[OC-05] Gene & Cell Therapy for Neurological Diseases

[OC-05-2] AAV mediated gene therapy targeting brain cholesterol for Huntington disease

^○Nathalie Cartier¹, Sandro Alves², Wilhelm Christaller³, Enejda Subashi⁴, Radhia Kacher⁵, Antonin Lamazière², Gaetan Despres⁶, Frederic Saudou³, Jocelyne Caboche⁵, Sandrine Betuing⁵ (1.INSERM UMR1169 Université Paris Saclay, France, 2.BrainVectis Therapeutics, France, 3.INSERM U1216 and University Grenoble Alpes, Grenoble Institut des Neurosciences, France, 4.INSERM UMR1169 Université Paris Saclay, France, 5.Institute of Biology Paris-Seine, Sorbonne Universités, France, 6.Laboratory of Mass Spectrometry, INSERM ERL 1157, Sorbonne Universités-)

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 and is Co-PI in gene therapy clinical trials for Adrenoleukodystrophy and metachromatic leukodystrophy.
Her research group (INSERM U1169) located in Paris at the CEA (Commission for Atomic Energy), in the Molecular Imaging center (MIRCen) focuses on gene therapy for Huntington‘s disease, Alzheimer’s diseases, spinocerebellar ataxias. Her lab is particularly interested in modulation of brain cholesterol metabolism.

Nathalie Cartier was member of the Scientific Committee of INSERM and former President of the European Society for Gene and Cell Therapy (ESGCT) and French Society for Cell and Gene Therapy. She received from the French Government the Medal of the Legion d’Honneur in 2010. She is currently President of the scientific committee of the French Foundation for Rare Diseases.