Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice

We have used a peptide-based targeting system to improve lysosomal delivery of acid α-glucosidase (GAA), the enzyme deficient in patients with Pompe disease. Human GAA was fused to the glycosylation-independent lysosomal targeting (GILT) tag, which contains a portion of insulin-like growth factor II...

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Bibliographische Detailangaben
Hauptverfasser: Maga, John A., Zhou, Jianghong, Kambampati, Ravi, Peng, Susan, Wang, Xu, Bohnsack, Richard N., Thomm, Angela, Golata, Sarah, Tom, Peggy, Dahms, Nancy M., Byrne, Barry J., LeBowitz, Jonathan H.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Biochemistry and Molecular Biology 2013
Schlagworte:
Molecular Bases of Disease
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3548456/
https://ncbi.nlm.nih.gov/pubmed/23188827
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1074/jbc.M112.438663
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