Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome von Gougeon, Marie-Lise, Poirier-Beaudouin, Béatrice, Ausseil, Jérome, Zérah, Michel, Artaud, Cécile, Heard, Jean-Michel, Deiva, Kumaran, Tardieu, Marc

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Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece von Héron, Bénédicte, Mikaeloff, Yann, Froissart, Roseline, Caridade, Guillaume, Maire, Irène, Caillaud, Catherine, Levade, Thierry, Chabrol, Brigitte, Feillet, François, Ogier, Hélène, Valayannopoulos, Vassili, Michelakakis, Helen, Zafeiriou, Dimitrios, Lavery, Lucy, Wraith, Ed, Danos, Olivier, Heard, Jean-Michel, Tardieu, Marc

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Oxidative stress is independent of inflammation in the neurodegenerative sanfilippo syndrome type B von Trudel, Stéphanie, Trécherel, Eric, Gomila, Cathy, Peltier, Maxime, Aubignat, Michaël, Gubler, Brigitte, Morlière, Patrice, Heard, Jean-Michel, Ausseil, Jérôme

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Early neurodegeneration progresses independently of microglial activation by heparan sulfate in the brain of mucopolysaccharidosis IIIB mice von Ausseil, Jérôme, Desmaris, Nathalie, Bigou, Stéphanie, Attali, Ruben, Corbineau, Sébastien, Vitry, Sandrine, Parent, Mathieu, Cheillan, David, Fuller, Maria, Maire, Irène, Vanier, Marie-Thérèse, Heard, Jean-Michel

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Surrogate Cerebrospinal Fluid Biomarkers for Assessing the Efficacy of Gene Therapy in Hurler Syndrome von Haseloff, Reiner F., Trudel, Stephanie, Birke, Ramona, Schümann, Michael, Krause, Eberhard, Gomila, Cathy, Heard, Jean-Michel, Blasig, Ingolf E., Ausseil, Jérôme

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Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial von Tardieu, Marc, Zérah, Michel, Gougeon, Marie-Lise, Ausseil, Jérome, de Bournonville, Stéphanie, Husson, Béatrice, Zafeiriou, Dimitrios, Parenti, Giancarlo, Bourget, Philippe, Poirier, Béatrice, Furlan, Valérie, Artaud, Cécile, Baugnon, Thomas, Roujeau, Thomas, Crystal, Ronald G, Meyer, Christian, Deiva, Kumaran, Heard, Jean-Michel

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Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results o... von Tardieu, Marc, Zérah, Michel, Husson, Béatrice, de Bournonville, Stéphanie, Deiva, Kumaran, Adamsbaum, Catherine, Vincent, Fanny, Hocquemiller, Michael, Broissand, Christine, Furlan, Valérie, Ballabio, Andrea, Fraldi, Alessandro, Crystal, Ronald G, Baugnon, Thomas, Roujeau, Thomas, Heard, Jean-Michel, Danos, Olivier

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Directed Evolution of Motor Neurons from Genetically Engineered Neural Precursors von Bohl, Delphine, Liu, Song, Blanchard, Stéphane, Hocquemiller, Michaël, Haase, Georg, Heard, Jean‐Michel

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Endocytosis of major histocompatibility complex class I molecules is induced by the HIV-1 Nef protein von Schwartz, Olivier, Maréchal, Valerie, Gall, Sylvie Le, Lemonnier, François, Heard, Jean-Michel

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Gene therapy of the brain in the dog model of Hurler's syndrome von Ciron, Carine, Desmaris, Nathalie, Colle, Marie-Anne, Raoul, Sylvie, Joussemet, Béatrice, Vérot, Lucie, Ausseil, Jérôme, Froissart, Roseline, Roux, Françoise, Chérel, Yan, Ferry, Nicolas, Lajat, Yaouen, Schwartz, Bertrand, Vanier, Marie-Thérèse, Maire, Irène, Tardieu, Marc, Moullier, Philippe, Heard, Jean-Michel

