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Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

✍ Scribed by Dwight D. Koeberl; Xiaoyan Luo; Baodong Sun; Alison McVie-Wylie; Jian Dai; Songtao Li; Suhrad G. Banugaria; Y.-T. Chen; Deeksha S. Bali

Book ID
116989297
Publisher
Elsevier Science
Year
2011
Tongue
English
Weight
933 KB
Volume
103
Category
Article
ISSN
1096-7192

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## 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