{"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\/nl\/2021\/03\/11\/exciting-breakthroughs-in-rna-therapeutics-for-progeria\/","title":{"rendered":"Spannende doorbraken in RNA-therapie voor 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 zijn verheugd om de resultaten van<strong> twee zeer opwindende baanbrekende studies over het gebruik van RNA-therapie\u00ebn<\/strong> in Progeria-onderzoek. Beide studies werden medegefinancierd door The Progeria Research Foundation (PRF) en mede geschreven door PRF&#039;s medisch directeur, Dr. Leslie Gordon.<\/p>\n<p>Progerine is het ziekteverwekkende eiwit in Progeria. De RNA-therapie\u00ebn verstoren het vermogen van het lichaam om progerine te produceren, door de productie ervan op RNA-niveau te blokkeren. Dit betekent dat<strong> de behandeling is specifieker dan de meeste therapie\u00ebn<\/strong> die progerine op prote\u00efneniveau aanpakken.<\/p>\n<p>Hoewel elke studie een ander medicijnafgiftesysteem gebruikte, richtten beide studies zich op dezelfde basisbehandelingsstrategie, namelijk het remmen van de productie van RNA dat codeert voor het abnormale eiwit, progerine. Beide werden geleid door onderzoekers van de National Institutes of Health (NIH) en werden vandaag gepubliceerd in het tijdschrift <em>Natuurgeneeskunde<\/em>.<\/p>\n<p><a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01274-0\">E\u00e9n studie<\/a>, onder leiding van Francis Collins, MD, PhD, directeur van het NIH, toonde aan dat de behandeling van Progeria-muizen met een medicijn genaamd SRP2001 r<strong>verminderde de schadelijke progerine mRNA- en prote\u00efne-expressie in de aorta<\/strong>, de belangrijkste slagader in het lichaam, en ook in andere weefsels. Aan het einde van de studie bleef de aortawand sterker en vertoonden de muizen een <strong>verhoogde overleving van meer dan 60%<\/strong>.<\/p>\n<p>&quot;Dat een gerichte RNA-therapie zulke significante resultaten laat zien in een diermodel geeft mij hoop dat dit kan leiden tot een grote vooruitgang in de behandeling van progeria&quot;, aldus Collins.<\/p>\n<p>De <a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01262-4\">andere studie<\/a>, onder leiding van Tom Misteli, PhD, directeur van het Center for Cancer Research, National Cancer Institute, NIH, toonde een <strong>90 \u2013 95% reductie van het toxische progerine-producerende RNA<\/strong> in verschillende weefsels na behandeling met een medicijn genaamd LB143. Misteli&#039;s lab ontdekte dat progerine-eiwitreductie het meest effectief was in de lever, met aanvullende verbeteringen in het hart en de aorta.<\/p>\n<p>We weten nu dat er meerdere manieren zijn om de productie van het schadelijke progerine-eiwit te verminderen met behulp van RNA-therapie\u00ebn. Elke studie vond verschillende stukken RNA in de muismodellen die, wanneer gericht, een effectief pad voor behandeling opleverden, wat resulteerde in <strong>Progeria-muizen die veel langer leefden dan de muizen die in eerdere onderzoeken met lonafarnib werden behandeld<\/strong>, het enige door de FDA goedgekeurde medicijn voor kinderen met Progeria. Bovendien ontdekten onderzoekers dat een combinatiebehandeling met RNA-therapie\u00ebn en lonafarnib de progerine-eiwitniveaus in de lever en het hart effectiever verlaagde dan beide behandelingen afzonderlijk.<\/p>\n<p>\u201cDeze twee uiterst belangrijke onderzoeken tonen aan dat <strong>grote ontwikkelingen die nu voor ons liggen<\/strong> op het gebied van gerichte Progeria-therapie\u00ebn,\u201d zei PRF medisch directeur, Dr. Leslie Gordon. \u201cIk was verheugd om met deze briljante onderzoeksgroepen samen te werken om RNA-therapie voor kinderen met Progeria te verbeteren. Beide zijn opwindende proof-of-principle-studies en <strong>PRF is enthousiast om door te gaan met klinische proeven<\/strong> die deze behandelingsstrategie\u00ebn toepassen.<\/p>\n<p>&nbsp;<\/p>\n<p>\u2014<\/p>\n<p>Erdos, MR, Cabral, WA, Tavarez, UL\u00a0<i>et al.<\/i>\u00a0Een gerichte antisense therapeutische aanpak voor het Hutchinson-Gilford progeriasyndroom.\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>\u00a0Systematische screening identificeert therapeutische antisense-oligonucleotiden voor het Hutchinson-Gilford-progeriasyndroom.\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>","protected":false},"excerpt":{"rendered":"<p>We zijn verheugd om de resultaten te delen van twee zeer opwindende baanbrekende studies over het gebruik van RNA-therapie\u00ebn in Progeria-onderzoek. Beide studies werden medegefinancierd door The Progeria Research Foundation (PRF) en mede geschreven door de medisch directeur van PRF, Dr. 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 v26.8 - 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\/nl\/2021\/03\/11\/exciting-breakthroughs-in-rna-therapeutics-for-progeria\/\" \/>\n<meta property=\"og:locale\" content=\"nl_NL\" \/>\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\" 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