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

LGMD \"Entrevista en el punto de mira de la investigaci\u00f3n\" <\/strong><\/span><\/h2>\n

Investigador del LGMD:<\/strong> Dra. Louise Rodino-Klapac\"\"<\/a><\/p>\n

Afiliaci\u00f3n:\u00a0 <\/strong>Nationwide Children's Hospital y Myonexus Therapeutics, Inc.<\/p>\n

Funci\u00f3n o cargo<\/strong>: Profesor Asociado (NCH) y Director Cient\u00edfico (Myonexus)<\/p>\n

 <\/p>\n

 <\/p>\n

\u00bfQu\u00e9 educaci\u00f3n y formaci\u00f3n ha recibido para llegar a su puesto actual?<\/strong><\/p>\n

Tengo un doctorado en gen\u00e9tica molecular que estudia el desarrollo neuromuscular. Despu\u00e9s hice una beca posdoctoral en terapia g\u00e9nica en el hospital infantil de todo el pa\u00eds con Jerry Mendell como mentor.<\/p>\n

\u00bfQu\u00e9 le llev\u00f3 a dedicarse a la investigaci\u00f3n y, en particular, al estudio de la distrofia muscular?<\/strong><\/p>\n

Desde muy joven supe que quer\u00eda ser investigadora. Me fascinaba la gen\u00e9tica y c\u00f3mo los genes defectuosos pod\u00edan causar enfermedades. Estudi\u00e9 el desarrollo neuromuscular en mi trabajo de posgrado y me interesaba aprovechar ese conocimiento para desarrollar tratamientos para las enfermedades musculares. Afortunadamente, la investigaci\u00f3n de la distrofia muscular es un \u00e1rea de \u00e9nfasis en el Nationwide Children's, lo que me brinda la oportunidad de cumplir mis objetivos.<\/p>\n

\u00bfQu\u00e9 temas est\u00e1 estudiando?<\/strong><\/p>\n

Desarrollo tratamientos de terapia g\u00e9nica para las distrofias musculares de cinturas y de Duchenne y traslado estos tratamientos a ensayos cl\u00ednicos. Mi laboratorio tambi\u00e9n estudia los mecanismos patol\u00f3gicos de las distrofias musculares de cinturas.<\/p>\n

\u00bfC\u00f3mo ayudar\u00e1 su trabajo a los pacientes? \u00bfTiene un car\u00e1cter m\u00e1s cient\u00edfico o podr\u00eda convertirse en un tratamiento para LGMD o MD en general?<\/strong><\/p>\n

Nuestro trabajo es directamente aplicable a los pacientes. Tenemos ensayos cl\u00ednicos en curso o inminentes para varias formas de LGMD (2B, 2D, 2E) y planeamos estudios cl\u00ednicos para otros subtipos. Somos muy optimistas en cuanto a que el reemplazo gen\u00e9tico ser\u00e1 un tratamiento significativo para la LGMD.<\/p>\n

\u00bfQu\u00e9 le gustar\u00eda que los pacientes y otras personas interesadas en la LGMD supieran sobre la investigaci\u00f3n (sus propios proyectos y sobre el campo en general)?<\/strong><\/p>\n

Es un momento muy emocionante para la investigaci\u00f3n de la LGMD. Las mejoras en las pruebas gen\u00e9ticas y los \u00e9xitos de la terapia g\u00e9nica en otros trastornos neuromusculares han acelerado la investigaci\u00f3n y los ensayos cl\u00ednicos de la LGMD. Podemos aplicar r\u00e1pidamente lo que aprendemos del estudio de una forma de LGMD a otra.<\/p>\n

\u00bfQu\u00e9 le inspira a seguir trabajando en este campo? <\/strong><\/p>\n

Los pacientes son mi mayor inspiraci\u00f3n. Es muy gratificante saber que todo el trabajo que hacemos para llevar los tratamientos a la cl\u00ednica beneficiar\u00e1 a los pacientes de una manera muy significativa.<\/p>\n

\u00bfC\u00f3mo pueden los pacientes animarle y ayudarle en su trabajo?<\/strong><\/p>\n

Escuchar historias de pacientes (tanto historias de esperanza como de retos) es extremadamente \u00fatil. Como investigadores, nos ayuda a conocer los retos espec\u00edficos de cada enfermedad para adaptar los tratamientos si es posible. Adem\u00e1s, es una fuente constante de motivaci\u00f3n para seguir trabajando duro sabiendo que hay un gran equipo de personas que te apoyan.<\/p>","protected":false},"excerpt":{"rendered":"

LGMD “Spotlight Interview on Research\u201d LGMD Researcher: Louise Rodino-Klapac, Ph.D […]<\/p>","protected":false},"author":1,"featured_media":1735,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[91,92],"tags":[],"class_list":["post-1737","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-researcher-interview","category-researcher"],"acf":[],"yoast_head":"\nLGMD RESEARCHER: Louise Rodino-Klapac, Ph.D - 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\/es\/researcher-interview\/2018\/03\/04\/1737\/\" \/>\n<meta property=\"og:locale\" content=\"es_MX\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"LGMD RESEARCHER: Louise Rodino-Klapac, Ph.D - 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