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Moss-Derived Human Recombinant GAA Provides an Optimized Enzyme Uptake in Differentiated Human Muscle Cells of Pompe Disease

Pompe disease is an autosomal recessive lysosomal storage disorder (LSD) caused by deficiency of lysosomal acid alpha-glucosidase (GAA). The result of the GAA deficiency is a ubiquitous lysosomal and non-lysosomal accumulation of glycogen. The most affected tissues are heart, skeletal muscle, liver,...

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Detalles Bibliográficos
Autores principales:	Hintze, Stefan, Limmer, Sarah, Dabrowska-Schlepp, Paulina, Berg, Birgit, Krieghoff, Nicola, Busch, Andreas, Schaaf, Andreas, Meinke, Peter, Schoser, Benedikt
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7177967/ https://www.ncbi.nlm.nih.gov/pubmed/32290314 http://dx.doi.org/10.3390/ijms21072642

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