Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Le Guiner, Caroline, Servais, Laurent, Montus, Marie, Larcher, Thibaut, Fraysse, Bodvaël, Moullec, Sophie, Allais, Marine, François, Virginie, Dutilleul, Maeva, Malerba, Alberto, Koo, Taeyoung, Thibaut, Jean-Laurent, Matot, Béatrice, Devaux, Marie, Le Duff, Johanne, Deschamps, Jack-Yves, Barthelemy, Inès, Blot, Stéphane, Testault, Isabelle, Wahbi, Karim, Ederhy, Stéphane, Martin, Samia, Veron, Philippe, Georger, Christophe, Athanasopoulos, Takis, Masurier, Carole, Mingozzi, Federico, Carlier, Pierre, Gjata, Bernard, Hogrel, Jean-Yves, Adjali, Oumeya, Mavilio, Fulvio, Voit, Thomas, Moullier, Philippe, Dickson, George
Published in Nature communications (25.07.2017)
Published in Nature communications (25.07.2017)
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Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System
Lattanzi, Annalisa, Duguez, Stephanie, Moiani, Arianna, Izmiryan, Araksya, Barbon, Elena, Martin, Samia, Mamchaoui, Kamel, Mouly, Vincent, Bernardi, Francesco, Mavilio, Fulvio, Bovolenta, Matteo
Published in Molecular therapy. Nucleic acids (16.06.2017)
Published in Molecular therapy. Nucleic acids (16.06.2017)
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Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
Collaud, Fanny, Bortolussi, Giulia, Guianvarc'h, Laurence, Aronson, Sem J, Bordet, Thierry, Veron, Philippe, Charles, Severine, Vidal, Patrice, Sola, Marcelo Simon, Rundwasser, Stephanie, Dufour, Delphine G, Lacoste, Florence, Luc, Cyril, Wittenberghe, Laetitia V, Martin, Samia, Le Bec, Christine, Bosma, Piter J, Muro, Andres F, Ronzitti, Giuseppe, Hebben, Matthias, Mingozzi, Federico
Published in Molecular therapy. Methods & clinical development (15.03.2019)
Published in Molecular therapy. Methods & clinical development (15.03.2019)
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VCN Analysis Using Droplet Digital PCR Method in Hematopoietic Stem Cells and T Lymphocytes after Lentiviral Transduction: Optimization and Limitations
Parcelier, Aude, Buisset, Julien, Neveu, Emilie, Kauffmann, Bastien, Fraboulet, Sandrine, Sanatine, Peggy, Martin, Samia, Rousseaux, Cédrick, Avenier, Nadia, Charrier, Sabine
Published in Re:GEN open (01.01.2022)
Published in Re:GEN open (01.01.2022)
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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
Ronzitti, Giuseppe, Bortolussi, Giulia, van Dijk, Remco, Collaud, Fanny, Charles, Severine, Leborgne, Christian, Vidal, Patrice, Martin, Samia, Gjata, Bernard, Sola, Marcelo Simon, van Wittenberghe, Laetitia, Vignaud, Alban, Veron, Philippe, Bosma, Piter J, Muro, Andres F, Mingozzi, Federico
Published in Molecular therapy. Methods & clinical development (01.01.2016)
Published in Molecular therapy. Methods & clinical development (01.01.2016)
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Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease
Poletti, Valentina, Urbinati, Fabrizia, Charrier, Sabine, Corre, Guillaume, Hollis, Roger P, Campo Fernandez, Beatriz, Martin, Samia, Rothe, Michael, Schambach, Axel, Kohn, Donald B, Mavilio, Fulvio
Published in Molecular therapy. Methods & clinical development (14.12.2018)
Published in Molecular therapy. Methods & clinical development (14.12.2018)
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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
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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Vectofusin-1, a New Viral Entry Enhancer, Strongly Promotes Lentiviral Transduction of Human Hematopoietic Stem Cells
Fenard, David, Ingrao, Dina, Seye, Ababacar, Buisset, Julien, Genries, Sandrine, Martin, Samia, Kichler, Antoine, Galy, Anne
Published in Molecular therapy. Nucleic acids (2013)
Published in Molecular therapy. Nucleic acids (2013)
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An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
Weber, Leslie, Poletti, Valentina, Magrin, Elisa, Antoniani, Chiara, Martin, Samia, Bayard, Charles, Sadek, Hanem, Felix, Tristan, Meneghini, Vasco, Antoniou, Michael N., El-Nemer, Wassim, Mavilio, Fulvio, Cavazzana, Marina, Andre-Schmutz, Isabelle, Miccio, Annarita
Published in Molecular therapy. Methods & clinical development (21.09.2018)
Published in Molecular therapy. Methods & clinical development (21.09.2018)
