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AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates
Michaël Hocquemiller, Laura Giersch, Xin Mei, Amanda L. Gross, Ashley N. Randle, Heather L. Gray-Edwards, Judith A. Hudson, Sophia Todeasa, Lorelei Stoica, Douglas R. Martin, Miguel Sena-Esteves, Karen Aiach and Ralph Laufer
Mol Ther Methods Clin Dev. 27: 281-292 (2022)
AAV-delivered diacylglycerol kinase DGKk achieves long-term rescue of fragile X syndrome mouse model
Karima Habbas, Oktay Cakil, Boglárka Zámbó, Ricardos Tabet, Fabrice Riet, Doulaye Dembele, Jean-Louis Mandel, Michaël Hocquemiller, Ralph Laufer, Françoise Piguet, Hervé Moine.
EMBO Mol Med (2022)
Understanding disease symptoms and impacts and producing qualitatively-derived severity stages for MPS IIIA: a mixed methods approach
Sally Lanar, Samantha Parker, Cara O’Neill, Alexia Marrel, Benoit Arnould, Bénédicte Héron, Nicole Muschol, Frits A Wijburg, Anupam Chakrapani, Sophie Olivier, Karen Aiach.
Orphanet J Rare Dis 17, 75 (2022)
AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains
Michaël Hocquemiller, Kim M. Hemsley, Meghan L. Douglass, Sarah J. Tamang, Daniel Neumann, Barbara M. King, Helen Beard, Paul J. Trim, Leanne K. Winner, Adeline A. Lau, Marten F. Snel, Cathy Gomila, Jérôme Ausseil, Xin Mei, Laura Giersch, Mark Plavsic, and Ralph Laufer.
Mol Ther Methods Clin Dev. 17:174-187 (2020)
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
Michaël Hocquemiller, Laura Giersch, Mickael Audrain, Samantha Parker, and Nathalie Cartier.
Hum Gene Ther. 27:478–496 (2016)
An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA.
Gray A, O’Leary C, Liao A, Agúndez L, Youshani A, Gleitz H, Parker H, Taylor J, Danos 0, Hocquemiller M, Palomar N, Linden M, Henckaerts E, Holley R, and Bigger B.
Hum Gene Ther. 30: 1052-1066 (2019)
A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome.
Winner L, Beard H, Hassiotis S, Lau A., Luck A, Hopwood J, Hemsley K.M.
Hum Gene Ther. 27, 363–375 (2016)
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 M, Zérah M, Husson H, de Bournonville S, Deiva K, Adamsbaum C, Vincent F, Hocquemiller M, Broissand C, Furlan F, Ballabio A, Fraldi A, Crystal R, Baugnon T, Roujeau T, Heard J.M, Danos O.
Hum Gene Ther. 25:506–516 (2014)