Cofilin-1 phosphorylation catalyzed by ERK1/2 alters cardiac actin dynamics in dilated cardiomyopathy caused by lamin A/C gene mutation
Chatzifrangkeskou, Maria, Yadin, David, Marais, Thibaut, Chardonnet, Solenne, Cohen-Tannoudji, Mathilde, Mougenot, Nathalie, Schmitt, Alain, Crasto, Silvia, Di Pasquale, Elisa, Macquart, Coline, Tanguy, Yannick, Jebeniani, Imen, Pucéat, Michel, Morales Rodriguez, Blanca, Goldmann, Wolfgang H, Dal Ferro, Matteo, Biferi, Maria-Grazia, Knaus, Petra, Bonne, Gisèle, Worman, Howard J, Muchir, Antoine
Published in Human molecular genetics (01.09.2018)
Published in Human molecular genetics (01.09.2018)
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Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice
Pontoizeau, Clément, Simon-Sola, Marcelo, Gaborit, Clovis, Nguyen, Vincent, Rotaru, Irina, Tual, Nolan, Colella, Pasqualina, Girard, Muriel, Biferi, Maria-Grazia, Arnoux, Jean-Baptiste, Rötig, Agnès, Ottolenghi, Chris, de Lonlay, Pascale, Mingozzi, Federico, Cavazzana, Marina, Schiff, Manuel
Published in Nature communications (07.06.2022)
Published in Nature communications (07.06.2022)
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The non-muscle ADF/cofilin-1 controls sarcomeric actin filament integrity and force production in striated muscle laminopathies
Vignier, Nicolas, Chatzifrangkeskou, Maria, Pinton, Luca, Wioland, Hugo, Marais, Thibaut, Lemaitre, Mégane, Le Dour, Caroline, Peccate, Cécile, Cardoso, Déborah, Schmitt, Alain, Wu, Wei, Biferi, Maria-Grazia, Naouar, Naïra, Macquart, Coline, Beuvin, Maud, Decostre, Valérie, Bonne, Gisèle, Romet-Lemonne, Guillaume, Worman, Howard J., Tedesco, Francesco Saverio, Jégou, Antoine, Muchir, Antoine
Published in Cell reports (Cambridge) (24.08.2021)
Published in Cell reports (Cambridge) (24.08.2021)
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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
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
Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins
Alves, Sandro, Marais, Thibaut, Biferi, Maria-Grazia, Furling, Denis, Marinello, Martina, El Hachimi, Khalid, Cartier, Nathalie, Ruberg, Merle, Stevanin, Giovanni, Brice, Alexis, Barkats, Martine, Sittler, Annie
Published in Molecular neurodegeneration (28.07.2016)
Published in Molecular neurodegeneration (28.07.2016)
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Journal Article
Gene Therapy for ALS-A Perspective
Cappella, Marisa, Ciotti, Chiara, Cohen-Tannoudji, Mathilde, Biferi, Maria Grazia
Published in International journal of molecular sciences (06.09.2019)
Published in International journal of molecular sciences (06.09.2019)
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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
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
Correction to: 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 (01.10.2023)
Published in Acta neuropathologica (01.10.2023)
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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
β-Amyloid Precursor Protein Intracellular Domain Controls Mitochondrial Function by Modulating Phosphatase and Tensin Homolog–Induced Kinase 1 Transcription in Cells and in Alzheimer Mice Models
Goiran, Thomas, Duplan, Eric, Chami, Mounia, Bourgeois, Alexandre, El Manaa, Wejdane, Rouland, Lila, Dunys, Julie, Lauritzen, Inger, You, Han, Stambolic, Vuk, Biféri, Maria-Grazia, Barkats, Martine, Pimplikar, Sanjay W, Sergeant, Nicolas, Colin, Morvane, Morais, Vanessa A, Pardossi-Piquard, Raphaelle, Checler, Frédéric, Alves da Costa, Cristine
Published in Biological psychiatry (1969) (01.03.2018)
Published in Biological psychiatry (1969) (01.03.2018)
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Journal Article
Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice
Lisjak, Michela, De Caneva, Alessia, Marais, Thibaut, Barbon, Elena, Biferi, Maria Grazia, Porro, Fabiola, Barzel, Adi, Zentilin, Lorena, Kay, Mark A, Mingozzi, Federico, Muro, Andrés F
Published in Frontiers in genome editing (11.03.2022)
Published in Frontiers in genome editing (11.03.2022)
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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
AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice
Besse, Aurore, Astord, Stephanie, Marais, Thibaut, Roda, Marianne, Giroux, Benoit, Lejeune, François-Xavier, Relaix, Frederic, Smeriglio, Piera, Barkats, Martine, Biferi, Maria Grazia
Published in Molecular therapy (05.08.2020)
Published in Molecular therapy (05.08.2020)
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