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A Conserved Mechanism of Retrovirus Restriction in Mammals
Towers, Greg, Bock, Michael, Martin, Samia, Takeuchi, Yasuhiro, Stoye, Jonathan P., Danos, Olivier
Published in Proceedings of the National Academy of Sciences - PNAS (24.10.2000)
Published in Proceedings of the National Academy of Sciences - PNAS (24.10.2000)
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Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency
Soheili, Tayebeh, Durand, Amandine, Sepulveda, Fernando E., Rivière, Julie, Lagresle-Peyrou, Chantal, Sadek, Hanem, de Saint Basile, Geneviève, Martin, Samia, Mavilio, Fulvio, Cavazzana, Marina, André-Schmutz, Isabelle
Published in Blood advances (26.12.2017)
Published in Blood advances (26.12.2017)
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Safety and Efficacy of AAV-Mediated Calpain 3 Gene Transfer in a Mouse Model of Limb-Girdle Muscular Dystrophy Type 2A
Bartoli, Marc, Roudaut, Carinne, Martin, Samia, Fougerousse, Françoise, Suel, Laurence, Poupiot, Jérôme, Gicquel, Evelyne, Noulet, Fanny, Danos, Olivier, Richard, Isabelle
Published in Molecular therapy (01.02.2006)
Published in Molecular therapy (01.02.2006)
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Highly efficient transduction of human plasmacytoid dendritic cells without phenotypic and functional maturation
Veron, Philippe, Boutin, Sylvie, Martin, Samia, Chaperot, Laurence, Plumas, Joel, Davoust, Jean, Masurier, Carole
Published in Journal of translational medicine (27.01.2009)
Published in Journal of translational medicine (27.01.2009)
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Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors
Veron, Philippe, Leborgne, Christian, Monteilhet, Virginie, Boutin, Sylvie, Martin, Samia, Moullier, Philippe, Masurier, Carole
Published in The Journal of immunology (1950) (15.06.2012)
Published in The Journal of immunology (1950) (15.06.2012)
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Liver-directed lentiviral gene therapy in a dog model of hemophilia B
Cantore, Alessio, Ranzani, Marco, Bartholomae, Cynthia C, Volpin, Monica, Valle, Patrizia Della, Sanvito, Francesca, Sergi, Lucia Sergi, Gallina, Pierangela, Benedicenti, Fabrizio, Bellinger, Dwight, Raymer, Robin, Merricks, Elizabeth, Bellintani, Francesca, Martin, Samia, Doglioni, Claudio, D'Angelo, Armando, VandenDriessche, Thierry, Chuah, Marinee K, Schmidt, Manfred, Nichols, Timothy, Montini, Eugenio, Naldini, Luigi
Published in Science translational medicine (04.03.2015)
Published in Science translational medicine (04.03.2015)
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Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy
Childers, Martin K, Joubert, Romain, Poulard, Karine, Moal, Christelle, Grange, Robert W, Doering, Jonathan A, Lawlor, Michael W, Rider, Branden E, Jamet, Thibaud, Danièle, Nathalie, Martin, Samia, Rivière, Christel, Soker, Thomas, Hammer, Caroline, Van Wittenberghe, Laetitia, Lockard, Mandy, Guan, Xuan, Goddard, Melissa, Mitchell, Erin, Barber, Jane, Williams, J Koudy, Mack, David L, Furth, Mark E, Vignaud, Alban, Masurier, Carole, Mavilio, Fulvio, Moullier, Philippe, Beggs, Alan H, Buj-Bello, Anna
Published in Science translational medicine (22.01.2014)
Published in Science translational medicine (22.01.2014)
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131. Targeted Genome Editing in Spinal Muscular Atrophy
Lattanzi, Annalisa, Bovolenta, Matteo, Martin, Samia, Mouly, Vincent, Mavilio, Fulvio
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice
Danièle, Nathalie, Moal, Christelle, Julien, Laura, Marinello, Martina, Jamet, Thibaud, Martin, Samia, Vignaud, Alban, Lawlor, Michael, Buj-Bello, Ana
Published in Journal of neuropathology and experimental neurology (01.04.2018)
Published in Journal of neuropathology and experimental neurology (01.04.2018)
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Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice
Danièle, Nathalie, Moal, Christelle, Julien, Laura, Marinello, Martina, Jamet, Thibaud, Martin, Samia, Vignaud, Alban, Lawlor, Michael W, Buj-Bello, Ana
Published in Journal of neuropathology and experimental neurology (01.04.2018)
Published in Journal of neuropathology and experimental neurology (01.04.2018)
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