{"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\/es\/researcher-interview\/2018\/06\/19\/lgmd-researcher-chamberlain\/","title":{"rendered":"INVESTIGADOR LGMD: Jeffrey S. Chamberlain, Ph.D."},"content":{"rendered":"<p><strong>\"LA LGMD EN EL PUNTO DE MIRA DE LA INVESTIGACI\u00d3N\"<\/strong><\/p>\n<p><strong>Investigador del LGMD:<\/strong> Doctor Jeffrey S. Chamberlain<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>Afiliaci\u00f3n:<\/strong>\u00a0 Facultad de Medicina de la Universidad de Washington<\/p>\n<p><strong>Funci\u00f3n o cargo<\/strong>:  Profesor, C\u00e1tedra McCaw de Distrofia Muscular, Director, Centro Wellstone de Investigaci\u00f3n de la Distrofia Muscular-Seattle<\/p>\n<p><strong>\u00bfQu\u00e9 educaci\u00f3n y formaci\u00f3n ha recibido para llegar a su puesto actual?<\/strong><\/p>\n<p>Obtuve una licenciatura y un doctorado en Bioqu\u00edmica, y despu\u00e9s hice un posdoctorado en Gen\u00e9tica Molecular.<\/p>\n<p><strong>\u00bfQu\u00e9 le llev\u00f3 a dedicarse a la investigaci\u00f3n y, en particular, al estudio de la distrofia muscular?<\/strong><\/p>\n<p>Mientras hac\u00eda el doctorado me interes\u00e9 por el desarrollo muscular y empec\u00e9 a aprender sobre enfermedades musculares.  Hice un posdoctorado estudiando la distrofia muscular de Duchenne y empec\u00e9 a interesarme e intrigarme por el concepto de terapia g\u00e9nica.  Cuando cre\u00e9 mi propio laboratorio, decid\u00ed centrarme en la terapia g\u00e9nica de las distrofias musculares, as\u00ed como en el estudio de la funci\u00f3n de las prote\u00ednas defectuosas en las distintas distrofias.<\/p>\n<p><strong>\u00bfQu\u00e9 temas est\u00e1 estudiando?<\/strong><\/p>\n<p>Actualmente estudio m\u00e9todos para terapia g\u00e9nica de des\u00f3rdenes musculares, incluyendo vectores para entregar genes a tejidos musculares, casetes reguladores de genes para activar y desactivar genes en diferentes tejidos, y el papel del sistema inmune en distrofias y en el contexto de terapia g\u00e9nica.  Tambi\u00e9n estudio la funci\u00f3n y las interacciones de prote\u00ednas diferentes que contribuyen a la funci\u00f3n muscular y la enfermedad, como la distrofina y FKRP.<\/p>\n<p><strong>\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<p>Nuestro trabajo se centra tanto en la ciencia como en el tratamiento.  Creemos que la mejor manera de desarrollar tratamientos eficaces es conocer en detalle la biolog\u00eda b\u00e1sica de los diversos trastornos musculares.  Utilizamos esta investigaci\u00f3n b\u00e1sica para guiar el desarrollo de los genes y los veh\u00edculos de administraci\u00f3n necesarios para una terapia g\u00e9nica segura y eficaz.<\/p>\n<p><strong>\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<p>La terapia g\u00e9nica ha pasado de ser un concepto futurista a un enfoque prometedor para tratar trastornos hereditarios.  Varias terapias g\u00e9nicas han sido aprobadas para su uso comercial y pueden ser prescritas por los m\u00e9dicos.  Creo que las terapias g\u00e9nicas son el enfoque m\u00e1s prometedor para tratar la mayor\u00eda de los distintos tipos de distrofia muscular.  Tambi\u00e9n es importante darse cuenta de que los retos que limitan el desarrollo de la terapia g\u00e9nica para muchas distrofias son los mismos para los distintos trastornos, y que los avances en uno de ellos ayudan a acercar los tratamientos a la cl\u00ednica para todas las distrofias.  Se est\u00e1n realizando ensayos de terapia g\u00e9nica para varias distrofias musculares y muchos m\u00e1s est\u00e1n a punto de ser probados.  Estamos al borde de una revoluci\u00f3n en el desarrollo y la aplicaci\u00f3n de nuevas terapias gen\u00e9ticas y creo que es un momento incre\u00edble para ver c\u00f3mo estas nuevas tecnolog\u00edas empiezan por fin a ayudar a los pacientes y sus familias.<\/p>\n<p><strong>\u00bfQu\u00e9 le inspira a seguir trabajando en este campo? <\/strong><\/p>\n<p>Llevo 30 a\u00f1os trabajando en la distrofia muscular y por fin estoy viendo c\u00f3mo se aplican y funcionan en la cl\u00ednica nuevas terapias.  Es el momento m\u00e1s emocionante de mi carrera y estoy encantada de poder contribuir al desarrollo de nuevos enfoques para tratar distintos tipos de trastornos musculares.<\/p>\n<p><strong>\u00bfC\u00f3mo pueden los pacientes animarle y ayudarle en su trabajo?<\/strong><\/p>\n<p>La financiaci\u00f3n de la investigaci\u00f3n cient\u00edfica est\u00e1 disminuyendo en todo el pa\u00eds, y en muchos sectores parece haber casi una guerra contra las ideas y principios cient\u00edficos que se ense\u00f1an y exploran.  Lo m\u00e1s importante que pueden hacer los pacientes y sus familiares es animar a los miembros de sus gobiernos locales y nacionales a que apoyen la investigaci\u00f3n cient\u00edfica en las universidades de todo el pa\u00eds.  Casi todas las terapias innovadoras y novedosas, como las terapias g\u00e9nicas y celulares, se desarrollan exclusivamente en las universidades antes de que las biotecnol\u00f3gicas y las grandes farmac\u00e9uticas las adopten.<\/p>\n<p>&nbsp;<\/p>\n<p>* Por favor, dale al LIKE, COMENTA y COMPARTE este post para ayudar a concienciar sobre la LGMD.<\/p>\n<p>* Si desea m\u00e1s informaci\u00f3n sobre la distrofia muscular de cinturas o leer m\u00e1s \"Entrevistas en primer plano\", visite nuestro sitio web en <a href=\"https:\/\/www.lgmd-info.org\/es\/\">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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