{"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\/es\/events-news\/news\/new-mooc-launch-from-dna-to-cure-gene-and-gene-modified-cell-therapies-for-rare-diseases\/","title":{"rendered":"New MOOC Launch: From DNA to Cure \u2013 Gene and Gene-Modified Cell Therapies for Rare Diseases"},"content":{"rendered":"<p class=\"wp-block-paragraph\">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 <strong>Massive Open Online Course (MOOC) on FutureLearn: \u201cFrom DNA to Cure: Gene and Gene-Modified Cell Therapies for Rare Diseases.\u201d<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Coordinated by FFRD and developed with support from the&nbsp;ASGCT &amp; Pfizer Global Medical Education Grant, this course brings together&nbsp;internationally recognized experts&nbsp;to guide participants through the latest technologies reshaping the medical landscape.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Key topics include:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Gene Editing:\u00a0CRISPR-Cas9, base editing, and prime editing<\/li>\n\n\n\n<li>RNA Therapeutics:\u00a0mRNA and Antisense Oligonucleotides (ASOs)<\/li>\n\n\n\n<li>Delivery Systems:\u00a0Viral vectors and non-viral systems<\/li>\n\n\n\n<li>Real-World Impact:\u00a0Clinical case studies in hemophilia and metabolic disorders, alongside moving patient testimonies<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Enrollment is open now:&nbsp;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\">Access to the content is\u00a0free for the first 3 weeks after enrollment. A<strong> facilitated session <\/strong>runs from\u00a016 March to 8 May. During this time, experts will be online to answer questions and support learners in real time.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">This course is primarily designed for<strong>\u00a0university students and early-career researchers\u00a0in biology, life sciences, and medicine<\/strong>. It may also be of interest to\u00a0healthcare professionals, policy makers, industry representatives, patient advocates, and anyone seeking to better understand the development of gene and gene-modified cell therapies for rare diseases. A\u00a0basic background in biology\u00a0is recommended.<\/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\/es\/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=\"es_ES\" \/>\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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