{"id":9413,"date":"2026-03-19T12:03:53","date_gmt":"2026-03-19T11:03:53","guid":{"rendered":"https:\/\/ern-ithaca.eu\/?post_type=news&#038;p=9413"},"modified":"2026-03-19T12:03:53","modified_gmt":"2026-03-19T11:03:53","slug":"new-mooc-launch-from-dna-to-cure-gene-and-gene-modified-cell-therapies-for-rare-diseases","status":"publish","type":"news","link":"https:\/\/ern-ithaca.eu\/it\/events-news\/news\/new-mooc-launch-from-dna-to-cure-gene-and-gene-modified-cell-therapies-for-rare-diseases\/","title":{"rendered":"Lancio di un nuovo MOOC: Dal DNA alla cura \u2013 Terapie geniche e con cellule geneticamente modificate per le malattie rare"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Con oltre 95% di malattie rare per le quali non esistono ancora trattamenti approvati, vi \u00e8 un\u2019urgente necessit\u00e0 di formazione specialistica sulle terapie di nuova generazione. Per colmare questa lacuna, la Fondazione per le Malattie Rare (FFRD) \u00e8 lieta di annunciare il lancio del suo nuovo <strong>Corso online aperto su larga scala (MOOC) su FutureLearn: \u201cDal DNA alla cura: terapie geniche e con cellule geneticamente modificate per le malattie rare\u201d.\u201d<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Coordinato da FFRD e realizzato con il sostegno dell\u2019ASGCT e della sovvenzione Pfizer Global Medical Education, questo corso riunisce esperti di fama internazionale per guidare i partecipanti alla scoperta delle tecnologie pi\u00f9 recenti che stanno rivoluzionando il panorama medico.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Tra gli argomenti principali figurano:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Modifica genetica: CRISPR-Cas9, base editing e prime editing<\/li>\n\n\n\n<li>Terapie a base di RNA: mRNA e oligonucleotidi antisenso (ASO)<\/li>\n\n\n\n<li>Sistemi di somministrazione: vettori virali e sistemi non virali<\/li>\n\n\n\n<li>Impatto nella pratica clinica: casi clinici relativi all\u2019emofilia e ai disturbi metabolici, accompagnati da toccanti testimonianze dei pazienti<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Le iscrizioni sono ora aperte: https:\/\/www.futurelearn.com\/courses\/from-dna-to-cure-gene-and-gene-modified-cell-therapies-for-rare-diseases<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">L'accesso ai contenuti \u00e8 gratuito per le prime 3 settimane dopo l'iscrizione. A<strong> sessione guidata <\/strong>si svolger\u00e0 dal 16 marzo all'8 maggio. Durante questo periodo, alcuni esperti saranno disponibili online per rispondere alle domande e fornire assistenza agli studenti in tempo reale.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Questo corso \u00e8 rivolto principalmente a<strong>\u00a0studenti universitari e ricercatori all\u2019inizio della carriera nei settori della biologia, delle scienze della vita e della medicina<\/strong>. Potrebbe inoltre risultare di interesse per gli operatori sanitari, i responsabili politici, i rappresentanti del settore, i difensori dei diritti dei pazienti e chiunque desideri comprendere meglio lo sviluppo delle terapie geniche e delle terapie cellulari modificate geneticamente per le malattie rare. Si raccomanda una conoscenza di base della biologia.<\/p>","protected":false},"excerpt":{"rendered":"<p>With over 95% of rare diseases still lacking approved treatments, there is a pressing need for specialized education in next-generation therapeutics. To address this gap, the Foundation for Rare Diseases (FFRD) is excited to announce the launch of their new Massive Open Online Course (MOOC) on FutureLearn: \u201cFrom DNA to Cure: Gene and Gene-Modified Cell [&hellip;]<\/p>\n","protected":false},"featured_media":0,"template":"","categories":[],"tags":[],"class_list":["post-9413","news","type-news","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.7 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>New MOOC Launch: From DNA to Cure \u2013 Gene and Gene-Modified Cell Therapies for Rare Diseases - ERN ITHACA<\/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:\/\/ern-ithaca.eu\/it\/events-news\/news\/new-mooc-launch-from-dna-to-cure-gene-and-gene-modified-cell-therapies-for-rare-diseases\/\" \/>\n<meta property=\"og:locale\" content=\"it_IT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"New MOOC Launch: From DNA to Cure \u2013 Gene and Gene-Modified Cell Therapies for Rare Diseases - ERN ITHACA\" \/>\n<meta property=\"og:description\" content=\"With over 95% of rare diseases still lacking approved treatments, there is a pressing need for specialized education in next-generation therapeutics. 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