{"id":2186,"date":"2024-11-16T22:01:21","date_gmt":"2024-11-16T21:01:21","guid":{"rendered":"https:\/\/rvh-synergie.org\/actualites\/une-therapie-genique-experimentale-alternative-retablit-laudition-et-ameliore-la-vision-lors-de-tests\/"},"modified":"2024-11-16T22:01:22","modified_gmt":"2024-11-16T21:01:22","slug":"une-therapie-genique-experimentale-alternative-retablit-laudition-et-ameliore-la-vision-lors-de-tests","status":"publish","type":"post","link":"https:\/\/rvh-synergie.org\/actualites\/une-therapie-genique-experimentale-alternative-retablit-laudition-et-ameliore-la-vision-lors-de-tests\/","title":{"rendered":"Une th\u00e9rapie g\u00e9nique exp\u00e9rimentale alternative r\u00e9tablit l&rsquo;audition et am\u00e9liore la vision lors de tests"},"content":{"rendered":"<div>\n<div class=\"article-gallery lightGallery\">\n<div data-thumb=\"https:\/\/scx1.b-cdn.net\/csz\/news\/tmb\/2024\/new-method-restores-he.jpg\" data-src=\"https:\/\/scx2.b-cdn.net\/gfx\/news\/2024\/new-method-restores-he.jpg\" data-sub-html=\"An experimental gene therapy raised levels of the key protein protocadherin-15, shown in green, in light-sensing cells in the retinas of nonhuman primates. Credit: Maryna Ivanchenko\">\n<figure class=\"article-img\">\n<\/figure><\/div>\n<\/div>\n<p>Les chercheurs de la Harvard Medical School ont franchi une nouvelle \u00e9tape d\u00e9cisive dans leurs efforts visant \u00e0 d\u00e9velopper une th\u00e9rapie g\u00e9nique pour les personnes atteintes du syndrome d&rsquo;Usher de type 1F, une maladie rare qui provoque la surdit\u00e9 et la c\u00e9cit\u00e9 progressive.<\/p>\n<p>Une nouvelle fa\u00e7on de fournir une version corrig\u00e9e du g\u00e8ne d\u00e9fectueux responsable du syndrome d&rsquo;Usher, PCDH15, a restaur\u00e9 l&rsquo;audition chez des mod\u00e8les de souris et a montr\u00e9 son potentiel chez les organo\u00efdes r\u00e9tiniens et les primates non humains pour am\u00e9liorer la vision, rapporte l&rsquo;\u00e9quipe dans le rapport. <i>Journal d&rsquo;investigation clinique<\/i>.<\/p>\n<p>Il s&rsquo;agit de la deuxi\u00e8me th\u00e9rapie g\u00e9nique exp\u00e9rimentale pour le syndrome d&rsquo;Usher d\u00e9velopp\u00e9e par le laboratoire de David Corey, professeur Bertarelli de sciences m\u00e9dicales translationnelles \u00e0 l&rsquo;Institut Blavatnik du HMS. Des recherches ant\u00e9rieures ont montr\u00e9 qu&rsquo;une strat\u00e9gie diff\u00e9rente de d\u00e9livrance de g\u00e8nes r\u00e9tablissait l&rsquo;audition chez la souris. La nouvelle m\u00e9thode offre une deuxi\u00e8me option si la premi\u00e8re approche s&rsquo;av\u00e8re dangereuse ou inefficace lorsqu&rsquo;elle est test\u00e9e sur des humains.<\/p>\n<p>\u00ab\u00a0Sans essai clinique chez l&rsquo;homme, nous ne pouvons pas savoir si notre premi\u00e8re th\u00e9rapie g\u00e9nique r\u00e9tablit une fonction normale\u00a0\u00bb, a d\u00e9clar\u00e9 Corey, auteur principal de l&rsquo;\u00e9tude. \u00ab\u00a0Cette nouvelle strat\u00e9gie nous donne une solution de secours au cas o\u00f9 la premi\u00e8re th\u00e9rapie ne fonctionnerait pas. Elle pourrait m\u00eame s&rsquo;av\u00e9rer meilleure que la premi\u00e8re, une fois test\u00e9e sur des patients.