{"id":3561,"date":"2022-11-03T19:07:02","date_gmt":"2022-11-03T19:07:02","guid":{"rendered":"https:\/\/neuropediatoolbox.org\/?p=3561"},"modified":"2026-06-22T20:07:21","modified_gmt":"2026-06-22T20:07:21","slug":"oligonucleotidos-antisentido","status":"publish","type":"post","link":"https:\/\/neuropediatoolkit.org\/en\/oligonucleotidos-antisentido\/","title":{"rendered":"In cis."},"content":{"rendered":"\n<p>Los <a href=\"https:\/\/es.wikipedia.org\/wiki\/Oligonucle%C3%B3tido_antisentido\">oligonucle\u00f3tidos antisentido<\/a> son la primera terapia g\u00e9nica (en realidad <a href=\"https:\/\/en.wikipedia.org\/wiki\/Antisense_therapy\">terapia RNA antisentido<\/a>, ya que no modifican el c\u00f3digo gen\u00e9tico) disponible en el mercado para el tratamiento de enfermedades neurogen\u00e9ticas. Se basan en la t\u00e9cnica del <a href=\"https:\/\/en.wikipedia.org\/wiki\/Exon_skipping\">salto del ex\u00f3n<\/a> (Duchenne) o de la inclusi\u00f3n del ex\u00f3n (AME). Pr\u00f3ximamente estar\u00e1 disponible tambi\u00e9n tratamiento para la distrofia miot\u00f3nica de Steinert. <\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><a href=\"https:\/\/es.wikipedia.org\/wiki\/Nusinersen\">Nusinersen <\/a>(<a href=\"https:\/\/www.spinraza.com\/\">Spinraza<\/a>). <\/li>\n\n\n\n<li><a href=\"https:\/\/es.wikipedia.org\/wiki\/Eteplirsen\">Eteplirsen <\/a>(<a href=\"https:\/\/www.exondys51.com\/\">Exondys51<\/a>). <\/li>\n\n\n\n<li>Golodirsen (<a href=\"https:\/\/www.vyondys53.com\/index-es.html\">Vyondys 53<\/a>). <\/li>\n<\/ul>\n\n\n\n<figure class=\"wp-block-image size-full\"><img fetchpriority=\"high\" decoding=\"async\" width=\"1024\" height=\"888\" src=\"https:\/\/neuropediatoolkit.org\/wp-content\/uploads\/2022\/11\/imagen-36.png\" alt=\"\" class=\"wp-image-3562\" srcset=\"https:\/\/neuropediatoolkit.org\/wp-content\/uploads\/2022\/11\/imagen-36.png 1024w, https:\/\/neuropediatoolkit.org\/wp-content\/uploads\/2022\/11\/imagen-36-300x260.png 300w, 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id=\"zp-ID-3561-19955111-JMSPQW4N\" data-zp-author-date='Daguenet-et-al.-2015-12' data-zp-date-author='2015-12-Daguenet-et-al.' data-zp-date='2015-12' data-zp-year='2015' data-zp-itemtype='journalArticle' class=\"zp-Entry zpSearchResultsItem\">\n<div class=\"csl-bib-body\" style=\"line-height: 1.35; \">\n  <div class=\"csl-entry\" style=\"clear: left; \">\n    <div class=\"csl-left-margin\" style=\"float: left; padding-right: 0.5em; text-align: right; width: 1em;\">1.<\/div><div class=\"csl-right-inline\" style=\"margin: 0 .4em 0 1.5em;\">Daguenet E, Dujardin G, Valc\u00e1rcel J. The pathogenicity of splicing defects: mechanistic insights into pre-mRNA processing inform novel therapeutic approaches. EMBO reports [Internet]. 2015 Dec [cited 2022 Nov 3];16(12):1640\u201355. Available from: <a class='zp-ItemURL' href='https:\/\/www.embopress.org\/doi\/full\/10.15252\/embr.201541116'>https:\/\/www.embopress.org\/doi\/full\/10.15252\/embr.201541116<\/a><\/div>\n  <\/div>\n<\/div>\n\t\t\t\t<\/div><!-- .zp-Entry .zpSearchResultsItem -->\n\t\t\t<\/div><!-- .zp-zp-SEO-Content -->\n\t\t<\/div><!-- .zp-List -->\n\t<\/div><!--.zp-Zotpress-->\n\n\n","protected":false},"excerpt":{"rendered":"<p>{332710:GSPGSS3E};{332710:HSPJSS3A};{332710:7QAVS6AS} <\/p>\n<p class=\"link-more\"><a href=\"https:\/\/neuropediatoolkit.org\/en\/oligonucleotidos-antisentido\/\" class=\"more-link\">Continue reading<span class=\"screen-reader-text\"> \"Antisense oligonucleotides.\"<\/span><\/a><\/p>","protected":false},"author":1,"featured_media":3564,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_themeisle_gutenberg_block_has_review":false,"footnotes":""},"categories":[10],"tags":[],"class_list":["post-3561","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-neurogenetica","entry"],"_links":{"self":[{"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/posts\/3561","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/comments?post=3561"}],"version-history":[{"count":3,"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/posts\/3561\/revisions"}],"predecessor-version":[{"id":8441,"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/posts\/3561\/revisions\/8441"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/media\/3564"}],"wp:attachment":[{"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/media?parent=3561"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/categories?post=3561"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/neuropediatoolkit.org\/en\/wp-json\/wp\/v2\/tags?post=3561"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}