{"id":9645,"date":"2026-04-20T11:12:47","date_gmt":"2026-04-20T09:12:47","guid":{"rendered":"https:\/\/ern-ithaca.eu\/?post_type=news&#038;p=9645"},"modified":"2026-04-20T11:12:47","modified_gmt":"2026-04-20T09:12:47","slug":"new-mooc-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-from-dna-to-cure-gene-and-gene-modified-cell-therapies-for-rare-diseases\/","title":{"rendered":"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, il\u00a0<strong>Fondazione per le Malattie Rare (FFRD)<\/strong>\u00a0sta lanciando il suo nuovo corso online aperto e di massa (MOOC) su\u00a0<strong>FutureLearn<\/strong>:\u00a0<strong>\u201cDal DNA alla cura: terapie geniche e con cellule geneticamente modificate per le malattie rare.\u201d<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Coordinato da FFRD e realizzato con il sostegno del&nbsp;<strong>Sovvenzione ASGCT e Pfizer per la formazione medica globale<\/strong>, questo corso riunisce&nbsp;<strong>esperti riconosciuti a livello internazionale<\/strong>&nbsp;per guidare i partecipanti alla scoperta delle ultime tecnologie che stanno rivoluzionando il panorama medico.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Tra gli argomenti principali figurano:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Modifica genetica:<\/strong>\u00a0CRISPR-Cas9, base editing e prime editing<\/li>\n\n\n\n<li><strong>Terapie a base di RNA:<\/strong>\u00a0mRNA e oligonucleotidi antisenso (ASO)<\/li>\n\n\n\n<li><strong>Sistemi di somministrazione:<\/strong>\u00a0Vettori virali e sistemi non virali<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Impatto nel mondo reale:<\/strong>\u00a0Casi 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\"><strong>Le iscrizioni sono ora aperte:\u00a0<\/strong><a href=\"https:\/\/www.futurelearn.com\/courses\/from-dna-to-cure-gene-and-gene-modified-cell-therapies-for-rare-diseases\" target=\"_blank\" rel=\"noreferrer noopener\">https:\/\/www.futurelearn.com\/courses\/from-dna-to-cure-gene-and-gene-modified-cell-therapies-for-rare-diseases<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><br>L'accesso ai contenuti \u00e8\u00a0<strong>gratuito per le prime 3 settimane dopo l'iscrizione<\/strong>. Una sessione guidata si svolge dalle\u00a0<strong>Dal 16 marzo all'8 maggio<\/strong>. Durante questo periodo, gli esperti saranno disponibili online per rispondere alle domande e fornire assistenza agli studenti in tempo reale.<br><br>Questo corso \u00e8 rivolto principalmente a\u00a0<strong>studenti universitari e ricercatori all\u2019inizio della carriera<\/strong>\u00a0nel campo della biologia, delle scienze della vita e della medicina. Potrebbe inoltre essere di interesse per\u00a0<strong>operatori sanitari, responsabili politici, rappresentanti del settore, difensori dei diritti dei pazienti<\/strong>, e chiunque desideri comprendere meglio lo sviluppo delle terapie geniche e delle terapie cellulari modificate geneticamente per le malattie rare. A\u00a0<strong>conoscenze di base di biologia<\/strong>\u00a0\u00e8 consigliabile.<\/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\u00a0Foundation for Rare Diseases (FFRD)\u00a0is launching its new Massive Open Online Course (MOOC) on\u00a0FutureLearn:\u00a0\u201cFrom DNA to Cure: Gene and Gene-Modified Cell Therapies for Rare Diseases.\u201d Coordinated by FFRD and developed [&hellip;]<\/p>\n","protected":false},"featured_media":0,"template":"","categories":[],"tags":[],"class_list":["post-9645","news","type-news","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>New MOOC: 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-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: 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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