{"id":277,"date":"2021-09-06T22:14:05","date_gmt":"2021-09-06T14:14:05","guid":{"rendered":"http:\/\/phosphoramidite.cn\/en\/?p=277"},"modified":"2021-09-06T22:16:18","modified_gmt":"2021-09-06T14:16:18","slug":"optimizing-modified-mrna-in-vitro-synthesis-protocol-for-heart-gene-therapy","status":"publish","type":"post","link":"https:\/\/nugene.com.cn\/en\/index.php\/optimizing-modified-mrna-in-vitro-synthesis-protocol-for-heart-gene-therapy\/","title":{"rendered":"Optimizing Modified mRNA In Vitro Synthesis Protocol for Heart Gene Therapy"},"content":{"rendered":"\n<p>Synthetic modified RNA (modRNA) is a novel vector for gene transfer to the heart and other organs. modRNA can mediate<br>strong, transient protein expression with minimal induction of the innate immune response and risk for genome integration.<br>modRNA is already being used in several human clinical trials, and its use in basic and translational science is growing.<br>Due to the complexity of preparing modRNA and the high cost of its reagents, there is a need for an improved, cost-efficient<br>protocol to make modRNA. Here we show that changing the ratio between anti-reverse cap analog (ARCA) and<br>N1-methyl-pseudouridine (N1mJ), favoring ARCA over N1mJ, significantly increases the yield per reaction, improves<br>modRNA translation, and reduces its immunogenicity in vitro. This protocol will make modRNA preparation more accessible<br>and financially affordable for basic and translational research.<\/p>\n\n\n\n<p><strong>author<\/strong>: Yoav Hadas,Nishat Sultana, Elias Youssef, Mohammad Tofael Kabir Sharkar, Keerat Kaur, Elena Chepurko and Lior Zangi<\/p>\n\n\n<ul class=\"wp-block-latest-posts__list wp-block-latest-posts\"><li><a class=\"wp-block-latest-posts__post-title\" href=\"https:\/\/nugene.com.cn\/en\/index.php\/patisiran\/\">Patisiran<\/a><\/li>\n<li><a class=\"wp-block-latest-posts__post-title\" href=\"https:\/\/nugene.com.cn\/en\/index.php\/givosiran\/\">Givosiran<\/a><\/li>\n<li><a class=\"wp-block-latest-posts__post-title\" href=\"https:\/\/nugene.com.cn\/en\/index.php\/lumasiran-sodium\/\">Lumasiran sodium<\/a><\/li>\n<li><a class=\"wp-block-latest-posts__post-title\" href=\"https:\/\/nugene.com.cn\/en\/index.php\/inclisiran-sodium\/\">Inclisiran sodium<\/a><\/li>\n<li><a class=\"wp-block-latest-posts__post-title\" href=\"https:\/\/nugene.com.cn\/en\/index.php\/vutrisiran\/\">Vutrisiran<\/a><\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>Synthetic modified RNA (modRNA) is a novel vector for gene transfer to the heart and other organs. modRNA can mediatestrong, transient protein expression with minimal induction of the innate immune response and risk for genome integration.modRNA is already being used in several human clinical trials, and its use in basic and translational science is growing.Due &hellip;<\/p>\n<p class=\"read-more\"> <a class=\"\" href=\"https:\/\/nugene.com.cn\/en\/index.php\/optimizing-modified-mrna-in-vitro-synthesis-protocol-for-heart-gene-therapy\/\"> <span class=\"screen-reader-text\">Optimizing Modified mRNA In Vitro Synthesis Protocol for Heart Gene Therapy<\/span> Read More &raquo;<\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"default","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","theme-transparent-header-meta":"default","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","footnotes":""},"categories":[6,9],"tags":[],"class_list":["post-277","post","type-post","status-publish","format-standard","hentry","category-news","category-rd"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v21.1 - 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