Michael Hocquemiller
Neuilly-sur-seine, France
Last Updated: 09/26/2026
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Found 9 publications
Tandem mass spectrometry-based assay for heparan-N-sulphatase in paediatric CSF: A potential pharmacodynamic biomarker for mucopolysaccharidosis type IIIA therapy.
Journal: Clinica chimica acta; international journal of clinical chemistry
Published: July 29, 2024
Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA.
Journal: Annals of clinical and translational neurology
Published: January 14, 2023
AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates.
Journal: Molecular therapy. Methods & clinical development
Published: July 12, 2022
AAV-delivered diacylglycerol kinase DGKk achieves long-term rescue of fragile X syndrome mouse model.
Journal: EMBO molecular medicine
Published: June 03, 2021
AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains.
Journal: Molecular therapy. Methods & clinical development
Published: August 13, 2019
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.
Journal: Human gene therapy
Published: June 09, 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.
Journal: Human gene therapy
Published: February 15, 2014
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Last Updated: 09/26/2026