{"id":1938,"date":"2018-05-17T10:30:28","date_gmt":"2018-05-17T15:30:28","guid":{"rendered":"https:\/\/restruct-lgmd-2021.pantheonsite.io\/?p=1938"},"modified":"2018-05-17T10:30:28","modified_gmt":"2018-05-17T15:30:28","slug":"lgmd-researcher-kathryn-wagner","status":"publish","type":"post","link":"https:\/\/www.lgmd-info.org\/tr\/researcher-interview\/2018\/05\/17\/lgmd-researcher-kathryn-wagner\/","title":{"rendered":"LGMD ARA\u015eTIRMACISI: Kathryn Wagner, MD, PhD"},"content":{"rendered":"

LGMD \"Ara\u015ft\u0131rmada G\u00fcndem\"<\/strong><\/span><\/h2>\n

 <\/p>\n

LGMD ARA\u015eTIRMACISI<\/strong>: Kathryn Wagner, MD, PhD\"\"<\/a><\/p>\n

\u00dcyelik<\/strong>: Kennedy Krieger Enstit\u00fcs\u00fc<\/p>\n

Rol veya Pozisyon<\/strong>: Profes\u00f6r ve Genetik Kas Bozukluklar\u0131 Merkezi Direkt\u00f6r\u00fc<\/p>\n

\u00a0<\/strong><\/p>\n

\u015eu anki pozisyonunuza gelmek i\u00e7in hangi e\u011fitim ve \u00f6\u011fretimi ald\u0131n\u0131z?<\/strong><\/p>\n

MD\/Doktora program\u0131n\u0131 tamamlad\u0131ktan sonra N\u00f6roloji ihtisas\u0131 yapt\u0131m ve ard\u0131ndan N\u00f6romusk\u00fcler ve N\u00f6rogenetik alanlar\u0131nda burslar ald\u0131m. Ayr\u0131ca Molek\u00fcler Biyoloji alan\u0131nda doktora sonras\u0131 burs yapt\u0131m.<\/p>\n

Sizi ara\u015ft\u0131rma alan\u0131nda kariyer yapmaya ve \u00f6zellikle kas distrofisi \u00fczerine \u00e7al\u0131\u015fmaya y\u00f6nlendiren neydi?<\/strong><\/p>\n

Babam tan\u0131nm\u0131\u015f bir bilim insan\u0131 ve ben de her zaman bilimle ilgilenece\u011fimi d\u00fc\u015f\u00fcnm\u00fc\u015ft\u00fcm. T\u0131p alan\u0131nda da ilerlemek istedi\u011fime \u00fcniversitede olduk\u00e7a ge\u00e7 karar verdim. Kas distrofisi ile ilgilenmeye ba\u015flamam, y\u00fcksek lisans okulundayken (\u015fans eseri) Duchenne kas distrofisinde (DMD) eksik olan distrofin geniyle baz\u0131 benzerlikleri olan distrobrevin adl\u0131 bir geni klonlamamla oldu. Kas distrofisi hakk\u0131nda \u00f6\u011frenebildi\u011fim her \u015feyi \u00f6\u011frendim ve o d\u00f6nemde di\u011fer kas distrofilerinde rol oynayan distrofinle ili\u015fkili molek\u00fcllerin ke\u015ffi beni b\u00fcy\u00fcledi.<\/p>\n

Hangi konular \u00fczerinde \u00e7al\u0131\u015f\u0131yorsunuz?<\/strong><\/p>\n

Kas distrofisi i\u00e7in yeni terap\u00f6tikler geli\u015ftirmeye odaklanan translasyonel bir laboratuvar y\u00f6netiyorum. \u015eu anda DMD, facioscapulohumeral musk\u00fcler distrofi ve LGMD modelleri \u00fczerinde \u00e7al\u0131\u015f\u0131yoruz. Klinikte, LGMD2I'de Pfizer taraf\u0131ndan \u00fcretilen bir anti-myostatin ilac\u0131n\u0131n etkilerini inceliyorum.<\/p>\n

\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

AAV ve h\u00fccre terapisi \u00fczerine laboratuvar ara\u015ft\u0131rmalar\u0131m\u0131z LGMD ve genel olarak MD'ler i\u00e7in tedavi olma potansiyeline sahiptir. Pfizer'in anti-myostatin ilac\u0131n\u0131n klinik denemesi ba\u015far\u0131l\u0131 olursa, bu tedaviye giden en k\u0131sa yola sahip olacakt\u0131r.<\/p>\n

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

Gen terapisi son birka\u00e7 y\u0131lda muazzam bir ilerleme kaydetmi\u015ftir. Yak\u0131n gelecekte LGMD'de sistemik gen terapisi denemeleri yapaca\u011f\u0131m\u0131z\u0131 varsaymak ger\u00e7ek\u00e7i olacakt\u0131r. Kendi \u00e7al\u0131\u015fmamda, karaci\u011fere de\u011fil de kaslara daha iyi yerle\u015fmesi i\u00e7in yeni AAV geli\u015ftiriyoruz.<\/p>\n

Bu alanda \u00e7al\u0131\u015fmaya devam etmek i\u00e7in size ilham veren nedir? <\/strong><\/p>\n

Hasta pop\u00fclasyonu kar\u015f\u0131 konulmaz. Fiziksel zorluklar\u0131na ra\u011fmen \u00f6d\u00fcllendirici bir ya\u015fam s\u00fcrmek i\u00e7in \u00e7abal\u0131yorlar.<\/p>\n

Hastalar sizi nas\u0131l te\u015fvik edebilir ve \u00e7al\u0131\u015fmalar\u0131n\u0131za nas\u0131l yard\u0131mc\u0131 olabilir?<\/strong><\/p>\n

Ara\u015ft\u0131rmalar\u0131m\u0131z i\u00e7in maddi destek kadar s\u00f6zl\u00fc te\u015fvik de harika.<\/p>\n

 <\/p>\n

* * * 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

* 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 https:\/\/lgmd-info.org<\/a>\u00a0 <\/u><\/p>","protected":false},"excerpt":{"rendered":"

LGMD “Spotlight on Research”   LGMD RESEARCHER: Kathryn Wagner, MD, […]<\/p>","protected":false},"author":1,"featured_media":1941,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[91,92],"tags":[],"class_list":["post-1938","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-researcher-interview","category-researcher"],"acf":[],"yoast_head":"\nLGMD RESEARCHER: Kathryn Wagner, MD, PhD - 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\/tr\/researcher-interview\/2018\/05\/17\/lgmd-researcher-kathryn-wagner\/\" \/>\n<meta property=\"og:locale\" content=\"tr_TR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"LGMD RESEARCHER: Kathryn Wagner, MD, PhD - 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