Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy

by Emilie Audouard, Nicolas Khefif, Charlotte Mansat, Océane Nelcha, Elena-Gaia Banchi, Camille Lupiet, Dominique Farabos, Antonin Lamaziere, Caroline Sevin, Françoise Piguet
Published 2024-06-01

Modulation of Brain Cholesterol Metabolism through CYP46A1 Overexpression for Rett Syndrome

by Emilie Audouard, Nicolas Khefif, Béatrix Gillet-Legrand, Fanny Nobilleau, Ouafa Bouazizi, Serena Stanga, Gaëtan Despres, Sandro Alves, Antonin Lamazière, Nathalie Cartier, Françoise Piguet
Published 2024-06-01