Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

por Hocquemiller, Michaël, Giersch, Laura, Audrain, Mickael, Parker, Samantha, Cartier, Nathalie
Publicado 2016

AAV‐delivered diacylglycerol kinase DGKk achieves long‐term rescue of fragile X syndrome mouse model

por Habbas, Karima, Cakil, Oktay, Zámbó, Boglárka, Tabet, Ricardos, Riet, Fabrice, Dembele, Doulaye, Mandel, Jean‐Louis, Hocquemiller, Michaël, Laufer, Ralph, Piguet, Françoise, Moine, Hervé
Publicado 2022

AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates

por 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
Publicado 2022

AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains

por 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
Publicado 2019

Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA

por Bugiani, Marianna, Abbink, Truus E. M., Edridge, Arthur W. D., van der Hoek, Lia, Hillen, Anne E. J., van Til, Niek P., Hu‐A‐Ng, Gino V., Breur, Marjolein, Aiach, Karen, Drevot, Philippe, Hocquemiller, Michaël, Laufer, Ralph, Wijburg, Frits A., van der Knaap, Marjo S.
Publicado 2023