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Large Animal Models of Lysosomal Storage Diseases: Lessons on the Limits of Gene/Enzyme Therapy

✍ Scribed by Mark Haskins; Meg Sleeper; Gus Aguirre; Steven U. Walkley; Van Knox; Charles Vite; Richard Steet; Brittney Gurda; Jim Wilson; Alberto Auricchio; Lachlan Smith; Lilla Simonaro; Katherine Ponder


Book ID
116989557
Publisher
Elsevier Science
Year
2012
Tongue
English
Weight
65 KB
Volume
105
Category
Article
ISSN
1096-7192

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Gene therapy for lysosomal storage disea
✍ N. Matthew Ellinwood; Charles H. Vite; Mark E. Haskins πŸ“‚ Article πŸ“… 2004 πŸ› John Wiley and Sons 🌐 English βš– 596 KB

## Abstract There are more than 40 different forms of inherited lysosomal storage diseases (LSDs) known to occur in humans and the aggregate incidence has been estimated to approach 1 in 7000 live births. Most LSDs are associated with high morbidity and mortality and represent a significant burden