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Induction of antigen-specific tolerance through hematopoietic stem cell-mediated gene therapy: The future for therapy of autoimmune disease?

✍ Scribed by Coleman, Miranda A.; Steptoe, Raymond J.

Book ID
118426127
Publisher
Elsevier Science
Year
2012
Tongue
English
Weight
273 KB
Volume
12
Category
Article
ISSN
1568-9972

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## Abstract ## Background Glycogen storage disease type II (GSDII) or Pompe disease is an inherited disease of glycogen metabolism caused by a lack of functional lysosomal acid α‐glucosidase (GAA). Affected individuals store glycogen in lysosomes resulting in fatal hypertrophic cardiomyopathy and