{"id":12401,"date":"2021-03-11T21:08:40","date_gmt":"2021-03-12T02:08:40","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?p=12401"},"modified":"2022-11-14T17:12:14","modified_gmt":"2022-11-14T22:12:14","slug":"exciting-breakthroughs-in-rna-therapeutics-for-progeria","status":"publish","type":"post","link":"https:\/\/www.progeriaresearch.org\/es\/2021\/03\/11\/exciting-breakthroughs-in-rna-therapeutics-for-progeria\/","title":{"rendered":"\u00a1Avances emocionantes en terapias con ARN para la progeria!"},"content":{"rendered":"<p>[et_pb_section fb_built=&#8221;1&#8243; admin_label=&#8221;section&#8221; _builder_version=&#8221;4.16&#8243; global_colors_info=&#8221;{}&#8221;][et_pb_row admin_label=&#8221;row&#8221; _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.18.1&#8243; _module_preset=&#8221;default&#8221; hover_enabled=&#8221;0&#8243; global_colors_info=&#8221;{}&#8221; sticky_enabled=&#8221;0&#8243;]<\/p>\n<p><img loading=\"lazy\" decoding=\"async\" class=\"wp-image-12360 alignnone size-full aligncenter\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2021\/03\/Carlos.jpg\" alt=\"\" width=\"150\" height=\"300\" \/><\/p>\n<p>Estamos encantados de compartir los resultados de<strong> Dos estudios innovadores y muy interesantes sobre el uso de terapias con ARN<\/strong> en la investigaci\u00f3n sobre la progeria. Ambos estudios fueron cofinanciados por la Fundaci\u00f3n para la Investigaci\u00f3n de la Progeria (PRF) y coescritos por el director m\u00e9dico de la PRF, el Dr. Leslie Gordon.<\/p>\n<p>La progerina es la prote\u00edna que causa la enfermedad en la progeria. Las terapias con ARN interfieren en la capacidad del cuerpo para producir progerina, al bloquear su producci\u00f3n a nivel del ARN. Esto significa que<strong> El tratamiento es m\u00e1s espec\u00edfico que la mayor\u00eda de las terapias.<\/strong> que atacan la progerina a nivel proteico.<\/p>\n<p>Aunque cada estudio utiliz\u00f3 un sistema de administraci\u00f3n de f\u00e1rmacos diferente, ambos estudios apuntaron a la misma estrategia b\u00e1sica de tratamiento, inhibir la producci\u00f3n de ARN que codifica la prote\u00edna anormal, la progerina. Ambos fueron dirigidos por investigadores de los Institutos Nacionales de Salud (NIH) y fueron publicados hoy en la revista <em>Medicina natural<\/em>.<\/p>\n<p><a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01274-0\">Un estudio<\/a>, dirigido por Francis Collins, MD, PhD, Director del NIH, demostr\u00f3 que el tratamiento de ratones con progeria con un f\u00e1rmaco llamado SRP2001 r<strong>Redujo la expresi\u00f3n da\u00f1ina del ARNm y de la prote\u00edna progerina en la aorta.<\/strong>, la arteria principal del cuerpo, as\u00ed como en otros tejidos. Al final del estudio, la pared a\u00f3rtica permaneci\u00f3 m\u00e1s fuerte y los ratones demostraron una <strong>aumento de supervivencia de m\u00e1s de 60%<\/strong>.<\/p>\n<p>\u201cEl hecho de que una terapia de ARN dirigida haya mostrado resultados tan significativos en un modelo animal me da esperanzas de que esto podr\u00eda conducir a un avance importante en el tratamiento de la progeria\u201d, afirm\u00f3 Collins.