Efficacy of Gene Therapy for a Prototypical Lysosomal Storage Disease (GSD-II) Is Critically Dependent on Vector Dose, Transgene Promoter, and the Tissues Targeted for Vector Transduction
Titel:
Efficacy of Gene Therapy for a Prototypical Lysosomal Storage Disease (GSD-II) Is Critically Dependent on Vector Dose, Transgene Promoter, and the Tissues Targeted for Vector Transduction
Auteur:
Ding, Enyu Hu, Huimin Hodges, Bradley L. Migone, Felicia Serra, Delila Xu, Fang Chen, Yuan-Tsong Amalfitano, Andrea