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Intracerebral Gene Therapy in Four Children with Sanfilippo B Syndrome: 5.5-Year Follow-Up Results von Deiva, Kumaran, Ausseil, Jérôme, de Bournonville, Stéphanie, Zérah, Michel, Husson, Béatrice, Gougeon, Marie-Lise, Poirier-Beaudouin, Béatrice, Zafeiriou, Dimitrios, Parenti, Giancarlo, Heard, Jean-Michel, Tardieu, Marc

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Lack of an Immune Response against the Tetracycline-Dependent Transactivator Correlates with Long-Term Doxycycline-Regulated Transgene Expression in Nonhuman Primates after Intramu... von Favre, David, Blouin, Véronique, Provost, Nathalie, Spisek, Radec, Porrot, Françoise, Bohl, Delphine, Marmé, Frederic, Chérel, Yan, Salvetti, Anna, Hurtrel, Bruno, Heard, Jean-Michel, Rivière, Yves, Moullier, Philippe

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Modeling neuronal defects associated with a lysosomal disorder using patient-derived induced pluripotent stem cells von LEMONNIER, Thomas, BLANCHARD, Stephane, TOLI, Diana, ROY, Elise, BIGOU, Stephanie, FROISSART, Roseline, ROUVET, Isabelle, VITRY, Sandrine, MICHEL HEARD, Jean, BOHL, Delphine

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Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes von Ellinwood, N Matthew, Ausseil, Jérôme, Desmaris, Nathalie, Bigou, Stéphanie, Liu, Song, Jens, Jackie K, Snella, Elizabeth M, Mohammed, Eman EA, Thomson, Christopher B, Raoul, Sylvie, Joussemet, Béatrice, Roux, Françoise, Chérel, Yan, Lajat, Yaouen, Piraud, Monique, Benchaouir, Rachid, Hermening, Stephan, Petry, Harald, Froissart, Roseline, Tardieu, Marc, Ciron, Carine, Moullier, Philippe, Parkes, Jennifer, Kline, Karen L, Maire, Irène, Vanier, Marie-Thérèse, Heard, Jean-Michel, Colle, Marie-Anne

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Antiviral Activity of the Proteasome on Incoming Human Immunodeficiency Virus Type 1 von Schwartz, Olivier, Maréchal, Valérie, Friguet, Bertrand, Arenzana-Seisdedos, Fernando, Heard, Jean-Michel

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Human α-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates : Vector Diffusion and Biodistribution von CIRON, Carine, CRESSANT, Arnaud, TARDIEU, Marc, MOULLIER, Philippe, COLLE, Marie-Anne, ROUX, Francoise, RAOUL, Sylvie, CHEREL, Yan, HANTRAYE, Philippe, DEGLON, Nicole, SCHWARTZ, Bertrand, BARKATS, Martine, HEARD, Jean-Michel

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GM130 gain-of-function induces cell pathology in a model of lysosomal storage disease von Roy, Elise, Bruyère, Julie, Flamant, Patricia, Bigou, Stéphanie, Ausseil, Jérôme, Vitry, Sandrine, Heard, Jean Michel

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Human Immunodeficiency Virus Type 1 Entry into Macrophages Mediated by Macropinocytosis von Maréchal, V, Prevost, M C, Petit, C, Perret, E, Heard, J M, Schwartz, O

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Improved Behavior and Neuropathology in the Mouse Model of Sanfilippo Type IIIB Disease after Adeno-Associated Virus-Mediated Gene Transfer in the Striatum von Cressant, Arnaud, Desmaris, Nathalie, Verot, Lucie, Brejot, Thomas, Froissart, Roseline, Vanier, Marie-T, Maire, Irene, Heard, Jean Michel

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Neurotrophin 3 Improves Delayed Reconstruction of Sensory Pathways After Cervical Dorsal Root Injury von Liu, Song, Blanchard, Stephane, Bigou, Stephanie, Vitry, Sandrine, Bohl, Delphine, Heard, Jean-Michel

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