<\/p>\n<p>El <a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01262-4\">Otro estudio<\/a>, dirigido por Tom Misteli, PhD, Director del Centro de Investigaci\u00f3n del C\u00e1ncer, Instituto Nacional del C\u00e1ncer, NIH, mostr\u00f3 una <strong>90 \u2013 95% reducci\u00f3n del ARN t\u00f3xico productor de progerina<\/strong> en diferentes tejidos despu\u00e9s del tratamiento con un f\u00e1rmaco llamado LB143. El laboratorio de Misteli descubri\u00f3 que la reducci\u00f3n de la prote\u00edna progerina era m\u00e1s eficaz en el h\u00edgado, con mejoras adicionales en el coraz\u00f3n y la aorta.<\/p>\n<p>Ahora sabemos que existen m\u00faltiples formas de disminuir la producci\u00f3n de la da\u00f1ina prote\u00edna progerina mediante terapias de ARN. Cada estudio encontr\u00f3 diferentes tramos de ARN en los modelos de rat\u00f3n que, cuando se dirig\u00edan a ellos, generaban una v\u00eda eficaz para el tratamiento, lo que daba como resultado <strong>Ratones con progeria que vivieron mucho m\u00e1s tiempo que los tratados en estudios previos con lonafarnib<\/strong>, el \u00fanico f\u00e1rmaco aprobado por la FDA para ni\u00f1os con progeria. Adem\u00e1s, los investigadores descubrieron que un tratamiento combinado con terapias de ARN y lonafarnib redujo los niveles de prote\u00edna progerina en el h\u00edgado y el coraz\u00f3n de manera m\u00e1s efectiva que cada tratamiento por separado.<\/p>\n<p>\u201cEstos dos estudios de gran importancia demuestran la <strong>Los principales avances que ya tenemos entre manos<\/strong> \u201cEn el campo de las terapias dirigidas contra la progeria\u201d, afirm\u00f3 la directora m\u00e9dica de PRF, la Dra. Leslie Gordon. \u201cMe encant\u00f3 trabajar con estos brillantes grupos de investigaci\u00f3n para avanzar en la terapia de ARN para ni\u00f1os con progeria. Ambos son estudios de prueba de principio apasionantes y <strong>PRF est\u00e1 entusiasmada por avanzar hacia los ensayos cl\u00ednicos<\/strong> que aplican estas estrategias de tratamiento.<\/p>\n<p>&nbsp;<\/p>\n<p>\u2014<\/p>\n<p>Erdos, MR, Cabral, WA, Tav\u00e1rez, UL\u00a0<i>y otros.<\/i>\u00a0Un enfoque terap\u00e9utico antisentido dirigido para el s\u00edndrome de progeria de Hutchinson-Gilford.\u00a0<i>Medicina natural<\/i>\u00a0(2021). https:\/\/doi.org\/10.1038\/s41591-021-01274-0<\/p>\n<p>Puttaraju, M., Jackson, M., Klein, S.\u00a0<i>y otros.<\/i>\u00a0La detecci\u00f3n sistem\u00e1tica identifica oligonucle\u00f3tidos antisentido terap\u00e9uticos para el s\u00edndrome de progeria de Hutchinson-Gilford.\u00a0<i>Medicina natural<\/i>\u00a0(2021). https:\/\/doi.org\/10.1038\/s41591-021-01262-4<\/p>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>","protected":false},"excerpt":{"rendered":"<p>Nos complace compartir los resultados de dos estudios innovadores y muy interesantes sobre el uso de terapias de ARN en la investigaci\u00f3n de la progeria. Ambos estudios fueron cofinanciados por la Fundaci\u00f3n para la Investigaci\u00f3n de la Progeria (PRF) y escritos en coautor\u00eda por el director m\u00e9dico de la PRF, el Dr. Leslie Gordon. <\/p>","protected":false},"author":2,"featured_media":12414,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[2,1],"tags":[],"class_list":["post-12401","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","category-uncategorized"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Exciting breakthroughs in RNA Therapeutics for Progeria! | The Progeria Research Foundation<\/title>\n<meta name=\"description\" content=\"Two very exciting breakthrough studies on the 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