{"id":10720,"date":"2018-03-28T09:27:34","date_gmt":"2018-03-28T13:27:34","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=10720"},"modified":"2018-03-28T09:30:48","modified_gmt":"2018-03-28T13:30:48","slug":"ee-launches-organoid-screening-of-cystic-fibrosis-nonsense-mutation","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/fr\/ee-launches-organoid-screening-of-cystic-fibrosis-nonsense-mutation\/","title":{"rendered":"EE lance le d\u00e9pistage par organo\u00efde de la mutation non-sens de la mucoviscidose"},"content":{"rendered":"

L'Entourage d'Emily est fier d'annoncer qu'il a accord\u00e9 une subvention d'un an \u00e0 l'Agence europ\u00e9enne pour la reconstruction. <\/span>bourse de recherche <\/span>\u00e0 Jeffrey Beekman, PhD, au Centre m\u00e9dical universitaire d'Utrecht, Pays-Bas.<\/span><\/p>\n

\"\"

Avec l'aimable autorisation de Jeffrey Beekman, PhD.<\/p><\/div>\n

Cette subvention refl\u00e8te une nouvelle fronti\u00e8re de la m\u00e9decine personnalis\u00e9e. Organo\u00efdes<\/a><\/strong>ou mini-organes - sont d\u00e9riv\u00e9s des cellules souches intestinales d'un patient atteint de FK et se sont r\u00e9v\u00e9l\u00e9s capables de pr\u00e9dire l'efficacit\u00e9 de certaines th\u00e9rapies pour des mutations particuli\u00e8res. \u00a0<\/span><\/p>\n

Pionnier de l'application des organo\u00efdes \u00e0 la th\u00e9rapeutique de la fibrose kystique, le Dr Be<\/span>ekman utilisera des organo\u00efdes pour \u00e9valuer si les modulateurs CFTR \u00e0 petites mol\u00e9cules actuels et exp\u00e9rimentaux sont efficaces pour la mutation W1282X.<\/span><\/p>\n

\"\"<\/p>\n

Ce projet est d'autant plus urgent qu'il existe des traitements r\u00e9volutionnaires en cours de commercialisation ou en phase avanc\u00e9e de d\u00e9veloppement pour environ 90% de la population FK. Les personnes porteuses de deux copies de mutations non sens, dont W1282X, font partie des 10% de la population FK pour lesquelles aucun traitement ciblant les mutations n'est en cours de d\u00e9veloppement. Les programmes de d\u00e9pistage de m\u00e9dicaments comme celui-ci permettent de d\u00e9terminer si les m\u00e9dicaments d\u00e9j\u00e0 d\u00e9velopp\u00e9s ou en cours de d\u00e9veloppement pour d'autres mutations de la FK peuvent \u00e9galement \u00eatre b\u00e9n\u00e9fiques pour les mutations non sens, ce qui pourrait permettre d'identifier des solutions th\u00e9rapeutiques \u00e0 court terme pour les 10% restants.<\/p>\n

\"\"Dans le cadre de l'op\u00e9ration \"Emily's Entourage\", leCatalyseur pour la cure<\/i><\/b><\/a><\/span>\"Ce projet s'inscrit dans le cadre de l'approche strat\u00e9gique de la fondation, qui consiste \u00e0 \u00e9tablir des partenariats avec des scientifiques de renomm\u00e9e mondiale afin d'acc\u00e9l\u00e9rer le d\u00e9veloppement de traitements vitaux pour les mutations non sens de la fibrose kystique.<\/p>\n

L'Entourage d'Emily soutient la recherche et le d\u00e9veloppement de m\u00e9dicaments par le biais de diff\u00e9rents m\u00e9canismes de financement, dont un programme de subventions annuelles. Gr\u00e2ce au soutien d'incroyables donateurs, la fondation a octroy\u00e9 plus de $2,7 millions de dollars \u00e0 la recherche sur les mutations non-sens de la FK depuis 2011. Pour en savoir plus, visitez le site de la\u00a0Subventions accord\u00e9es<\/a><\/strong> page.\u00a0<\/span><\/p>\n

Strat\u00e9gie : Naviguer dans la biblioth\u00e8que : Identification et r\u00e9affectation de mol\u00e9cules th\u00e9rapeutiques<\/i><\/span><\/p>","protected":false},"excerpt":{"rendered":"

Emily\u2019s Entourage is proud to announce that it has awarded a one-year research grant to Jeffrey Beekman, PhD, at the University Medical Center, Utrecht, Netherlands. This grant reflects a new frontier of personalized medicine. Organoids\u2014or mini organs\u2014are derived from a CF patient’s own intestinal stem cells and have been shown to predict whether certain therapies… <\/p>\n

<\/div>\n

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