Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases
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
Published in The Journal of clinical investigation (01.04.2016)
Published in The Journal of clinical investigation (01.04.2016)
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Journal Article
Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons
Duque, Sandra, Joussemet, Béatrice, Riviere, Christel, Marais, Thibaut, Dubreil, Laurence, Douar, Anne-Marie, Fyfe, John, Moullier, Philippe, Colle, Marie-Anne, Barkats, Martine
Published in Molecular therapy (01.07.2009)
Published in Molecular therapy (01.07.2009)
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Journal Article
A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model
Biferi, Maria Grazia, Cohen-Tannoudji, Mathilde, Cappelletto, Ambra, Giroux, Benoit, Roda, Marianne, Astord, Stéphanie, Marais, Thibaut, Bos, Corinne, Voit, Thomas, Ferry, Arnaud, Barkats, Martine
Published in Molecular therapy (06.09.2017)
Published in Molecular therapy (06.09.2017)
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Journal Article
A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome
Matagne, Valerie, Ehinger, Yann, Saidi, Lydia, Borges-Correia, Ana, Barkats, Martine, Bartoli, Marc, Villard, Laurent, Roux, Jean-Christophe
Published in Neurobiology of disease (01.03.2017)
Published in Neurobiology of disease (01.03.2017)
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Journal Article
Lentiviral Vectors with a Defective Integrase Allow Efficient and Sustained Transgene Expression in vitro and in vivo
Philippe, Stéphanie, Sarkis, Chamsy, Barkats, Martine, Mammeri, Hamid, Ladroue, Charline, Petit, Caroline, Mallet, Jacques, Serguera, Che
Published in Proceedings of the National Academy of Sciences - PNAS (21.11.2006)
Published in Proceedings of the National Academy of Sciences - PNAS (21.11.2006)
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Journal Article
Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice
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
Published in Human molecular genetics (15.03.2018)
Published in Human molecular genetics (15.03.2018)
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Journal Article
Interferon beta induces clearance of mutant ataxin 7 and improves locomotion in SCA7 knock-in mice
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
Published in Brain (London, England : 1878) (01.06.2013)
Published in Brain (London, England : 1878) (01.06.2013)
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Journal Article
The autophagy/lysosome pathway is impaired in SCA7 patients and SCA7 knock-in mice
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
Published in Acta neuropathologica (01.11.2014)
Published in Acta neuropathologica (01.11.2014)
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Journal Article
Targeting γ-secretase triggers the selective enrichment of oligomeric APP-CTFs in brain extracellular vesicles from Alzheimer cell and mouse models
Lauritzen, Inger, Bécot, Anaïs, Bourgeois, Alexandre, Pardossi-Piquard, Raphaëlle, Biferi, Maria-Grazia, Barkats, Martine, Checler, Fréderic
Published in Translational neurodegeneration (05.12.2019)
Published in Translational neurodegeneration (05.12.2019)
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Journal Article
scAAV9 intracisternal delivery results in efficient gene transfer to the central nervous system of a feline model of motor neuron disease
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
Published in Human gene therapy (01.07.2013)
Published in Human gene therapy (01.07.2013)
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Journal Article
Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A
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
Published in Molecular therapy. Methods & clinical development (12.03.2021)
Published in Molecular therapy. Methods & clinical development (12.03.2021)
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Journal Article
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy
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
Published in Molecular therapy. Methods & clinical development (01.01.2016)
Published in Molecular therapy. Methods & clinical development (01.01.2016)
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Journal Article
A single intravenous AAV9 injection mediates bilateral gene transfer to the adult mouse retina
Bemelmans, Alexis-Pierre, Duqué, Sandra, Rivière, Christel, Astord, Stéphanie, Desrosiers, Mélissa, Marais, Thibault, Sahel, José-Alain, Voit, Thomas, Barkats, Martine
Published in PloS one (15.04.2013)
Published in PloS one (15.04.2013)
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Journal Article
Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice
Tanguy, Yannick, Biferi, Maria G., Besse, Aurore, Astord, Stephanie, Cohen-Tannoudji, Mathilde, Marais, Thibaut, Barkats, Martine
Published in Frontiers in molecular neuroscience (28.07.2015)
Published in Frontiers in molecular neuroscience (28.07.2015)
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Journal Article
1-methyl-4-phenylpyridinium neurotoxicity is attenuated by adenoviral gene transfer of human Cu/Zn superoxide dismutase
Barkats, Martine, Horellou, Philippe, Colin, Philippe, Millecamps, Stéphanie, Faucon-Biguet, Nicole, Mallet, Jacques
Published in Journal of neuroscience research (01.02.2006)
Published in Journal of neuroscience research (01.02.2006)
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Journal Article
Correction to: Intraneuronal aggregation of the [beta]âCTF fragment of APP induces A[beta]âindependent lysosomal-autophagic pathology
Lauritzen, Inger, PardossiâPiquard, Raphaëlle, Bourgeois, Alexandre, Pagnotta, Sophie, Biferi, MariaâGrazia, Barkats, Ma, Lacor, Pascale
Published in Acta neuropathologica (01.10.2023)
Published in Acta neuropathologica (01.10.2023)
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Journal Article
Intraneuronal aggregation of the β-CTF fragment of APP (C99) induces Aβ-independent lysosomal-autophagic pathology
Lauritzen, Inger, Pardossi-Piquard, Raphaëlle, Bourgeois, Alexandre, Pagnotta, Sophie, Biferi, Maria-Grazia, Barkats, Martine, Lacor, Pascale, Klein, William, Bauer, Charlotte, Checler, Frederic
Published in Acta neuropathologica (01.08.2016)
Published in Acta neuropathologica (01.08.2016)
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Journal Article