{"id":12401,"date":"2021-03-11T21:08:40","date_gmt":"2021-03-12T02:08:40","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?p=12401"},"modified":"2022-11-14T17:12:14","modified_gmt":"2022-11-14T22:12:14","slug":"exciting-breakthroughs-in-rna-therapeutics-for-progeria","status":"publish","type":"post","link":"https:\/\/www.progeriaresearch.org\/it\/2021\/03\/11\/exciting-breakthroughs-in-rna-therapeutics-for-progeria\/","title":{"rendered":"Emozionanti scoperte nella terapia a base di RNA per la progeria!"},"content":{"rendered":"<p>[et_pb_section fb_built=&#8221;1&#8243; admin_label=&#8221;section&#8221; _builder_version=&#8221;4.16&#8243; global_colors_info=&#8221;{}&#8221;][et_pb_row admin_label=&#8221;row&#8221; _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.18.1&#8243; _module_preset=&#8221;default&#8221; hover_enabled=&#8221;0&#8243; global_colors_info=&#8221;{}&#8221; sticky_enabled=&#8221;0&#8243;]<\/p>\n<p><img loading=\"lazy\" decoding=\"async\" class=\"wp-image-12360 alignnone size-full aligncenter\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2021\/03\/Carlos.jpg\" alt=\"\" width=\"150\" height=\"300\" \/><\/p>\n<p>We\u2019re thrilled to share the results from<strong> two very exciting breakthrough studies on the use of RNA therapeutics<\/strong> in Progeria research.\u00a0 Both studies were co-funded by The Progeria Research Foundation (PRF) and co-authored by PRF\u2019s Medical Director, Dr. Leslie Gordon.<\/p>\n<p>Progerin is the disease-causing protein in Progeria. The RNA therapies interfere with the body\u2019s ability to produce progerin, by blocking its production at the RNA level.\u00a0 This means that<strong> the treatment is more specific than most therapies<\/strong> that target progerin at the protein level.<\/p>\n<p>Though each study used a different drug delivery system, both studies targeted the same basic treatment strategy, inhibiting production of RNA coding for the abnormal protein, progerin. Both were led by researchers at the National Institutes of Health (NIH), and were published today in the journal <em>Nature Medicine<\/em>.<\/p>\n<p><a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01274-0\">One study<\/a>, led by Francis Collins, MD, PhD, Director of the NIH, showed that treating Progeria mice with a drug named SRP2001 r<strong>educed the harmful progerin mRNA and protein expression in the aorta<\/strong>, the main artery in the body, as well as in other tissues. At the end of the study, the aortic wall remained stronger and the mice demonstrated an <strong>increased survival of over 60%<\/strong>.<\/p>\n<p>\u201cTo have a targeted RNA-therapy show such significant results in an animal model gives me hope that this could lead to a major advance for the treatment of progeria,\u201d said Collins.<\/p>\n<p>The <a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01262-4\">other study<\/a>, led by Tom Misteli, PhD, Director of the Center for Cancer Research, National Cancer Institute, NIH, showed a <strong>90 \u2013 95% reduction of the toxic progerin-producing RNA<\/strong> in different tissues after treatment with a drug called LB143. Misteli\u2019s lab found that progerin protein reduction was most effective in the liver, with additional improvements in the heart and aorta.<\/p>\n<p>We now know there are multiple ways to decrease production of the harmful progerin protein using RNA therapeutics. Each study found different stretches of RNA in the mouse models that, when targeted, delivered an effective pathway for treatment, resulting in <strong>Progeria mice that lived much longer than those treated in previous studies with lonafarnib<\/strong>, the only FDA-approved drug for children with Progeria. Furthermore, researchers found that a combination treatment with RNA therapeutics and lonafarnib reduced progerin protein levels in liver and heart more effectively than either single treatment on its own.<\/p>\n<p>\u201cThese two highly important studies demonstrate the <strong>major advancements that are now upon us<\/strong> in the field of targeted Progeria therapeutics,\u201d said PRF Medical Director, Dr. Leslie Gordon. \u201cI was thrilled to work with these brilliant research groups to advance RNA therapy for children with Progeria. Both are exciting proof-of-principle studies, and <strong>PRF is excited to forge ahead towards clinical trials<\/strong> that apply these treatment strategies.<\/p>\n<p>&nbsp;<\/p>\n<p>&#8212;<\/p>\n<p>Erdos, M.R., Cabral, W.A., Tavarez, U.L.\u00a0<i>et al.<\/i>\u00a0A targeted antisense therapeutic approach for Hutchinson\u2013Gilford progeria syndrome.\u00a0<i>Nat Med<\/i>\u00a0(2021). https:\/\/doi.org\/10.1038\/s41591-021-01274-0<\/p>\n<p>Puttaraju, M., Jackson, M., Klein, S.\u00a0<i>et al.<\/i>\u00a0Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson\u2013Gilford progeria syndrome.\u00a0<i>Nat Med<\/i>\u00a0(2021). https:\/\/doi.org\/10.1038\/s41591-021-01262-4<\/p>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Siamo entusiasti di condividere i risultati di due entusiasmanti studi innovativi sull&#039;uso della terapia a base di RNA nella ricerca sulla Progeria. Entrambi gli studi sono stati cofinanziati dalla Progeria Research Foundation (PRF) e co-redatti dal Direttore medico della PRF, il dott. Leslie Gordon. <\/p>","protected":false},"author":2,"featured_media":12414,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[2,1],"tags":[],"class_list":["post-12401","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","category-uncategorized"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Exciting breakthroughs in RNA Therapeutics for Progeria! | The Progeria Research Foundation<\/title>\n<meta name=\"description\" content=\"Two very exciting breakthrough studies on the use of RNA therapeutics in Progeria research.\u00a0 Both studies were co-funded by The Progeria Research Foundation (PRF) and co-authored by PRF\u2019s Medical Director, Dr. Leslie Gordon.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.progeriaresearch.org\/it\/2021\/03\/11\/exciting-breakthroughs-in-rna-therapeutics-for-progeria\/\" \/>\n<meta property=\"og:locale\" content=\"it_IT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Exciting breakthroughs in RNA Therapeutics for Progeria! | The Progeria Research Foundation\" \/>\n<meta property=\"og:description\" content=\"Two very exciting breakthrough studies on the use of RNA therapeutics in Progeria research.\u00a0 Both studies were co-funded by The Progeria Research Foundation (PRF) and co-authored by PRF\u2019s Medical Director, Dr. Leslie Gordon.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.progeriaresearch.org\/it\/2021\/03\/11\/exciting-breakthroughs-in-rna-therapeutics-for-progeria\/\" \/>\n<meta property=\"og:site_name\" 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use of RNA therapeutics in Progeria research.\u00a0 Both studies were co-funded by The Progeria Research Foundation (PRF) and co-authored by PRF\u2019s Medical Director, Dr. Leslie Gordon.\" \/>\n<meta name=\"twitter:image\" content=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2021\/03\/Carlos-with-crocheted-sloth-cropped.jpg\" \/>\n<meta name=\"twitter:creator\" content=\"@Progeria\" \/>\n<meta name=\"twitter:site\" content=\"@Progeria\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Karen Betournay\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"3 minuti\" \/>\n<script type=\"application\/ld+json\" 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