{"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>Siamo entusiasti di condividere i risultati di<strong> due studi innovativi molto interessanti sull&#039;uso della terapia a base di RNA<\/strong> 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>\n<p>La progerina \u00e8 la proteina che causa la malattia nella Progeria. Le terapie a base di RNA interferiscono con la capacit\u00e0 del corpo di produrre progerina, bloccandone la produzione a livello di RNA. Ci\u00f2 significa che<strong> il trattamento \u00e8 pi\u00f9 specifico della maggior parte delle terapie<\/strong> che prendono di mira la progerina a livello proteico.<\/p>\n<p>Sebbene ogni studio abbia utilizzato un sistema di somministrazione del farmaco diverso, entrambi gli studi hanno mirato alla stessa strategia di trattamento di base, inibendo la produzione di RNA che codifica per la proteina anomala, la progerina. Entrambi sono stati condotti da ricercatori del National Institutes of Health (NIH) e sono stati pubblicati oggi sulla rivista <em>Medicina della Natura<\/em>.<\/p>\n<p><a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01274-0\">Uno studio<\/a>, guidato da Francis Collins, MD, PhD, Direttore del NIH, ha dimostrato che il trattamento dei topi Progeria con un farmaco denominato SRP2001 r<strong>ha ridotto l&#039;espressione dannosa dell&#039;mRNA e delle proteine della progerina nell&#039;aorta<\/strong>, l&#039;arteria principale del corpo, cos\u00ec come in altri tessuti. Alla fine dello studio, la parete aortica \u00e8 rimasta pi\u00f9 forte e i topi hanno dimostrato un <strong>aumento della sopravvivenza di oltre 60%<\/strong>.<\/p>\n<p>&quot;Il fatto che una terapia a RNA mirata abbia mostrato risultati cos\u00ec significativi in un modello animale mi d\u00e0 la speranza che ci\u00f2 possa portare a un importante progresso nel trattamento della progeria&quot;, ha affermato Collins.<\/p>\n<p>IL <a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01262-4\">altro studio<\/a>, guidato da Tom Misteli, PhD, Direttore del Centro per la ricerca sul cancro, National Cancer Institute, NIH, ha mostrato un <strong>90 \u2013 95% riduzione dell\u2019RNA tossico produttore di progerina<\/strong> in tessuti diversi dopo il trattamento con un farmaco chiamato LB143. Il laboratorio di Misteli ha scoperto che la riduzione della proteina progerina era pi\u00f9 efficace nel fegato, con ulteriori miglioramenti nel cuore e nell&#039;aorta.<\/p>\n<p>Ora sappiamo che ci sono diversi modi per ridurre la produzione della proteina progerina nociva usando terapie a base di RNA. Ogni studio ha trovato diversi tratti di RNA nei modelli di topi che, quando presi di mira, hanno fornito un percorso efficace per il trattamento, con conseguente <strong>Topi progeria che vivono molto pi\u00f9 a lungo di quelli trattati in studi precedenti con lonafarnib<\/strong>, l&#039;unico farmaco approvato dalla FDA per i bambini affetti da Progeria. Inoltre, i ricercatori hanno scoperto che un trattamento combinato con RNA therapeutics e lonafarnib ha ridotto i livelli di proteina progerina nel fegato e nel cuore in modo pi\u00f9 efficace rispetto a entrambi i trattamenti singoli presi singolarmente.<\/p>\n<p>\u201cQuesti due studi di grande importanza dimostrano la <strong>grandi progressi che sono ormai su di noi<\/strong> nel campo della terapia mirata alla Progeria&quot;, ha affermato il direttore medico della PRF, la dott. ssa Leslie Gordon. &quot;Ero entusiasta di lavorare con questi brillanti gruppi di ricerca per far progredire la terapia a RNA per i bambini affetti da Progeria. Entrambi sono entusiasmanti studi di prova di principio e <strong>PRF \u00e8 entusiasta di procedere verso le sperimentazioni cliniche<\/strong> che applicano queste strategie di trattamento.<\/p>\n<p>&nbsp;<\/p>\n<p>\u2014<\/p>\n<p>Erdos, MR, Cabral, WA, Tavarez, UL\u00a0<i>e altri<\/i>\u00a0Un approccio terapeutico antisenso mirato per la sindrome di progeria di Hutchinson-Gilford.\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>e altri<\/i>\u00a0Lo screening sistematico identifica gli oligonucleotidi antisenso terapeutici per la sindrome di progeria di Hutchinson-Gilford.\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>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 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 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