{"id":84229,"date":"2024-01-29T10:21:47","date_gmt":"2024-01-29T15:21:47","guid":{"rendered":"https:\/\/thecitizenfortheworld.com\/?p=84229"},"modified":"2024-01-29T10:21:47","modified_gmt":"2024-01-29T15:21:47","slug":"seis-ninos-con-sordera-total-recuperan-la-audicion-con-terapia-genetica","status":"publish","type":"post","link":"https:\/\/thecitizenfortheworld.com\/?p=84229","title":{"rendered":"Seis ni\u00f1os con sordera total recuperan la audici\u00f3n con terapia gen\u00e9tica"},"content":{"rendered":"<p>La sordera cong\u00e9nita la sufren alrededor de 26 millones de personas<\/p>\n<div class=\"paragraph\" data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">La p\u00e9rdida de audici\u00f3n afecta a m\u00e1s <b>de 1.500 millones de personas\u00a0<\/b>en todo el mundo, y la sordera cong\u00e9nita representa alrededor de 26 millones de esas personas. En el caso de la p\u00e9rdida auditiva en ni\u00f1os, m\u00e1s del 60 % se debe a razones gen\u00e9ticas.<\/div>\n<div class=\"paragraph\" data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">Un\u00a0<a href=\"http:\/\/dx.doi.org\/10.1016\/S0140-6736(23)02874-X\" target=\"_blank\" rel=\"noopener nofollow\" data-mrf-link=\"http:\/\/dx.doi.org\/10.1016\/S0140-6736(23)02874-X\">nuevo estudio<\/a>\u00a0codirigido por investigadores de Mass Eye and Ear acaba de demostrar la eficacia de una terapia gen\u00e9tica para restaurar la funci\u00f3n auditiva en ni\u00f1os que padecen sordera hereditaria. En un ensayo con\u00a0<b>seis ni\u00f1os\u00a0<\/b>que se llev\u00f3 a cabo en el Hospital Oftalmol\u00f3gico y Otorrinolaring\u00f3logo de la Universidad de Fudan en Shanghai, China, los investigadores descubrieron que la nueva terapia gen\u00e9tica es un tratamiento eficaz para pacientes con una forma espec\u00edfica de sordera autos\u00f3mica recesiva causada por mutaciones del OTOF. (otoferlina), llamado DFNB9.<\/div>\n<div data-mrf-recirculation=\"Enlaces p\u00e1rrafos\"><\/div>\n<div class=\"paragraph\" data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">Con su primer paciente tratado en diciembre de 2022, esta investigaci\u00f3n, publicada en\u00a0<i>The Lancet<\/i>, representa el\u00a0<b>primer ensayo cl\u00ednico en humanos<\/b>\u00a0que administra terapia g\u00e9nica para tratar esta afecci\u00f3n, con la mayor cantidad de pacientes tratados y el seguimiento m\u00e1s largo hasta la fecha.<\/div>\n<div data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">\u00abSi los ni\u00f1os no pueden o\u00edr, sus cerebros pueden desarrollarse anormalmente sin intervenci\u00f3n\u00bb, dijo\u00a0<b>Zheng-Yi Chen<\/b>, cient\u00edfico asociado de los Laboratorios Eaton-Peabody en Mass Eye and Ear y profesor asociado de Otorrinolaringolog\u00eda-Cirug\u00eda de Cabeza y Cuello en Escuela M\u00e9dica de Harvard. \u00abLos resultados de este estudio son realmente notables. Vimos que la capacidad auditiva de los ni\u00f1os mejoraba dr\u00e1sticamente semana tras semana, as\u00ed como la recuperaci\u00f3n del habla\u00bb, explica el cient\u00edfico en un comunicado.<\/div>\n<div data-mrf-recirculation=\"Enlaces p\u00e1rrafos\"><\/div>\n<div data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">\n<h2 class=\"subtitle\">Enfermedad hereditaria<\/h2>\n<div class=\"paragraph\" data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">El DFNB9 es una<b>\u00a0enfermedad hereditaria<\/b>\u00a0causada por\u00a0<b>mutaciones del gen OTOF\u00a0<\/b>y una falla en la producci\u00f3n de una prote\u00edna otoferlina funcional, necesaria para la transmisi\u00f3n de se\u00f1ales sonoras desde el o\u00eddo al cerebro. Actualmente, no existen medicamentos aprobados por la FDA para ayudar con la sordera hereditaria, lo que ha abierto la puerta a nuevas soluciones como las terapias gen\u00e9ticas.<\/div>\n<div class=\"paragraph\" data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">\u00abDesde que se inventaron los implantes cocleares hace 60 a\u00f1os, no ha habido un tratamiento eficaz para la sordera\u00bb, dijo Chen. \u00abEste es un gran hito que simboliza una nueva era en la lucha contra todo tipo de p\u00e9rdida auditiva\u00bb.