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Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responses Frati, Giacomo; Brusson, Megane; Sartre, Gilles; Mlayah, Bochra; Felix, Tristan; Chalumeau, Anne; Antoniou, Panagiotis; Hardouin, Giulia; Concordet, Jean-Paul; Romano, Oriana; Turchiano, Giandomenico; Miccio, Annarita Share Save
CRITICAL CONTRIBUTION OF MITOCHONDRIA IN THE DEVELOPMENT OF CARDIOMYOPATHY LINKED TO DESMIN MUTATION Hovhannisyan, Yeranuhi; Li, Zhenlin; Callon, Domitille; Suspene, Rodolphe; Batoumeni, Vivien; Canette, Alexis; Blanc, Jocelyne; Hocini, Hakim; Lefebvre, Cecile; El-Jahrani, Nora; Kitsara, Maria; L'honore, Aurore; Kordeli, Ekaterini; Fornes, Paul; Concordet, Jean-Paul; Tachdjian, Gerard; Rodriguez, Anne-Marie; Vartanian, Jean-Pierre; Behin, Anthony; Wahbi, Karim; Joanne, Pierre; Agbulut, Onnik Share Save
Making genome editing a success story in Africa Abkallo, Hussein M.; Arbuthnot, Patrick; Auer, Thomas O.; Berger, Dave K.; Burger, Johan; Chakauya, Ereck; Concordet, Jean-Paul; Diabate, Abdoulaye; Di Donato, Vincenzo; Groenewald, Jan-Hendrik; Guindo, Amadou; Koekemoer, Lizette L.; Nazare, Florence; Nolan, Tony; Okumu, Fredros; Orefuwa, Emma; Paemka, Lily; Prieto-Godino, Lucia; Runo, Steven; Sadler, Marie; Tesfaye, Kassahun; Tripathi, Leena; Wondji, Charles Share Save
Critical contribution of mitochondria in the development of cardiomyopathy linked to desmin mutation Hovhannisyan, Yeranuhi; Li, Zhenlin; Callon, Domitille; Suspene, Rodolphe; Batoumeni, Vivien; Canette, Alexis; Blanc, Jocelyne; Hocini, Hakim; Lefebvre, Cecile; El-Jahrani, Nora; Kitsara, Maria; L'honore, Aurore; Kordeli, Ekaterini; Fornes, Paul; Concordet, Jean-Paul; Tachdjian, Gerard; Rodriguez, Anne-Marie; Vartanian, Jean-Pierre; Behin, Anthony; Wahbi, Karim; Joanne, Pierre; Agbulut, Onnik Share Save
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Changes in social behavior with MAPK2 and KCTD13/CUL3 pathways alterations in two new outbred rat models for the 16p11.2 syndromes with autism spectrum disorders Martin Lorenzo, Sandra; Muniz Moreno, Maria del Mar; Atas, Helin; Pellen, Marion; Nalesso, Valerie; Raffelsberger, Wolfgang; Prevost, Geraldine; Lindner, Loic; Birling, Marie-Christine; Menoret, Severine; Tesson, Laurent; Negroni, Luc; Concordet, Jean-Paul; Anegon, Ignacio; Herault, Yann Share Save
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Six1 and Eya1 expression can reprogram adult muscle from the slow-twitch phenotype into the fast-twitch phenotype Grifone, R; Laclef, C; Spitz, F; Lopez, S; Demignon, J; Guidotti, JE; Kawakami, K; Xu, PX; Kelly, R; Petrof, BJ; Daegelen, D; Concordet, JP; Maire, P Share Save
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Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression Antoniou, Panagiotis; Hardouin, Giulia; Martinucci, Pierre; Frati, Giacomo; Felix, Tristan; Chalumeau, Anne; Fontana, Letizia; Martin, Jeanne; Masson, Cecile; Brusson, Megane; Maule, Giulia; Rosello, Marion; Giovannangeli, Carine; Abramowski, Vincent; De Villartay, Jean-Pierre; Concordet, Jean-Paul; Del Bene, Filippo; El Nemer, Wassim; Amendola, Mario; Cavazzana, Marina; Cereseto, Anna; Romano, Oriana; Miccio, Annarita Share Save
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A fast Myosin super enhancer dictates muscle fiber phenotype through competitive interactions with Myosin genes Dos Santos, Matthieu; Backer, Stephanie; Aurade, Frederic; Wong, Matthew Man-Kin; Wurmser, Maud; Pierre, Remi; Langa, Francina; Do Cruzeiro, Marcio; Schmitt, Alain; Concordet, Jean-Paul; Sotiropoulos, Athanassia; Jeffrey Dilworth, F.; Noordermeer, Daan; Relaix, Frederic; Sakakibara, Iori; Maire, Pascal Share Save
ChREBPβ is dispensable for the control of glucose homeostasis and energy balance Recazens, Emeline; Tavernier, Genevieve; Dufau, Jeremy; Bergoglio, Camille; Benhamed, Fadila; Cassant-Sourdy, Stephanie; Marques, Marie-Adeline; Caspar-Bauguil, Sylvie; Brion, Alice; Monbrun, Laurent; Dentin, Renaud; Ferrier, Clara; Leroux, Melanie; Denechaud, Pierre-Damien; Moro, Cedric; Concordet, Jean-Paul; Postic, Catherine; Mouisel, Etienne; Langin, Dominique Share Save
Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease Ramadier, Sophie; Chalumeau, Anne; Felix, Tristan; Othman, Nadia; Aknoun, Sherazade; Casini, Antonio; Maule, Giulia; Masson, Cecile; De Cian, Anne; Frati, Giacomo; Brusson, Megane; Concordet, Jean-Paul; Cavazzana, Marina; Cereseto, Anna; El Nemer, Wassim; Amendola, Mario; Wattellier, Benoit; Meneghini, Vasco; Miccio, Annarita Share Save
HEK293T Cells with TFAM Disruption by CRISPR-Cas9 as a Model for Mitochondrial Regulation de Oliveira, Vanessa Cristina; Santos Roballo, Kelly Cristine; Mariano Junior, Clesio Gomes; Santos, Sarah Ingrid Pinto; Bressan, Fabiana Fernandes; Chiaratti, Marcos Roberto; Tucker, Elena J.; Davis, Erica E.; Concordet, Jean-Paul; Ambrosio, Carlos Eduardo Share Save
CRISPR gene editing in pluripotent stem cells reveals the function of MBNL proteins during human in vitro myogenesis Merien, Antoine; Tahraoui-Bories, Julie; Cailleret, Michel; Dupont, Jean-Baptiste; Leteur, Celine; Polentes, Jerome; Carteron, Alexandre; Polveche, Helene; Concordet, Jean-Paul; Pinset, Christian; Jarrige, Margot; Furling, Denis; Martinat, Cecile Share Save