{"id":130327,"date":"2024-12-11T16:11:06","date_gmt":"2024-12-11T21:11:06","guid":{"rendered":"https:\/\/wfh.org\/article\/el-new-england-journal-of-medicine-publica-articulo-sobre-innovadora-y-revolucionaria-terapia-genica\/"},"modified":"2025-01-15T17:08:25","modified_gmt":"2025-01-15T22:08:25","slug":"el-new-england-journal-of-medicine-publica-articulo-sobre-innovadora-y-revolucionaria-terapia-genica","status":"publish","type":"post","link":"https:\/\/wfh.org\/es\/article\/el-new-england-journal-of-medicine-publica-articulo-sobre-innovadora-y-revolucionaria-terapia-genica\/","title":{"rendered":"El New England Journal of Medicine publica art\u00edculo sobre innovadora y revolucionaria terapia g\u00e9nica"},"content":{"rendered":"\t\t<div data-elementor-type=\"wp-post\" data-elementor-id=\"130327\" class=\"elementor elementor-130327 elementor-130302\" data-elementor-post-type=\"post\">\n\t\t\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-2bf28c1 elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"2bf28c1\" data-element_type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-688b1ac\" data-id=\"688b1ac\" data-element_type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-f5f67b6 elementor-widget elementor-widget-text-editor\" data-id=\"f5f67b6\" data-element_type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t<style>\/*! elementor - v3.23.0 - 25-07-2024 *\/\n.elementor-widget-text-editor.elementor-drop-cap-view-stacked .elementor-drop-cap{background-color:#69727d;color:#fff}.elementor-widget-text-editor.elementor-drop-cap-view-framed .elementor-drop-cap{color:#69727d;border:3px solid;background-color:transparent}.elementor-widget-text-editor:not(.elementor-drop-cap-view-default) .elementor-drop-cap{margin-top:8px}.elementor-widget-text-editor:not(.elementor-drop-cap-view-default) .elementor-drop-cap-letter{width:1em;height:1em}.elementor-widget-text-editor .elementor-drop-cap{float:left;text-align:center;line-height:1;font-size:50px}.elementor-widget-text-editor .elementor-drop-cap-letter{display:inline-block}<\/style>\t\t\t\t<p>En el estudio participaron cinco hombres de entre 22 y 41 a\u00f1os con hemofilia A grave, pero sin inhibidores del factor VIII. Recibieron seguimiento durante una mediana de 14 meses (desde 9 hasta 27 meses). Los resultados mostraron que la terapia logr\u00f3 mantener el factor VIII en niveles que impidieron hemorragias espont\u00e1neas (mediana del nivel de actividad del factor de 20-40%), sin episodios hemorr\u00e1gicos durante o despu\u00e9s del tratamiento. Antes del tratamiento, los participantes ten\u00edan una tasa anual de hemorragias de 20 a 120 episodios.<\/p>\n<p>Se observaron algunos efectos secundarios temporales esperados, entre ellos neutropenia grave (recuento bajo de gl\u00f3bulos blancos) y trombocitopenia grave (recuento bajo de plaquetas), los cuales se resolvieron en un plazo de 11 d\u00edas. Adem\u00e1s de estos, no se observaron efectos secundarios graves. Es importante destacar que ning\u00fan participante present\u00f3 inhibidores del factor VIII despu\u00e9s de la terapia y que no hubo muertes durante el per\u00edodo del estudio.<\/p>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-34eac75 elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"34eac75\" data-element_type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-77800f6\" data-id=\"77800f6\" data-element_type=\"column\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-7938b67 elementor-view-default elementor-widget elementor-widget-icon\" data-id=\"7938b67\" data-element_type=\"widget\" data-widget_type=\"icon.