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Team “Gene Therapy”


Gene therapy Main domain: Clinical & translational neurosciences Subdomain : Molecular & cellular neuroscience Nathalie CARTIER’s team develops gene therapy strategies for severe neurodegenerative diseases including Huntington's disease, Spinocerebellar ataxias, Alzheimer's disease and genetic leukodystrophies. Research includes proof of concept in animal models and translational steps to clinical applications. The team aims to propose a phase I/II therapeutic trial in 2020 for Huntington disease. The team will also develop tools for the delivery of therapeutic molecules in the brain (use of microglia, optimization of AAV for intravenous administration, optogenerapy for time-controlled delivery).

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