Uptake of moss‐derived human recombinant GAA in Gaa (−/−) mice

Pompe disease, an autosomal recessive lysosomal storage disorder, is caused by deficiency of lysosomal acid alpha‐glucosidase (GAA). On cellular level, there is lysosomal‐bound and free accumulation of glycogen and subsequent damage of organelles and organs. The most severe affected tissues are skel...

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Detalles Bibliográficos
Autores principales: Hintze, Stefan, Dabrowska‐Schlepp, Paulina, Berg, Birgit, Graupner, Alexandra, Busch, Andreas, Schaaf, Andreas, Schoser, Benedikt, Meinke, Peter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2021
Materias:
Research Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8100399/
https://www.ncbi.nlm.nih.gov/pubmed/33977033
http://dx.doi.org/10.1002/jmd2.12203

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8100399/
https://www.ncbi.nlm.nih.gov/pubmed/33977033
http://dx.doi.org/10.1002/jmd2.12203

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