{"id":557183,"date":"2026-06-06T12:42:24","date_gmt":"2026-06-06T10:42:24","guid":{"rendered":"https:\/\/medizinonline.com\/ttr-stabilizers-gene-silencing-and-gene-scissors-where-do-we-stand\/"},"modified":"2026-06-06T12:42:32","modified_gmt":"2026-06-06T10:42:32","slug":"ttr-stabilizers-gene-silencing-and-gene-scissors-where-do-we-stand","status":"publish","type":"post","link":"https:\/\/medizinonline.com\/en\/ttr-stabilizers-gene-silencing-and-gene-scissors-where-do-we-stand\/","title":{"rendered":"TTR stabilizers, gene silencing and gene scissors: where do we stand?"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\"><strong>Advances in the understanding of the pathophysiology of transthyretin amyloid cardiomyopathy (ATTR-CM) have revolutionized the therapeutic landscape in recent years. TTR stabilizers such as tafamidis and acoramidis can reduce TTR instability and subsequent amyloid fibril formation, and gene silencing therapies with small interfering RNAs such as patisiran and vutrisiran or antisense oligonucleotide inhibitors such as inotersen and eplontersen also represent promising therapeutic options as they reduce TTR production. Further gene silencers, gene therapies and combined treatment strategies are being researched in ongoing studies.    <\/strong><\/p>\n\n<div class=\"pmpro\"><div class=\"pmpro_card pmpro_content_message\"><div class=\"pmpro_card_content\">Dieser Inhalt ist nur f\u00fcr Benutzer der Gruppe: Medizinische Fachperson zug\u00e4nglich <br \/><a href=\"https:\/\/medizinonline.com\/en\/my-account\/packages\/\">Jetzt beitreten<\/a><\/div><div class=\"pmpro_card_actions pmpro_font-medium\">Already a member? <a href=\"https:\/\/medizinonline.com\/en\/log-in\/?redirect_to=https%3A%2F%2Fmedizinonline.com%2Fen%2Fttr-stabilizers-gene-silencing-and-gene-scissors-where-do-we-stand%2F\">Log in here<\/a><\/div><\/div><\/div>","protected":false},"excerpt":{"rendered":"<p>Advances in the understanding of the pathophysiology of transthyretin amyloid cardiomyopathy (ATTR-CM) have revolutionized the therapeutic landscape in recent years. TTR stabilizers such as tafamidis and acoramidis can reduce TTR&#8230;&hellip;... <\/p>\n<div class=\"pmpro\"><div class=\"pmpro_card pmpro_content_message\"><div class=\"pmpro_card_content\">Dieser Inhalt ist nur f\u00fcr Benutzer der Gruppe: Medizinische Fachperson zug\u00e4nglich <br \/><a href=\"https:\/\/medizinonline.com\/en\/my-account\/packages\/\">Jetzt beitreten<\/a><\/div><div class=\"pmpro_card_actions pmpro_font-medium\">Already a member? <a href=\"https:\/\/medizinonline.com\/en\/log-in\/?redirect_to=https%3A%2F%2Fmedizinonline.com%2Fen%2Fttr-stabilizers-gene-silencing-and-gene-scissors-where-do-we-stand%2F\">Log in here<\/a><\/div><\/div><\/div>","protected":false},"author":7,"featured_media":557184,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"pmpro_default_level":"","cat_1_feature_home_top":false,"cat_2_editor_pick":false,"csco_eyebrow_text":"Disease-modifying therapies for ATTR-CM","footnotes":""},"category":[11548,11359,11513,11396,11503],"tags":[94143,94144,94145,94141,94140,74876,74873,94142,74881],"powerkit_post_featured":[],"class_list":["post-557183","post","type-post","status-publish","format-standard","has-post-thumbnail","category-rx-en","category-cardiology","category-congress-reports","category-genetics","category-studies","tag-antisense-oligonucleotide-inhibitors","tag-attr-cm","tag-disease-modifying-therapies","tag-gene-scissors","tag-gene-silencing","tag-inotersen-en","tag-patisiran-en","tag-ttr-stabilizers","tag-vutrisiran-en","pmpro-level-required","pmpro-level-1","pmpro-no-access"],"acf":[],"publishpress_future_action":{"enabled":false,"date":"2026-06-26 23:58:52","action":"change-status","newStatus":"draft","terms":[],"taxonomy":"category","extraData":[]},"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"wpml_current_locale":"en_US","wpml_translations":{"fr_FR":{"locale":"fr_FR","id":557180,"slug":"stabilisateurs-ttr-mise-sous-silence-des-genes-et-ciseaux-genetiques-ou-en-sommes-nous","post_title":"Stabilisateurs TTR, mise sous silence des g\u00e8nes et ciseaux g\u00e9n\u00e9tiques : o\u00f9 en sommes-nous ?","href":"https:\/\/medizinonline.com\/fr\/stabilisateurs-ttr-mise-sous-silence-des-genes-et-ciseaux-genetiques-ou-en-sommes-nous\/"},"it_IT":{"locale":"it_IT","id":557177,"slug":"stabilizzatori-ttr-silenziamento-genico-e-forbici-geniche-a-che-punto-siamo","post_title":"Stabilizzatori TTR, silenziamento genico e forbici geniche: a che punto siamo?","href":"https:\/\/medizinonline.com\/it\/stabilizzatori-ttr-silenziamento-genico-e-forbici-geniche-a-che-punto-siamo\/"},"pt_PT":{"locale":"pt_PT","id":557174,"slug":"estabilizadores-de-ttr-silenciamento-de-genes-e-tesouras-de-genes-em-que-ponto-estamos","post_title":"Estabilizadores de TTR, silenciamento de genes e tesouras de genes: em que ponto estamos?","href":"https:\/\/medizinonline.com\/pt-pt\/estabilizadores-de-ttr-silenciamento-de-genes-e-tesouras-de-genes-em-que-ponto-estamos\/"},"es_ES":{"locale":"es_ES","id":557171,"slug":"estabilizadores-de-la-ttr-silenciamiento-y-tijeras-geneticas-en-que-punto-nos-encontramos","post_title":"Estabilizadores de la TTR, silenciamiento y tijeras gen\u00e9ticas: \u00bfen qu\u00e9 punto nos encontramos?","href":"https:\/\/medizinonline.com\/es\/estabilizadores-de-la-ttr-silenciamiento-y-tijeras-geneticas-en-que-punto-nos-encontramos\/"}},"_links":{"self":[{"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/posts\/557183","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/users\/7"}],"replies":[{"embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/comments?post=557183"}],"version-history":[{"count":1,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/posts\/557183\/revisions"}],"predecessor-version":[{"id":557185,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/posts\/557183\/revisions\/557185"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/media\/557184"}],"wp:attachment":[{"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/media?parent=557183"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/category?post=557183"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/tags?post=557183"},{"taxonomy":"powerkit_post_featured","embeddable":true,"href":"https:\/\/medizinonline.com\/en\/wp-json\/wp\/v2\/powerkit_post_featured?post=557183"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}