Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases von Dragin, Nadine, Bismuth, Jacky, Cizeron-Clairac, Géraldine, Biferi, Maria Grazia, Berthault, Claire, Serraf, Alain, Nottin, Rémi, Klatzmann, David, Cumano, Ana, Barkats, Martine, Le Panse, Rozen, Berrih-Aknin, Sonia

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Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons von Duque, Sandra, Joussemet, Béatrice, Riviere, Christel, Marais, Thibaut, Dubreil, Laurence, Douar, Anne-Marie, Fyfe, John, Moullier, Philippe, Colle, Marie-Anne, Barkats, Martine

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A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model von Biferi, Maria Grazia, Cohen-Tannoudji, Mathilde, Cappelletto, Ambra, Giroux, Benoit, Roda, Marianne, Astord, Stéphanie, Marais, Thibaut, Bos, Corinne, Voit, Thomas, Ferry, Arnaud, Barkats, Martine

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A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome von Matagne, Valerie, Ehinger, Yann, Saidi, Lydia, Borges-Correia, Ana, Barkats, Martine, Bartoli, Marc, Villard, Laurent, Roux, Jean-Christophe

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Lentiviral Vectors with a Defective Integrase Allow Efficient and Sustained Transgene Expression in vitro and in vivo von Philippe, Stéphanie, Sarkis, Chamsy, Barkats, Martine, Mammeri, Hamid, Ladroue, Charline, Petit, Caroline, Mallet, Jacques, Serguera, Che

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The autophagy/lysosome pathway is impaired in SCA7 patients and SCA7 knock-in mice von Alves, Sandro, Cormier-Dequaire, Florence, Marinello, Martina, Marais, Thibaut, Muriel, Marie-Paule, Beaumatin, Florian, Charbonnier-Beaupel, Fanny, Tahiri, Khadija, Seilhean, Danielle, El Hachimi, Khalid, Ruberg, Merle, Stevanin, Giovanni, Barkats, Martine, den Dunnen, Wilfred, Priault, Muriel, Brice, Alexis, Durr, Alexandra, Corvol, Jean-Christophe, Sittler, Annie

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Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice von Niemir, Natalia, Rouvière, Laura, Besse, Aurore, Vanier, Marie T, Dmytrus, Jasmin, Marais, Thibaut, Astord, Stéphanie, Puech, Jean-Philippe, Panasyuk, Ganna, Cooper, Jonathan D, Barkats, Martine, Caillaud, Catherine

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Interferon beta induces clearance of mutant ataxin 7 and improves locomotion in SCA7 knock-in mice von CHORT, Alice, ALVES, Sandro, RUBERG, Merle, JANER, Alexandre, STEVANIN, Giovanni, BRICE, Alexis, SITTLER, Annie, MARINELLO, Martina, DUFRESNOIS, Béatrice, DORNBIERER, Jean-Gabriel, TESSON, Christelle, LATOUCHE, Morwena, BAKER, Darren P, BARKATS, Martine, EL HACHIMI, Khalid H

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Targeting γ-secretase triggers the selective enrichment of oligomeric APP-CTFs in brain extracellular vesicles from Alzheimer cell and mouse models von Lauritzen, Inger, Bécot, Anaïs, Bourgeois, Alexandre, Pardossi-Piquard, Raphaëlle, Biferi, Maria-Grazia, Barkats, Martine, Checler, Fréderic

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SMA: from gene discovery to gene therapy von Barkats, Martine

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scAAV9 intracisternal delivery results in efficient gene transfer to the central nervous system of a feline model of motor neuron disease von Bucher, Thomas, Colle, Marie-Anne, Wakeling, Erin, Dubreil, Laurence, Fyfe, John, Briot-Nivard, Delphine, Maquigneau, Maud, Raoul, Sylvie, Cherel, Yan, Astord, Stéphanie, Duque, Sandra, Marais, Thibaut, Voit, Thomas, Moullier, Philippe, Barkats, Martine, Joussemet, Béatrice

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Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A von Biferi, Maria Grazia, Cohen-Tannoudji, Mathilde, García-Silva, Andrea, Souto-Rodríguez, Olga, Viéitez-González, Irene, San-Millán-Tejado, Beatriz, Fernández-Carrera, Andrea, Pérez-Márquez, Tania, Teijeira-Bautista, Susana, Barrera, Soraya, Domínguez, Vanesa, Marais, Thibaut, González-Fernández, África, Barkats, Martine, Ortolano, Saida

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A single intravenous AAV9 injection mediates bilateral gene transfer to the adult mouse retina von Bemelmans, Alexis-Pierre, Duqué, Sandra, Rivière, Christel, Astord, Stéphanie, Desrosiers, Mélissa, Marais, Thibault, Sahel, José-Alain, Voit, Thomas, Barkats, Martine

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Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy von Armbruster, Nicole, Lattanzi, Annalisa, Jeavons, Matthieu, Van Wittenberghe, Laetitia, Gjata, Bernard, Marais, Thibaut, Martin, Samia, Vignaud, Alban, Voit, Thomas, Mavilio, Fulvio, Barkats, Martine, Buj-Bello, Ana

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Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice von Tanguy, Yannick, Biferi, Maria G., Besse, Aurore, Astord, Stephanie, Cohen-Tannoudji, Mathilde, Marais, Thibaut, Barkats, Martine

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Correction to: Intraneuronal aggregation of the [beta]âCTF fragment of APP induces A[beta]âindependent lysosomal-autophagic pathology von Lauritzen, Inger, PardossiâPiquard, Raphaëlle, Bourgeois, Alexandre, Pagnotta, Sophie, Biferi, MariaâGrazia, Barkats, Ma, Lacor, Pascale

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Amyotrophie spinale infantile: De la découverte du gène à la thérapie génique von Barkats, Martine

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Intraneuronal aggregation of the β-CTF fragment of APP (C99) induces Aβ-independent lysosomal-autophagic pathology von Lauritzen, Inger, Pardossi-Piquard, Raphaëlle, Bourgeois, Alexandre, Pagnotta, Sophie, Biferi, Maria-Grazia, Barkats, Martine, Lacor, Pascale, Klein, William, Bauer, Charlotte, Checler, Frederic

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AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice von Besse, Aurore, Astord, Stephanie, Marais, Thibaut, Roda, Marianne, Giroux, Benoit, Lejeune, François-Xavier, Relaix, Frederic, Smeriglio, Piera, Barkats, Martine, Biferi, Maria Grazia

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Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice von Benkhelifa-Ziyyat, Sofia, Besse, Aurore, Roda, Marianne, Duque, Sandra, Astord, Stéphanie, Carcenac, Romain, Marais, Thibaut, Barkats, Martine

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