Establishment of heterozygous and homozygous SHANK3 knockout clonal pluripotent stem cells from the parental hESC line SA001 using CRISPR/Cas9 von Chatrousse, Laure, Poullion, Thifaine, El-Kassar, Lina, Giraud-Triboult, Karine, Boissart, Claire, Sanatine, Peggy, Sommer, Peter, Benchoua, Alexandra

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Spastin regulates ER-mitochondrial contact sites and mitochondrial homeostasis von Raby, Amelie, Missiroli, Sonia, Sanatine, Peggy, Langui, Dominique, Pansiot, Julien, Beaude, Nissai, Vezzana, Lucie, Saleh, Rachelle, Marinello, Martina, Laforge, Mireille, Pinton, Paolo, Buj-Bello, Ana, Burgo, Andrea

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Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle von Poupiot, Jérôme, Costa Verdera, Helena, Hardet, Romain, Colella, Pasqualina, Collaud, Fanny, Bartolo, Laurent, Davoust, Jean, Sanatine, Peggy, Mingozzi, Federico, Richard, Isabelle, Ronzitti, Giuseppe

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Monobac System-A Single Baculovirus for the Production of rAAV von Galibert, Lionel, Jacob, Aurelien, Savy, Adrien, Dickx, Yohann, Bonnin, Delphine, Lecomte, Christophe, Rivollet, Lise, Sanatine, Peggy, Boutin Fontaine, Marjorie, Le Bec, Christine, Merten, Otto-Wilhelm

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VCN Analysis Using Droplet Digital PCR Method in Hematopoietic Stem Cells and T Lymphocytes after Lentiviral Transduction: Optimization and Limitations von Parcelier, Aude, Buisset, Julien, Neveu, Emilie, Kauffmann, Bastien, Fraboulet, Sandrine, Sanatine, Peggy, Martin, Samia, Rousseaux, Cédrick, Avenier, Nadia, Charrier, Sabine

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Bistable cell fate specification as a result of stochastic fluctuations and collective spatial cell behaviour von Stockholm, Daniel, Edom-Vovard, Frédérique, Coutant, Sophie, Sanatine, Peggy, Yamagata, Yoshiaki, Corre, Guillaume, Le Guillou, Laurent, Neildez-Nguyen, Thi My Anh, Pàldi, Andràs

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Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics von Barbon, Elena, Ferrarese, Mattia, van Wittenberghe, Laetitia, Sanatine, Peggy, Ronzitti, Giuseppe, Collaud, Fanny, Colella, Pasqualina, Pinotti, Mirko, Mingozzi, Federico

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Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in the dystrophic muscle in Duchenne muscular dystrophy von Vu Hong, Ai, Bourg, Nathalie, Sanatine, Peggy, Poupiot, Jerome, Charton, Karine, Gicquel, Evelyne, Massourides, Emmanuelle, Spinazzi, Marco, Richard, Isabelle, Israeli, David

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Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in Duchenne muscular dystrophy von Vu Hong, Ai, Bourg, Nathalie, Sanatine, Peggy, Poupiot, Jerome, Charton, Karine, Gicquel, Evelyne, Massourides, Emmanuelle, Spinazzi, Marco, Richard, Isabelle, Israeli, David

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Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle von Poupiot, Jérôme, Costa Verdera, Helena, Hardet, Romain, Colella, Pasqualina, Collaud, Fanny, Bartolo, Laurent, Davoust, Jean, Sanatine, Peggy, Mingozzi, Federico, Richard, Isabelle, Ronzitti, Giuseppe

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Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics von Barbon, Elena, Ferrarese, Mattia, van Wittenberghe, Laetitia, Sanatine, Peggy, Ronzitti, Giuseppe, Collaud, Fanny, Colella, Pasqualina, Pinotti, Mirko, Mingozzi, Federico

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P51. Bistable cell fate specification as a result of stochastic fluctuations and collective spatial cell behaviour von Andràs, Pàldi, Daniel, Stockholm, Frédérique, Edom-Vovard, Sophie, Coutant, Peggy, Sanatine, Yoshiaki, Yamagata, Guillaume, Corre, Le Guillou, Laurent, Thi My Anh, Neildez-Nguyen

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