{"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\/it\/researcher-interview\/2018\/03\/04\/1737\/","title":{"rendered":"RICERCATORE LGMD: Louise Rodino-Klapac, Ph.D."},"content":{"rendered":"

LGMD \"Intervista a riflettori accesi sulla ricerca <\/strong><\/span><\/h2>\n

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

Affiliazione:\u00a0 <\/strong>Nationwide Children's Hospital e Myonexus Therapeutics, Inc.<\/p>\n

Ruolo o posizione<\/strong>: Professore associato (NCH) e Direttore scientifico (Myonexus)<\/p>\n

 <\/p>\n

 <\/p>\n

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

Ho conseguito un dottorato di ricerca in genetica molecolare studiando lo sviluppo neuromuscolare. Ho poi svolto una borsa di studio post-dottorato in terapia genica presso l'ospedale pediatrico nazionale con Jerry Mendell come mentore.<\/p>\n

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

Sapevo di voler diventare un ricercatore fin da giovane. Ero affascinata dalla genetica e da come i geni difettosi potessero causare malattie. Ho studiato lo sviluppo neuromuscolare durante il mio corso di laurea ed ero interessata a sfruttare queste conoscenze per sviluppare trattamenti per le malattie muscolari. Fortunatamente, la ricerca sulla distrofia muscolare \u00e8 un'area di rilievo al Nationwide Children's che mi offre l'opportunit\u00e0 di realizzare i miei obiettivi.<\/p>\n

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

Sviluppo trattamenti di terapia genica per la distrofia muscolare del cinto arto e la distrofia muscolare di Duchenne e traduco questi trattamenti in sperimentazioni cliniche. Il mio laboratorio studia anche i meccanismi patologici delle distrofie muscolari della cintura degli arti.<\/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

Il nostro lavoro \u00e8 direttamente applicabile ai pazienti. Abbiamo in corso o imminenti studi clinici per diverse forme di LGMD (2B, 2D, 2E) e stiamo pianificando studi clinici per altri sottotipi. Siamo molto ottimisti sul fatto che la sostituzione genica sar\u00e0 un trattamento significativo per le MD.<\/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

\u00c8 un momento molto emozionante per la ricerca sulla LGMD. I miglioramenti nei test genetici e i successi nella terapia genica di altri disturbi neuromuscolari hanno accelerato la ricerca e gli studi clinici per la LGMD. Possiamo applicare rapidamente ci\u00f2 che impariamo studiando una forma di LGMD a un'altra.<\/p>\n

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

I pazienti sono la mia pi\u00f9 grande ispirazione. \u00c8 cos\u00ec gratificante sapere che tutto il duro lavoro che stiamo facendo per portare le terapie in ambito clinico porter\u00e0, si spera, benefici ai pazienti in modo molto significativo!<\/p>\n

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

Ascoltare le storie dei pazienti (storie di speranza e di sfide) \u00e8 estremamente utile. Aiuta noi ricercatori a conoscere le sfide uniche di ogni specifica malattia per adattare i trattamenti, se possibile. Inoltre, \u00e8 una fonte costante di motivazione per continuare a lavorare sodo sapendo che c'\u00e8 un enorme team di sostenitori dietro di voi!<\/p>","protected":false},"excerpt":{"rendered":"

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