Skeletal muscle expression of clathrin and mannose 6-phosphate receptor in experimental chloroquine-induced myopathy

Antibody formation and mannose-6-phospha

Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease

✍ Baodong Sun; Songtao Li; Andrew Bird; Haiqing Yi; Alex Kemper; Beth L. Thurberg; 📂 Article 📅 2010 🏛 John Wiley and Sons 🌐 English ⚖ 460 KB

## Abstract ## Background Lysosomal storage disorders such as Pompe disease can be more effectively treated, if immune tolerance to enzyme or gene replacement therapy can be achieved. Alternatively, immune responses against acid α‐glucosidase (GAA) might be evaded in Pompe disease through muscle‐s