scholarly journals 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

2002 ◽  
Vol 5 (4) ◽  
pp. 436-446 ◽  
Author(s):  
Enyu Ding ◽  
Huimin Hu ◽  
Bradley L. Hodges ◽  
Felicia Migone ◽  
Delila Serra ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Lysosomal Storage Disease ◽  
Storage Disease ◽  
Lysosomal Storage ◽  
Vector Transduction ◽  
Gsd Ii

scholarly journals Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment

2012 ◽  
Vol 23 (8) ◽  
pp. 808-815 ◽  
Author(s):  
Barry J. Byrne ◽  
Darin J. Falk ◽  
Nathalie Clément ◽  
Cathryn S. Mah
Keyword(s):  
Gene Therapy ◽  
Lysosomal Storage Disease ◽  
Storage Disease ◽  
Next Generation ◽  
Lysosomal Storage

Gene therapy for lysosomal storage disease: A no-brainer?

1997 ◽  
Vol 3 (7) ◽  
pp. 719-720 ◽  
Author(s):  
William S. Sly ◽  
Carole Vogler
Keyword(s):  
Gene Therapy ◽  
Lysosomal Storage Disease ◽  
Storage Disease ◽  
Lysosomal Storage
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