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Murine Muscle Cell Models for Pompe Disease and Their Use in Studying Therapeutic Approaches

Lysosomes filled with glycogen are a major pathologic feature of Pompe disease, a fatal myopathy and cardiomyopathy caused by a deficiency of the glycogen-degrading lysosomal enzyme, acid α-glucosidase (GAA). To facilitate studies germane to this genetic disorder, we developed two in vitro Pompe mod...

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Detaylı Bibliyografya
Asıl Yazarlar: Takikita, Shoichi, Myerowitz, Rachel, Zaal, Kristien, Raben, Nina, Plotz, Paul H.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2009
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC2680079/
https://ncbi.nlm.nih.gov/pubmed/19167256
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2008.12.012
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