{"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\/id\/2021\/03\/11\/exciting-breakthroughs-in-rna-therapeutics-for-progeria\/","title":{"rendered":"Terobosan menarik dalam Terapi RNA untuk 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>Kami sangat senang untuk berbagi hasil dari<strong> dua studi terobosan yang sangat menarik tentang penggunaan terapi RNA<\/strong> dalam penelitian Progeria. Kedua penelitian tersebut didanai bersama oleh The Progeria Research Foundation (PRF) dan ditulis bersama oleh Direktur Medis PRF, Dr. Leslie Gordon.<\/p>\n<p>Progerin adalah protein penyebab penyakit pada Progeria. Terapi RNA mengganggu kemampuan tubuh untuk memproduksi progerin, dengan menghalangi produksinya pada tingkat RNA. Ini berarti bahwa<strong> Perawatannya lebih spesifik dibandingkan kebanyakan terapi lainnya<\/strong> yang menargetkan progerin pada tingkat protein.<\/p>\n<p>Meskipun setiap penelitian menggunakan sistem pemberian obat yang berbeda, kedua penelitian tersebut menargetkan strategi pengobatan dasar yang sama, yaitu menghambat produksi RNA yang mengkode protein abnormal, progerin. Keduanya dipimpin oleh para peneliti di National Institutes of Health (NIH), dan diterbitkan hari ini di jurnal <em>Obat Alami<\/em>.<\/p>\n<p><a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01274-0\">Satu studi<\/a>, yang dipimpin oleh Francis Collins, MD, PhD, Direktur NIH, menunjukkan bahwa mengobati tikus Progeria dengan obat bernama SRP2001 r<strong>mengurangi ekspresi mRNA dan protein progerin yang berbahaya di aorta<\/strong>, arteri utama dalam tubuh, serta jaringan lainnya. Pada akhir penelitian, dinding aorta tetap kuat dan tikus menunjukkan <strong>peningkatan kelangsungan hidup lebih dari 60%<\/strong>.<\/p>\n<p>\u201cAgar terapi RNA yang ditargetkan dapat menunjukkan hasil yang signifikan pada model hewan, saya berharap ini dapat menghasilkan kemajuan besar dalam pengobatan progeria,\u201d kata Collins.<\/p>\n<p>Itu <a href=\"https:\/\/www.nature.com\/articles\/s41591-021-01262-4\">studi lainnya<\/a>, yang dipimpin oleh Tom Misteli, PhD, Direktur Pusat Penelitian Kanker, Institut Kanker Nasional, NIH, menunjukkan <strong>90 \u2013 95% pengurangan RNA penghasil progerin yang beracun<\/strong> di jaringan yang berbeda setelah perawatan dengan obat yang disebut LB143. Laboratorium Misteli menemukan bahwa pengurangan protein progerin paling efektif di hati, dengan perbaikan tambahan di jantung dan aorta.<\/p>\n<p>Kini kita tahu ada beberapa cara untuk mengurangi produksi protein progerin yang berbahaya menggunakan terapi RNA. Setiap penelitian menemukan bentangan RNA yang berbeda pada model tikus yang, ketika ditargetkan, menghasilkan jalur pengobatan yang efektif, yang menghasilkan <strong>Tikus progeria yang hidup lebih lama dibandingkan tikus yang diobati dengan lonafarnib dalam penelitian sebelumnya<\/strong>, satu-satunya obat yang disetujui FDA untuk anak-anak penderita Progeria. Lebih jauh, para peneliti menemukan bahwa pengobatan kombinasi dengan terapi RNA dan lonafarnib mengurangi kadar protein progerin di hati dan jantung secara lebih efektif daripada pengobatan tunggal saja.<\/p>\n<p>\u201cKedua studi yang sangat penting ini menunjukkan <strong>kemajuan besar yang kini telah kita lihat<\/strong> di bidang terapi Progeria yang ditargetkan,\u201d kata Direktur Medis PRF, Dr. Leslie Gordon. \u201cSaya sangat senang bekerja dengan kelompok penelitian yang brilian ini untuk memajukan terapi RNA bagi anak-anak penderita Progeria. Keduanya merupakan studi pembuktian prinsip yang menarik, dan <strong>PRF bersemangat untuk terus maju menuju uji klinis<\/strong> yang menerapkan strategi perawatan ini.<\/p>\n<p>&nbsp;<\/p>\n<p>\u2014<\/p>\n<p>Erdos, MR, Cabral, WA, Tavarez, UL\u00a0<i>dan lain-lain.<\/i>\u00a0Pendekatan terapi antisense yang ditargetkan untuk sindrom progeria Hutchinson\u2013Gilford.\u00a0<i>Kedokteran Nat<\/i>\u00a0(2021). https:\/\/doi.org\/10.1038\/s41591-021-01274-0<\/p>\n<p>Puttaraju, M., Jackson, M., Klein, S.\u00a0<i>dan lain-lain.<\/i>\u00a0Skrining sistematis mengidentifikasi oligonukleotida antisense terapeutik untuk sindrom progeria Hutchinson\u2013Gilford.\u00a0<i>Kedokteran Nat<\/i>\u00a0(2021). https:\/\/doi.org\/10.1038\/s41591-021-01262-4<\/p>\n<p>[\/et_pb_teks][\/et_pb_kolom][\/et_pb_baris][\/et_pb_bagian]<\/p>","protected":false},"excerpt":{"rendered":"<p>Kami sangat gembira dapat berbagi hasil dari dua studi terobosan yang sangat menarik tentang penggunaan terapi RNA dalam penelitian Progeria. Kedua studi tersebut didanai bersama oleh The Progeria Research Foundation (PRF) dan ditulis bersama oleh Direktur Medis 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\/id\/2021\/03\/11\/exciting-breakthroughs-in-rna-therapeutics-for-progeria\/\" \/>\n<meta property=\"og:locale\" content=\"id_ID\" \/>\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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