen web sitemizi ziyaret edin <a href=\"https:\/\/www.lgmd-info.org\/tr\/\">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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