Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
Titel:
Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
Auteur:
Tardieu, Marc Zérah, Michel Gougeon, Marie-Lise Ausseil, Jérome de Bournonville, Stéphanie Husson, Béatrice Zafeiriou, Dimitrios Parenti, Giancarlo Bourget, Philippe Poirier, Béatrice Furlan, Valérie Artaud, Cécile Baugnon, Thomas Roujeau, Thomas Crystal, Ronald G Meyer, Christian Deiva, Kumaran Heard, Jean-Michel