Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial
Tardieu, Marc, Zérah, Michel, Husson, Béatrice, de Bournonville, Stéphanie, Deiva, Kumaran, Adamsbaum, Catherine, Vincent, Fanny, Hocquemiller, Michael +9 more·2014·Human Gene Therapy
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