{"id":47262,"date":"2023-10-16T08:46:50","date_gmt":"2023-10-16T11:46:50","guid":{"rendered":"https:\/\/academiadepacientes.com.br\/?p=47262"},"modified":"2026-02-18T12:27:19","modified_gmt":"2026-02-18T15:27:19","slug":"deja-vu-o-recente-fiasco-da-terapia-genica-em-distrofia-muscular","status":"publish","type":"post","link":"https:\/\/academiadepacientes.com.br\/?p=47262","title":{"rendered":"Deja v\u00fa? O recente fiasco da terapia g\u00eanica em distrofia muscular"},"content":{"rendered":"\n<div class=\"wp-block-uagb-blockquote uagb-block-b1037190 uagb-blockquote__skin-border uagb-blockquote__stack-img-none\"><blockquote class=\"uagb-blockquote\"><div class=\"uagb-blockquote__content\"><strong>O\u00a0<em>d\u00e9j\u00e0<\/em>&#8211;<em>vu<\/em>\u00a0\u00e9 a sensa\u00e7\u00e3o de j\u00e1 ter visto ou vivido uma situa\u00e7\u00e3o que est\u00e1 acontecendo no presente.<\/strong><\/div><footer><div class=\"uagb-blockquote__author-wrap uagb-blockquote__author-at-left\"><\/div><\/footer><\/blockquote><\/div>\n\n\n\n<p><strong>O que h\u00e1 de novo<\/strong>: Promessas terap\u00eauticas exacerbadas na m\u00eddia,  desafios de concretiza\u00e7\u00e3o na pesquisa cl\u00ednica e \u00f3bitos de pacientes definem, a m\u00e9dio prazo, um futuro pouco promissor para as terapias g\u00eanicas. Apesar de todo oba-oba criado por lobbies de todos os matizes, apenas seis terapias do g\u00eanero foram aprovadas pelo FDA at\u00e9 o momento. E para doen\u00e7as que acometem poucas pessoas. <\/p>\n\n\n\n<p><strong>Por que isso importa<\/strong>: A terapia gen\u00e9tica carrega o potencial revolucion\u00e1rio de tratar doen\u00e7as g\u00eanicas, mas a viabilidade e seguran\u00e7a ainda s\u00e3o quest\u00f5es pendentes a serem resolvidas.<\/p>\n\n\n\n<p><strong>O quadro geral<\/strong>:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>A terapia gen\u00e9tica, embora promissora, ainda \u00e9 um campo emergente com apenas seis terapias aprovadas pelo FDA at\u00e9 agora, indicando que h\u00e1 muito espa\u00e7o para explora\u00e7\u00e3o e melhorias (FDA).<\/li>\n\n\n\n<li>Um caso recente de morte p\u00f3s-terapia g\u00eanica em um paciente com Distrofia Muscular de Duchenne ressalta os riscos associados, especialmente as rea\u00e7\u00f5es imunol\u00f3gicas adversas que podem ser fatalmente imprevis\u00edveis.<\/li>\n\n\n\n<li>As ferramentas de edi\u00e7\u00e3o gen\u00e9tica como CRISPR s\u00e3o avan\u00e7os tecnol\u00f3gicos significativos, mas a resposta imunol\u00f3gica do corpo e outros fatores biol\u00f3gicos complexos s\u00e3o desafios que ainda precisam ser superados para garantir a seguran\u00e7a e efic\u00e1cia da terapia gen\u00e9tica.<\/li>\n<\/ul>\n\n\n\n<div style=\"height:100px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<div class=\"wp-block-uagb-blockquote uagb-block-c8d84f3e uagb-blockquote__skin-border uagb-blockquote__stack-img-none\"><blockquote class=\"uagb-blockquote\"><div class=\"uagb-blockquote__content\"><strong>\u00c9 dif\u00edcil desafiar um bilh\u00e3o de anos de evolu\u00e7\u00e3o<\/strong><\/div><footer><div class=\"uagb-blockquote__author-wrap uagb-blockquote__author-at-left\"><cite class=\"uagb-blockquote__author\">Ricky Lewis, PhD<\/cite><\/div><\/footer><\/blockquote><\/div>\n\n\n\n<div style=\"height:100px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<h2 class=\"wp-block-heading\"><a href=\"https:\/\/academiadepacientes.com.br\/seria-um-deja-vu-o-recente-fiasco-da-terapia-genica-para-distrofia-muscular-de-duchenne\/\" target=\"_blank\" rel=\"noopener\" title=\"\">Clique aqui para saber mais<\/a><\/h2>\n","protected":false},"excerpt":{"rendered":"<p>Promessas terap\u00eauticas exacerbadas na m\u00eddia,  desafios de concretiza\u00e7\u00e3o na pesquisa cl\u00ednica e \u00f3bitos de pacientes definem, a m\u00e9dio prazo, um futuro pouco promissor para as terapias g\u00eanicas. <\/p>\n","protected":false},"author":2,"featured_media":3055,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"om_disable_all_campaigns":false,"_uag_custom_page_level_css":"","_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"footnotes":""},"categories":[267,121],"tags":[],"class_list":["post-47262","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-arquivos-de-transicao","category-artigos","infinite-scroll-item","resize-featured-image"],"aioseo_notices":[],"uagb_featured_image_src":{"full":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2020\/04\/dna-4092762_640.jpg",640,412,false],"thumbnail":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:150\/h:150\/q:mauto\/rt:fill\/g:ce\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2020\/04\/dna-4092762_640.jpg",150,150,true],"medium":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:300\/h:193\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2020\/04\/dna-4092762_640.jpg",300,193,true],"medium_large":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2020\/04\/dna-4092762_640.jpg",640,412,false],"large":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2020\/04\/dna-4092762_640.jpg",640,412,false],"1536x1536":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2020\/04\/dna-4092762_640.jpg",640,412,false],"2048x2048":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2020\/04\/dna-4092762_640.jpg",640,412,false],"mailpoet_newsletter_max":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2020\/04\/dna-4092762_640.jpg",640,412,false]},"uagb_author_info":{"display_name":"Cl\u00e1udio Cordovil","author_link":"https:\/\/academiadepacientes.com.br\/?author=2"},"uagb_comment_info":0,"uagb_excerpt":"Promessas terap\u00eauticas exacerbadas na m\u00eddia, desafios de concretiza\u00e7\u00e3o na pesquisa cl\u00ednica e \u00f3bitos de pacientes definem, a m\u00e9dio prazo, um futuro pouco promissor para as terapias g\u00eanicas.","_links":{"self":[{"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/posts\/47262","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=47262"}],"version-history":[{"count":5,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/posts\/47262\/revisions"}],"predecessor-version":[{"id":47284,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/posts\/47262\/revisions\/47284"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/media\/3055"}],"wp:attachment":[{"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=47262"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=47262"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=47262"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}