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Mannose-6-Phosphate receptor expression modulates efficacy from enzyme replacement therapy in Pompe disease

✍ Scribed by Dwight Koeberl; Baodong Sun; Alison McVie-Wylie; Jian Dai; Songtao Li; Y.-T. Chen; Deeksha Bali

Book ID: 116989182
Publisher: Elsevier Science
Year: 2011
Tongue: English
Weight: 59 KB
Volume: 102
Category: Article
ISSN: 1096-7192
DOI: 10.1016/j.ymgme.2010.11.085

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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