AAV‐delivered diacylglycerol kinase DGKk achieves long‐term rescue of fragile X syndrome mouse model
Habbas, Karima, Cakil, Oktay, Zámbó, Boglárka, Tabet, Ricardos, Riet, Fabrice, Dembele, Doulaye, Mandel, Jean‐Louis, Hocquemiller, Michaël, Laufer, Ralph, Piguet, Françoise, Moine, Hervé
Published in EMBO molecular medicine (09.05.2022)
Published in EMBO molecular medicine (09.05.2022)
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Journal Article
An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA
Gray, Anna L, O'Leary, Claire, Liao, Aiyin, Agúndez, Leticia, Youshani, Amir S, Gleitz, Hélène F, Parker, Helen, Taylor, Jessica T, Danos, Olivier, Hocquemiller, Michaël, Palomar, Nuria, Linden, R Michael, Henckaerts, Els, Holley, Rebecca J, Bigger, Brian W
Published in Human gene therapy (01.09.2019)
Published in Human gene therapy (01.09.2019)
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Design of a stable human acid‐β‐glucosidase: towards improved Gaucher disease therapy and mutation classification
Pokorna, Sarka, Khersonsky, Olga, Lipsh‐Sokolik, Rosalie, Goldenzweig, Adi, Nielsen, Rebekka, Ashani, Yacov, Peleg, Yoav, Unger, Tamar, Albeck, Shira, Dym, Orly, Tirosh, Asa, Tarayra, Rana, Hocquemiller, Michaël, Laufer, Ralph, Ben‐Dor, Shifra, Silman, Israel, Sussman, Joel L., Fleishman, Sarel J., Futerman, Anthony H.
Published in The FEBS journal (01.07.2023)
Published in The FEBS journal (01.07.2023)
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Journal Article
AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates
Hocquemiller, Michaël, Giersch, Laura, Mei, Xin, Gross, Amanda L., Randle, Ashley N., Gray-Edwards, Heather L., Hudson, Judith A., Todeasa, Sophia, Stoica, Lorelei, Martin, Douglas R., Sena-Esteves, Miguel, Aiach, Karen, Laufer, Ralph
Published in Molecular therapy. Methods & clinical development (08.12.2022)
Published in Molecular therapy. Methods & clinical development (08.12.2022)
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AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains
Hocquemiller, Michaël, Hemsley, Kim M., Douglass, Meghan L., Tamang, Sarah J., Neumann, Daniel, King, Barbara M., Beard, Helen, Trim, Paul J., Winner, Leanne K., Lau, Adeline A., Snel, Marten F., Gomila, Cathy, Ausseil, Jérôme, Mei, Xin, Giersch, Laura, Plavsic, Mark, Laufer, Ralph
Published in Molecular therapy. Methods & clinical development (12.06.2020)
Published in Molecular therapy. Methods & clinical development (12.06.2020)
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Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA
Bugiani, Marianna, Abbink, Truus E. M., Edridge, Arthur W. D., Hoek, Lia, Hillen, Anne E. J., Til, Niek P., Hu‐A‐Ng, Gino V., Breur, Marjolein, Aiach, Karen, Drevot, Philippe, Hocquemiller, Michaël, Laufer, Ralph, Wijburg, Frits A., Knaap, Marjo S.
Published in Annals of clinical and translational neurology (01.06.2023)
Published in Annals of clinical and translational neurology (01.06.2023)
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Journal Article
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
Hocquemiller, Michaël, Giersch, Laura, Audrain, Mickael, Parker, Samantha, Cartier, Nathalie
Published in Human gene therapy (01.07.2016)
Published in Human gene therapy (01.07.2016)
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Journal Article
Storage Vesicles in Neurons Are Related to Golgi Complex Alterations in Mucopolysaccharidosis IIIB
Vitry, Sandrine, Bruyère, Julie, Hocquemiller, Michaël, Bigou, Stéphanie, Ausseil, Jérôme, Colle, Marie-Anne, Prévost, Marie-Christine, Heard, Jean Michel
Published in The American journal of pathology (01.12.2010)
Published in The American journal of pathology (01.12.2010)
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Journal Article
α- l-Iduronidase transport in neurites
Chen, Fengtian, Vitry, Sandrine, Hocquemiller, Michaël, Desmaris, Nathalie, Ausseil, Jérôme, Heard, Jean-Michel
Published in Molecular genetics and metabolism (01.04.2006)
Published in Molecular genetics and metabolism (01.04.2006)
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Directed Evolution of Motor Neurons from Genetically Engineered Neural Precursors
Bohl, Delphine, Liu, Song, Blanchard, Stéphane, Hocquemiller, Michaël, Haase, Georg, Heard, Jean‐Michel
Published in Stem cells (Dayton, Ohio) (01.10.2008)
Published in Stem cells (Dayton, Ohio) (01.10.2008)
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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 of a phase I/II trial
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
Published in Human gene therapy (01.06.2014)
Published in Human gene therapy (01.06.2014)
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Journal Article
GAP43 overexpression and enhanced neurite outgrowth in mucopolysaccharidosis type IIIB cortical neuron cultures
Hocquemiller, Michaël, Vitry, Sandrine, Bigou, Stéphanie, Bruyère, Julie, Ausseil, Jérôme, Heard, Jean Michel
Published in Journal of neuroscience research (01.01.2010)
Published in Journal of neuroscience research (01.01.2010)
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Journal Article
AAV-delivery of diacylglycerol kinase kappa achieves long-term rescue of Fmr1-KO mouse model deficits of fragile X syndrome
Habbas, Karima, Oktay Cakil, Zambo, Boglarka, Tabet, Ricardos, Fabrice, Fabrice, Dembele, Doulaye, Mandel, Jean-Louis, Hocquemiller, Michael, Laufer, Ralph, Piguet, Francoise, Moine, Herve
Published in bioRxiv (14.04.2021)
Published in bioRxiv (14.04.2021)
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Paper
Enhanced degradation of synaptophysin by the proteasome in mucopolysaccharidosis type IIIB
Vitry, Sandrine, Ausseil, Jérôme, Hocquemiller, Michael, Bigou, Stéphanie, dos Santos Coura, Renata, Heard, Jean Michel
Published in Molecular and cellular neuroscience (01.05.2009)
Published in Molecular and cellular neuroscience (01.05.2009)
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Journal Article
Tandem mass spectrometry-based assay for heparan-N-sulphatase in paediatric CSF: A potential pharmacodynamic biomarker for mucopolysaccharidosis type IIIA therapy
Speziale, Roberto, Hocquemiller, Michaël, Mei, Xin, Fabbrini, Danilo, Malancona, Savina, Aiach, Karen, Laufer, Ralph, Orsatti, Laura
Published in Clinica chimica acta (03.10.2024)
Published in Clinica chimica acta (03.10.2024)
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