{"id":2051,"date":"2024-11-12T02:09:14","date_gmt":"2024-11-12T01:09:14","guid":{"rendered":"https:\/\/rvh-synergie.org\/actualites\/une-nouvelle-recherche-soutient-la-premiere-therapie-genique-universelle-chez-lhumain-pour-lanemie-diamond-blackfan\/"},"modified":"2024-11-12T02:09:16","modified_gmt":"2024-11-12T01:09:16","slug":"une-nouvelle-recherche-soutient-la-premiere-therapie-genique-universelle-chez-lhumain-pour-lanemie-diamond-blackfan","status":"publish","type":"post","link":"https:\/\/rvh-synergie.org\/actualites\/une-nouvelle-recherche-soutient-la-premiere-therapie-genique-universelle-chez-lhumain-pour-lanemie-diamond-blackfan\/","title":{"rendered":"Une nouvelle recherche soutient la premi\u00e8re th\u00e9rapie g\u00e9nique universelle chez l\u2019humain pour l\u2019an\u00e9mie Diamond-Blackfan"},"content":{"rendered":"<div>\n<div class=\"article-gallery lightGallery\">\n<div data-thumb=\"https:\/\/scx1.b-cdn.net\/csz\/news\/tmb\/2024\/first-in-human-univers.jpg\" data-src=\"https:\/\/scx2.b-cdn.net\/gfx\/news\/2024\/first-in-human-univers.jpg\" data-sub-html=\"Credit: &lt;i&gt;Cell Stem Cell&lt;\/i&gt; (2024). DOI: 10.1016\/j.stem.2024.10.012\">\n<figure class=\"article-img\">\n<\/figure><\/div>\n<\/div>\n<p>L&rsquo;an\u00e9mie Diamond-Blackfan (DBA) est une maladie potentiellement mortelle qui peut provoquer une an\u00e9mie grave et d&rsquo;autres anomalies. Il s&rsquo;agit d&rsquo;une maladie g\u00e9n\u00e9tique rare du sang qui survient lorsque la moelle osseuse ne parvient pas \u00e0 produire des globules rouges.<\/p>\n<p>Actuellement, le seul rem\u00e8de est une greffe de cellules souches, mais celle-ci ne constitue une option viable que pour certains patients. Sinon, les enfants atteints de DBA n\u00e9cessitent des soins de suivi tout au long de leur vie pour g\u00e9rer les sympt\u00f4mes, tels que les st\u00e9ro\u00efdes et les transfusions sanguines.<\/p>\n<p>Une nouvelle \u00e9tude publi\u00e9e dans la revue <i>Cellule souche<\/i> apporte un soutien solide au premier essai de th\u00e9rapie g\u00e9nique universelle chez l&rsquo;homme pour le DBA via l&rsquo;expression r\u00e9gul\u00e9e de GATA1. Il s\u2019agit notamment de l\u2019un des premiers exemples de traitement de 30 mutations g\u00e9n\u00e9tiques diff\u00e9rentes avec un seul vecteur. Ce vecteur de qualit\u00e9 clinique est pr\u00eat et l\u2019\u00e9quipe esp\u00e8re le soumettre ensuite aux essais cliniques.<\/p>\n<p>                                        <!-- print only --><\/p><\/div>\n","protected":false},"excerpt":{"rendered":"<p>L&rsquo;an\u00e9mie Diamond-Blackfan (DBA) est une maladie potentiellement mortelle qui peut provoquer une an\u00e9mie grave et<\/p>\n","protected":false},"author":1,"featured_media":2052,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[86],"tags":[56,2402,1064,750,2401,244,8,932,499,2399,1063,47,2400],"class_list":["post-2051","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-les-maladies","tag-chez","tag-diamondblackfan","tag-genique","tag-lanemie","tag-lhumain","tag-nouvelle","tag-pour","tag-premiere","tag-recherche","tag-soutient","tag-therapie","tag-une","tag-universelle","generate-columns","tablet-grid-50","mobile-grid-100","grid-parent","grid-50","resize-featured-image"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v23.4 - 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