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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).
https://doi.org/10.1089/hum.2018.189
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)
https://doi.org/10.1089/hum.2015.170
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).
https://doi.org/10.1089/hum.2013.238