\u00a0\u00bb<\/p>\n<figure class=\"mb-4\" itemscope=\"\" itemtype=\"http:\/\/schema.org\/VideoObject\">\n    <meta itemprop=\"name\" content=\"New method restores hearing, boosts vision in tests\"><br \/>\n    <meta itemprop=\"url\" content=\"https:\/\/vimeo.com\/869552432\"><br \/>\n    <meta itemprop=\"description\" content=\"New method restores hearing, boosts vision in tests\"><br \/>\n    <meta itemprop=\"uploadDate\" content=\"2024-11-15T10:15:52-05:00\"><br \/>\n            <meta itemprop=\"embedUrl\" content=\"https:\/\/player.vimeo.com\/video\/869552432\"><\/p>\n<p>\n             <iframe class=\"embed-responsive-item\" src=\"https:\/\/player.vimeo.com\/video\/869552432?color=ffffff\" loading=\"lazy\" frameborder=\"0\" webkitallowfullscreen=\"\" mozallowfullscreen=\"\" allowfullscreen=\"\" style=\"width: 100%; height: 100%; position: absolute\"><\/iframe>\n        <\/p>\n<\/figure>\n<h2>Plus de strat\u00e9gies, plus de chances de gu\u00e9rir<\/h2>\n<p>Un d\u00e9fi majeur dans la conception d&rsquo;une th\u00e9rapie avec le g\u00e8ne PCDH15 est qu&rsquo;il est trop gros pour tenir dans la coque \u00e9vid\u00e9e d&rsquo;un virus ad\u00e9no-associ\u00e9, ou AAV, le v\u00e9hicule le plus courant et le plus s\u00fbr utilis\u00e9 pour transporter les g\u00e8nes dans les cellules cibles.<\/p>\n<p>La strat\u00e9gie ant\u00e9rieure de l&rsquo;\u00e9quipe impliquait de r\u00e9duire PCDH15 en un mini g\u00e8ne pouvant s&rsquo;ins\u00e9rer dans un AAV.<\/p>\n<p>La nouvelle strat\u00e9gie consiste \u00e0 couper le g\u00e8ne complet en deux, \u00e0 ins\u00e9rer chaque moiti\u00e9 dans un AAV et \u00e0 administrer les AAV \u00e0 l&rsquo;oreille interne ou \u00e0 l&rsquo;\u0153il. L\u00e0, les moiti\u00e9s se rejoignent et commencent \u00e0 demander aux cellules de fabriquer correctement la prot\u00e9ine pour laquelle le g\u00e8ne code, la protocadh\u00e9rine-15.<\/p>\n<p>Les personnes atteintes du syndrome d&rsquo;Usher de type 1F naissent sans audition ni sens de l&rsquo;\u00e9quilibre et perdent progressivement la vision. La nouvelle approche a restaur\u00e9 l&rsquo;audition et l&rsquo;\u00e9quilibre chez les souris.<\/p>\n<p>Les mod\u00e8les de souris existants ne subissent pas le type de perte de vision observ\u00e9 dans le syndrome d&rsquo;Usher de type 1F, l&rsquo;\u00e9quipe n&rsquo;a donc pas pu \u00e9tudier si leur vision s&rsquo;est am\u00e9lior\u00e9e. Cependant, la th\u00e9rapie double-AAV a augment\u00e9 les niveaux de protocadh\u00e9rine-15 dans les cellules photosensibles des organo\u00efdes r\u00e9tiniens humains et des r\u00e9tines de primates non humains. La prot\u00e9ine a \u00e9galement migr\u00e9 vers le bon endroit dans ces cellules. Les deux r\u00e9sultats sugg\u00e8rent que la strat\u00e9gie th\u00e9rapeutique pourrait un jour agir pour pr\u00e9server la vision des patients.<\/p>\n<p>\u00ab\u00a0Ces r\u00e9sultats sont particuli\u00e8rement int\u00e9ressants car, m\u00eame si les implants cochl\u00e9aires peuvent traiter la perte auditive chez les patients humains, il n&rsquo;existe actuellement aucun traitement pour le dysfonctionnement de la vision associ\u00e9 au syndrome d&rsquo;Usher\u00a0\u00bb, a d\u00e9clar\u00e9 la premi\u00e8re auteure Maryna Ivanchenko, instructrice HMS en neurobiologie au Corey Lab et ophtalmologiste.<\/p>\n<p>                                        <!-- print only --><\/p><\/div>\n","protected":false},"excerpt":{"rendered":"<p>Les chercheurs de la Harvard Medical School ont franchi une nouvelle \u00e9tape d\u00e9cisive dans leurs<\/p>\n","protected":false},"author":1,"featured_media":2187,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[86],"tags":[2538,1378,1890,1064,2540,71,2539,72,1063,47,2541],"class_list":["post-2186","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-les-maladies","tag-alternative","tag-ameliore","tag-experimentale","tag-genique","tag-laudition","tag-lors","tag-retablit","tag-tests","tag-therapie","tag-une","tag-vision","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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