<\/div>\n<blockquote id=\"m24621-24620-24622\">\n<p class=\"summary\">El tratamiento introdujo cuidadosamente el gen en los o\u00eddos internos de los pacientes mediante un procedimiento quir\u00fargico especial<\/p>\n<\/blockquote>\n<div class=\"paragraph\" data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">Para probar este novedoso tratamiento, se observ\u00f3 a seis ni\u00f1os con DFNB9 durante un per\u00edodo de 26 semanas en el Hospital Oftalmol\u00f3gico y Otorrinolaring\u00f3logo de la Universidad de Fudan. Los colaboradores de Mass Eye and Ear utilizaron un\u00a0<b>virus adenoasociado<\/b>\u00a0(AAV) que porta una versi\u00f3n del gen OTOF humano para introducir cuidadosamente el gen en los o\u00eddos internos de los pacientes mediante un procedimiento quir\u00fargico especial. Se utilizaron diferentes dosis de una \u00fanica inyecci\u00f3n del vector viral.<\/div>\n<\/div>\n<div data-mrf-recirculation=\"Enlaces p\u00e1rrafos\"><\/div>\n<div data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">\n<h2 class=\"subtitle\">Ni\u00f1os con sordera total<\/h2>\n<div class=\"paragraph\" data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">Los seis ni\u00f1os del estudio ten\u00edan sordera total, como lo indica un umbral promedio de respuesta auditiva del tronco encef\u00e1lico (ABR) de m\u00e1s de 95 decibeles. Despu\u00e9s de 26 semanas, cinco ni\u00f1os demostraron<b>\u00a0recuperaci\u00f3n auditiva<\/b>, mostrando una reducci\u00f3n de 40 a 57 decibelios en las pruebas ABR, mejoras dram\u00e1ticas en la percepci\u00f3n del habla y la capacidad restaurada para mantener una conversaci\u00f3n normal. En general,\u00a0<b>no se observ\u00f3 toxicidad limitante<\/b>\u00a0de la dosis. Durante el seguimiento de los pacientes se observaron 48 eventos adversos, siendo una importante mayor\u00eda (96 %) de bajo grado y el resto transitorios sin impacto a largo plazo.<\/div>\n<div data-mrf-recirculation=\"Enlaces p\u00e1rrafos\"><\/div>\n<div class=\"paragraph\" data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">Los investigadores planean ampliar el ensayo a un tama\u00f1o de muestra m\u00e1s grande, as\u00ed como realizar un seguimiento de sus resultados durante un per\u00edodo de tiempo m\u00e1s largo.<\/div>\n<\/div>\n<div data-mrf-recirculation=\"Enlaces p\u00e1rrafos\"><\/div>\n<div data-mrf-recirculation=\"Enlaces p\u00e1rrafos\">(<em>Con informaci\u00f3n de Infobae<\/em>)<\/div>\n<div data-mrf-recirculation=\"Enlaces p\u00e1rrafos\"><\/div>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>La sordera cong\u00e9nita la sufren alrededor de 26 millones de personas La p\u00e9rdida de audici\u00f3n afecta a m\u00e1s de 1.500 millones de personas\u00a0en todo el mundo, y la sordera cong\u00e9nita representa alrededor de 26 millones de esas personas. En el caso de la p\u00e9rdida auditiva en ni\u00f1os, m\u00e1s del 60 % se debe a razones [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":84230,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"om_disable_all_campaigns":false,"_mi_skip_tracking":false},"categories":[6],"tags":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v20.5 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Seis ni\u00f1os con sordera total recuperan la audici\u00f3n con terapia gen\u00e9tica - The citizen for the world<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/thecitizenfortheworld.com\/?p=84229\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Seis ni\u00f1os con sordera total recuperan la audici\u00f3n con terapia gen\u00e9tica - The citizen for the world\" \/>\n<meta property=\"og:description\" content=\"La sordera cong\u00e9nita la sufren alrededor de 26 millones de personas La p\u00e9rdida de audici\u00f3n afecta a m\u00e1s de 1.500 millones de personas\u00a0en todo el mundo, y la sordera cong\u00e9nita representa alrededor de 26 millones de esas personas. 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