{"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\/pt\/2021\/03\/11\/exciting-breakthroughs-in-rna-therapeutics-for-progeria\/","title":{"rendered":"Avan\u00e7os emocionantes na terap\u00eautica de RNA para Prog\u00e9ria!"},"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>Estamos entusiasmados em compartilhar os resultados de<strong> dois estudos inovadores muito interessantes sobre o uso de terapias de RNA<\/strong> na pesquisa sobre Progeria. Ambos os estudos foram cofinanciados pela The Progeria Research Foundation (PRF) e coautorados pela Diretora M\u00e9dica da PRF, Dra. Leslie Gordon.<\/p>\n<p>Progerina \u00e9 a prote\u00edna causadora da doen\u00e7a na Progeria. As terapias de RNA interferem na capacidade do corpo de produzir progerina, bloqueando sua produ\u00e7\u00e3o no n\u00edvel de RNA. Isso significa que<strong> o tratamento \u00e9 mais espec\u00edfico do que a maioria das terapias<\/strong> que t\u00eam como alvo a progerina no n\u00edvel proteico.<\/p>\n<p>Embora cada estudo tenha usado um sistema de administra\u00e7\u00e3o de medicamentos diferente, ambos os estudos tiveram como alvo a mesma estrat\u00e9gia b\u00e1sica de tratamento, inibindo a produ\u00e7\u00e3o de RNA codificador da prote\u00edna anormal, progerina. Ambos foram liderados por pesquisadores do National Institutes of Health (NIH) e foram publicados hoje no peri\u00f3dico <em>Medicina Natural<\/em>.<\/p>\n<p><a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01274-0\">Um estudo<\/a>, liderado por Francis Collins, MD, PhD, Diretor do NIH, mostrou que o tratamento de camundongos Progeria com um medicamento chamado SRP2001 r<strong>reduziu a express\u00e3o prejudicial do mRNA e da prote\u00edna da progerina na aorta<\/strong>, a principal art\u00e9ria do corpo, assim como de outros tecidos. Ao final do estudo, a parede a\u00f3rtica permaneceu mais forte e os camundongos demonstraram uma <strong>aumento da sobreviv\u00eancia de mais de 60%<\/strong>.<\/p>\n<p>\u201cTer uma terapia de RNA direcionada mostrando resultados t\u00e3o significativos em um modelo animal me d\u00e1 esperan\u00e7a de que isso possa levar a um grande avan\u00e7o no tratamento da prog\u00e9ria\u201d, disse Collins.<\/p>\n<p>O <a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01262-4\">outro estudo<\/a>, liderado por Tom Misteli, PhD, Diretor do Centro de Pesquisa do C\u00e2ncer, Instituto Nacional do C\u00e2ncer, NIH, mostrou uma <strong>90 \u2013 95% redu\u00e7\u00e3o do RNA t\u00f3xico produtor de progerina<\/strong> em diferentes tecidos ap\u00f3s tratamento com um medicamento chamado LB143. O laborat\u00f3rio de Misteli descobriu que a redu\u00e7\u00e3o da prote\u00edna progerina foi mais eficaz no f\u00edgado, com melhorias adicionais no cora\u00e7\u00e3o e na aorta.<\/p>\n<p>Agora sabemos que h\u00e1 v\u00e1rias maneiras de diminuir a produ\u00e7\u00e3o da prote\u00edna progerina prejudicial usando terapias de RNA. Cada estudo encontrou diferentes trechos de RNA nos modelos de camundongos que, quando direcionados, forneceram um caminho eficaz para o tratamento, resultando em <strong>Camundongos com progeria viveram muito mais do que aqueles tratados em estudos anteriores com lonafarnib<\/strong>, o \u00fanico medicamento aprovado pela FDA para crian\u00e7as com Progeria. Al\u00e9m disso, os pesquisadores descobriram que um tratamento combinado com terap\u00eauticas de RNA e lonafarnib reduziu os n\u00edveis de prote\u00edna progerina no f\u00edgado e no cora\u00e7\u00e3o de forma mais eficaz do que qualquer tratamento isolado.<\/p>\n<p>\u201cEstes dois estudos de grande import\u00e2ncia demonstram a <strong>grandes avan\u00e7os que agora est\u00e3o sobre n\u00f3s<\/strong> no campo da terap\u00eautica direcionada para Progeria\u201d, disse a Diretora M\u00e9dica da PRF, Dra. Leslie Gordon. \u201cFiquei emocionada em trabalhar com esses grupos de pesquisa brilhantes para avan\u00e7ar a terapia de RNA para crian\u00e7as com Progeria. Ambos s\u00e3o estudos de prova de princ\u00edpio empolgantes e <strong>A PRF est\u00e1 animada para avan\u00e7ar em dire\u00e7\u00e3o aos ensaios cl\u00ednicos<\/strong> que aplicam essas estrat\u00e9gias de tratamento.<\/p>\n<p>&nbsp;<\/p>\n<p>\u2014<\/p>\n<p>Erdos, MR, Cabral, WA, Tavarez, UL\u00a0<i>e outros.<\/i>\u00a0Uma abordagem terap\u00eautica antisense direcionada para a s\u00edndrome de progeria de Hutchinson-Gilford.\u00a0<i>NatM\u00e9dico<\/i>\u00a0(2021). https:\/\/doi.org\/10.1038\/s41591-021-01274-0<\/p>\n<p>Puttaraju, M., Jackson, M., Klein, S.\u00a0<i>e outros.<\/i>\u00a0A triagem sistem\u00e1tica identifica oligonucleot\u00eddeos antisense terap\u00eauticos para a s\u00edndrome de progeria de Hutchinson-Gilford.\u00a0<i>NatM\u00e9dico<\/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>Estamos entusiasmados em compartilhar os resultados de dois estudos inovadores muito empolgantes sobre o uso de terap\u00eauticas de RNA na pesquisa da Progeria. Ambos os estudos foram cofinanciados pela The Progeria Research Foundation (PRF) e coautorados pela Diretora M\u00e9dica da PRF, Dra. 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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