Speaker

Nathalie Cartier obtained her Medical degree from the University René Descartes in Paris where she completed her residency and specialized in Pediatrics. She became Research Director at INSERM and associate Professor at University Descartes.  

Her research interest focuses on the development of gene therapy approaches for neurodegenerative diseases. She developed with Patrick Aubourg the pioneer work on hematopoietic stem cell gene therapy for X-linked Adrenoleukodystrophy, first clinical trial using an HIV-derived lentiviral vector.  Her research group (INSERM U1127) located in Paris at the Pitié Salpêtrière Hospital in the Institute for Brain and Spine (ICM) develops gene and cell therapy strategies for genetic and complex neurodegenerative diseases, leukodystrophies, Huntington’s and Alzheimer’s diseases, Amyotrophic Lateral Sclerosis  based on improving defects of brain metabolism.

Her work includes all translation steps from preclinical proof of concept in animal models to clinical application in human patients: vector development, animal models characterization, gene transfer and validation in animal models, submission of clinical application to regulatory agencies, clinical trials in human patients. Nathalie Cartier is Co-PI in gene therapy clinical trials for Adrenoleukodystrophy and metachromatic leukodystrophy.
She was member of the Scientific Committee of INSERM. She is President of the the scientific committee of the Fondation Maladies Rares,  and former President of the European Society for Gene and Cell Therapy (ESGCT) and of the French Society for Cell and Gene Therapy.

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