The generation of animal models to design novel gene correction approaches for Pompe disease
The generation of animal models to design novel gene correction approaches for Pompe disease
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Author / Creator
Geel, M , Niezen-Koning, K , Ruiters, M , Voorn, P , McLaughlin, P , Pingout, A , Siksnys, V and Rots, M
Journal title
Language
English
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Scope and Contents
Contents
Background: Pompe disease is a lysosomal storage disease caused by autosomal recessive mutations in the acid-a-glucosidase (GAA) gene. The GAA deficiency results in glycogen accumulation in the lysosomes, often causing death. The only available therapy is enzyme replacement therapy. Several gene therapeutic approaches can be envisioned for this dis...
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Full title
The generation of animal models to design novel gene correction approaches for Pompe disease
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Record Identifier
TN_cdi_proquest_miscellaneous_19803809
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_19803809
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ISSN
1043-0342