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<div class=\"elementor-icon-wrapper\">\n\t\t\t<div class=\"elementor-icon\">\n\t\t\t<i aria-hidden=\"true\" class=\"fas fa-quote-left\"><\/i>\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-2c8a724\" data-id=\"2c8a724\" data-element_type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-acc04bf elementor-widget elementor-widget-heading\" data-id=\"acc04bf\" data-element_type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t<style>\/*! elementor - v3.23.0 - 25-07-2024 *\/\n.elementor-heading-title{padding:0;margin:0;line-height:1}.elementor-widget-heading .elementor-heading-title[class*=elementor-size-]>a{color:inherit;font-size:inherit;line-height:inherit}.elementor-widget-heading .elementor-heading-title.elementor-size-small{font-size:15px}.elementor-widget-heading .elementor-heading-title.elementor-size-medium{font-size:19px}.elementor-widget-heading .elementor-heading-title.elementor-size-large{font-size:29px}.elementor-widget-heading .elementor-heading-title.elementor-size-xl{font-size:39px}.elementor-widget-heading .elementor-heading-title.elementor-size-xxl{font-size:59px}<\/style><h2 class=\"elementor-heading-title elementor-size-default\">Este estudio de cinco personas con hemofilia A establece que un nuevo vector viral puede transportar el gene del factor VIII a las c\u00e9lulas madre de la m\u00e9dula \u00f3sea y producir prote\u00edna del FVIII circulante y estable. Dado que el factor VIII se integra a los cromosomas, se espera que dure muchos a\u00f1os, posiblemente toda la vida. Si bien a\u00fan es necesario mucho trabajo antes de que esta tecnolog\u00eda se convierta en una modalidad de tratamiento viable, el estudio ofrece pruebas s\u00f3lidas de que este enfoque puede resultar seguro y eficaz para, a la larga, lograr una cura.<\/h2>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-756db7e elementor-widget elementor-widget-heading\" data-id=\"756db7e\" data-element_type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t<p class=\"elementor-heading-title elementor-size-default\">\u2014Dr. Glenn Pierce, PhD, vicepresidente m\u00e9dico de la FMH<\/p>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<div class=\"elementor-element elementor-element-08f6f9a elementor-widget elementor-widget-text-editor\" data-id=\"08f6f9a\" data-element_type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<p>Estos primeros resultados abordan algunos de los desaf\u00edos observados con otros tipos de terapia g\u00e9nica y son prometedores como tratamiento a largo plazo (y posiblemente de por vida) para la hemofilia A. Este enfoque tambi\u00e9n podr\u00eda hacer que la terapia g\u00e9nica resulte accesible para m\u00e1s pacientes, entre ellos ni\u00f1os.<\/p>\n<p>Para leer el art\u00edculo (en ingl\u00e9s) del profesor Srivastava haga <a href=\"https:\/\/www.nejm.org\/doi\/full\/10.1056\/NEJMoa2410597\" target=\"_blank\" rel=\"noopener\">clic aqu\u00ed<\/a>. Acompa\u00f1a al art\u00edculo un editorial del profesor Johnny Mahlangu, de la Universidad de Witswatersrand, que compara y contrasta la terapia g\u00e9nica con lentivirus en relaci\u00f3n con el vector viral adenoasociado (VAA) utilizado hasta ahora en la elaboraci\u00f3n de terapias g\u00e9nicas para la hemofilia: <em>Could Lentivirus Overcome the AAV Gene-Therapy Challenges in Hemophilia A? <\/em>(\u00bfPodr\u00eda el lentivirus superar los desaf\u00edos de la terapia g\u00e9nica con VAA para la hemofilia A?), publicado en el New England Journal of Medicine<em>.<\/em> Haga <a href=\"https:\/\/www.nejm.org\/doi\/full\/10.1056\/NEJMe2414214\" target=\"_blank\" rel=\"noopener\">clic aqu\u00ed<\/a> para leer el editorial (en ingl\u00e9s).<\/p>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<\/div>\n\t\t","protected":false},"excerpt":{"rendered":"<p>Un nuevo art\u00edculo publicado en el New England Journal of Medicine, Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A (Terapia g\u00e9nica lentiviral con c\u00e9lulas hematopoy\u00e9ticas CD34+ para la hemofilia A) describe un estudio en el que se utiliza una innovadora y revolucionaria terapia g\u00e9nica con un virus diferente al usado hasta ahora para transportar el gene del factor VIII. El estudio ofrece pruebas prometedoras de la seguridad y eficacia de este nuevo enfoque de terapia g\u00e9nica.