Targeted therapy in patients with PIK3CA-related overgrowth syndrome - Nature

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ID: 270255
2018
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Abstract
A PI3KCA inhibitor reverses symptoms in a mouse model of PROS/CLOVES syndrome, which results from gain-of-function mutations in PI3KCA, and produces improvements in patients with PROS/CLOVES syndrome.
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Authors Venot, Quitterie;Blanc, Thomas;Rabia, Smail Hadj;Berteloot, Laureline;Ladraa, Sophia;Duong, Jean-Paul;Blanc, Estelle;Johnson, Simon C.;Hoguin, Clément;Boccara, Olivia;Sarnacki, Sabine;Boddaert, Nathalie;Pannier, Stephanie;Martinez, Frank;Magassa, Sato;Yamaguchi, Junna;Knebelmann, Bertrand;Merville, Pierre;Grenier, Nicolas;Joly, Dominique;Cormier-Daire, Valérie;Michot, Caroline;Bole-Feysot, Christine;Picard, Arnaud;Soupre, Véronique;Lyonnet, Stanislas;Sadoine, Jeremy;Slimani, Lotfi;Chaussain, Catherine;Laroche-Raynaud, Cécile;Guibaud, Laurent;Broissand, Christine;Amiel, Jeanne;Legendre, Christophe;Terzi, Fabiola;Canaud, Guillaume;Venot, Quitterie;Blanc, Thomas;Rabia, Smail Hadj;Berteloot, Laureline;Ladraa, Sophia;Duong, Jean-Paul;Blanc, Estelle;Johnson, Simon C.;Hoguin, Clément;Boccara, Olivia;Sarnacki, Sabine;Boddaert, Nathalie;Pannier, Stephanie;Martinez, Frank;Magassa, Sato;Yamaguchi, Junna;Knebelmann, Bertrand;Merville, Pierre;Grenier, Nicolas;Joly, Dominique;Cormier-Daire, Valérie;Michot, Caroline;Bole-Feysot, Christine;Picard, Arnaud;Soupre, Véronique;Lyonnet, Stanislas;Sadoine, Jeremy;Slimani, Lotfi;Chaussain, Catherine;Laroche-Raynaud, Cécile;Guibaud, Laurent;Broissand, Christine;Amiel, Jeanne;Legendre, Christophe;Terzi, Fabiola;Canaud, Guillaume;
Journal Nature
Year 2018
DOI
doi:10.1038/s41586-018-0217-9
URL
https://doi.org/10.1038/s41586-018-0217-9
Keywords
translational research molecularly targeted therapy

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