{"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\/it\/researcher-interview\/2018\/05\/17\/lgmd-researcher-kathryn-wagner\/","title":{"rendered":"RICERCATORE LGMD: Kathryn Wagner, MD, PhD"},"content":{"rendered":"

LGMD \"Riflettori sulla ricerca<\/strong><\/span><\/h2>\n

 <\/p>\n

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

Affiliazione<\/strong>: Istituto Kennedy Krieger<\/p>\n

Ruolo o posizione<\/strong>: Professore e direttore del Centro per i disturbi muscolari genetici<\/p>\n

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

Che tipo di istruzione e formazione ha seguito per arrivare alla sua posizione attuale?<\/strong><\/p>\n

Dopo aver completato il programma MD\/PhD, ho fatto una specializzazione in Neurologia seguita da borse di studio in Neuromuscolare e Neurogenetica. Ho anche svolto una borsa di studio post-dottorato in Biologia Molecolare.<\/p>\n

Cosa l'ha spinta a intraprendere una carriera nella ricerca e nello studio della distrofia muscolare in particolare?<\/strong><\/p>\n

Mio padre \u00e8 un importante scienziato e ho sempre pensato che avrei intrapreso una carriera scientifica. \u00c8 stato piuttosto tardi, durante l'universit\u00e0, che ho deciso che avrei voluto dedicarmi anche alla medicina. Mi sono interessata alla distrofia muscolare quando, durante la scuola di specializzazione, ho clonato (per caso) un gene, la distrobrevina, che ha alcune somiglianze con la distrofina, il gene mancante nella distrofia muscolare di Duchenne (DMD). Ho imparato tutto quello che potevo sulla distrofia muscolare e sono rimasto affascinato dalle scoperte di allora sulle molecole associate alla distrofina e implicate in altre distrofie muscolari.<\/p>\n

Quali argomenti state studiando?<\/strong><\/p>\n

Dirigo un laboratorio traslazionale incentrato sullo sviluppo di nuove terapie per la distrofia muscolare. Attualmente stiamo studiando modelli di DMD, distrofia muscolare facioscapolo-omerale e LGMD. In clinica, sto studiando gli effetti di un farmaco anti-miostatina prodotto da Pfizer nella LGMD2I.<\/p>\n

In che modo il suo lavoro aiuter\u00e0 i pazienti? \u00c8 di natura pi\u00f9 scientifica o potrebbe diventare un trattamento per le LGMD o per i medici in generale?<\/strong><\/p>\n

La nostra ricerca di laboratorio sull'AAV e sulla terapia cellulare ha il potenziale per diventare un trattamento per la LGMD e le MD in generale. Se la sperimentazione clinica del farmaco anti-miostatina di Pfizer avr\u00e0 successo, il percorso verso il trattamento sar\u00e0 il pi\u00f9 breve.<\/p>\n

Cosa vorrebbe che i pazienti e gli altri interessati alla LGMD sapessero sulla ricerca (sui vostri progetti e sul campo in generale)?<\/strong><\/p>\n

La terapia genica ha compiuto enormi progressi negli ultimi anni. \u00c8 realistico ipotizzare che nel prossimo futuro saranno condotti studi di terapia genica sistemica nella LGMD. Nel mio lavoro, stiamo sviluppando un nuovo AAV che si concentri meglio sul muscolo e non sul fegato.<\/p>\n

Cosa la ispira a continuare a lavorare in questo campo? <\/strong><\/p>\n

La popolazione di pazienti \u00e8 irresistibile. Si sforzano di condurre una vita piena e gratificante nonostante le loro sfide fisiche.<\/p>\n

In che modo i pazienti possono incoraggiarvi e aiutare il vostro lavoro?<\/strong><\/p>\n

L'incoraggiamento verbale \u00e8 meraviglioso, cos\u00ec come il sostegno finanziario alla nostra ricerca.<\/p>\n

 <\/p>\n

* * * Vi invitiamo a mettere MI PIACE, COMMENTARE e CONDIVIDERE questo post per contribuire a sensibilizzare l'opinione pubblica sulla LGMD!<\/p>\n

* * * Per saperne di pi\u00f9 sulla distrofia muscolare della cintura degli arti (LGMD) o per leggere altre \"Interviste Spotlight\", visitate il nostro sito web all'indirizzo 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\/it\/researcher-interview\/2018\/05\/17\/lgmd-researcher-kathryn-wagner\/\" \/>\n<meta property=\"og:locale\" content=\"it_IT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"LGMD RESEARCHER: Kathryn Wagner, MD, PhD - 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