<\/p>\n","protected":false},"author":13,"featured_media":130307,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"inline_featured_image":false,"footnotes":""},"categories":[339,196],"tags":[],"search-category":[85],"class_list":["post-130327","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-featured-3-es","category-noticias-medicas","search-category-article"],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v23.4 (Yoast SEO v23.4) - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>El New England Journal of Medicine publica art\u00edculo sobre innovadora y revolucionaria terapia g\u00e9nica | FMH - Federaci\u00f3n Mundial de Hemofilia<\/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:\/\/wfh.org\/es\/article\/el-new-england-journal-of-medicine-publica-articulo-sobre-innovadora-y-revolucionaria-terapia-genica\/\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"El New England Journal of Medicine publica art\u00edculo sobre innovadora y revolucionaria terapia g\u00e9nica\" \/>\n<meta property=\"og:description\" content=\"Un nuevo art\u00edculo publicado en el New England Journal of Medicine, Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A (Terapia g\u00e9nica lentiviral con c\u00e9lulas hematopoy\u00e9ticas CD34+ para la hemofilia A) describe un estudio en el que se utiliza una innovadora y revolucionaria terapia g\u00e9nica con un virus diferente al usado hasta ahora para transportar el gene del factor VIII. El estudio ofrece pruebas prometedoras de la seguridad y eficacia de este nuevo enfoque de terapia g\u00e9nica.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/wfh.org\/es\/article\/el-new-england-journal-of-medicine-publica-articulo-sobre-innovadora-y-revolucionaria-terapia-genica\/\" \/>\n<meta property=\"og:site_name\" content=\"FMH - Federaci\u00f3n Mundial de Hemofilia\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/wfhemophilia\/\" \/>\n<meta property=\"article:published_time\" content=\"2024-12-11T21:11:06+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2025-01-15T22:08:25+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/wfh.org\/wp-content\/uploads\/2024\/12\/New-England-Journal-of-Medicine-NEJM-article-header-SP.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"1200\" \/>\n\t<meta property=\"og:image:height\" content=\"627\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Mitch Semienchuk\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Escrito por\" \/>\n\t<meta name=\"twitter:data1\" content=\"Mitch Semienchuk\" \/>\n\t<meta name=\"twitter:label2\" content=\"Tiempo de lectura\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutos\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/wfh.org\/es\/article\/el-new-england-journal-of-medicine-publica-articulo-sobre-innovadora-y-revolucionaria-terapia-genica\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/wfh.org\/es\/article\/el-new-england-journal-of-medicine-publica-articulo-sobre-innovadora-y-revolucionaria-terapia-genica\/\"},\"author\":{\"name\":\"Mitch Semienchuk\",\"@id\":\"https:\/\/wfh.org\/es\/#\/schema\/person\/f57928f0dd0ca8906b81082895256d8f\"},\"headline\":\"El New England Journal of Medicine publica art\u00edculo sobre innovadora y revolucionaria terapia g\u00e9nica\",\"datePublished\":\"2024-12-11T21:11:06+00:00\",\"dateModified\":\"2025-01-15T22:08:25+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/wfh.org\/es\/article\/el-new-england-journal-of-medicine-publica-articulo-sobre-innovadora-y-revolucionaria-terapia-genica\/\"},\"wordCount\":452,\"publisher\":{\"@id\":\"https:\/\/wfh.org\/es\/#organization\"},\"image\":{\"@id\":\"https:\/\/wfh.org\/es\/article\/el-new-england-journal-of-medicine-publica-articulo-sobre-innovadora-y-revolucionaria-terapia-genica\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/wfh.org\/wp-content\/uploads\/2024\/12\/New-England-Journal-of-Medicine-NEJM-article-header-SP.jpg\",\"articleSection\":[\"Featured 3 - 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