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Immunomodulatory, Liver Depot Gene Therapy for Pompe Disease

Pompe disease is caused by mutations in acid alpha glucosidase (GAA) that causes accumulation of lysosomal glycogen affecting the heart and skeletal muscles, and can be fatal. Enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA) improves muscle function by reducing glycogen accumulati...

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Bibliografiska uppgifter
I publikationen:Cell Immunol
Huvudupphovsmän: Bond, JE, Kishnani, PS, Koeberl, DD
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2017
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC6026080/
https://ncbi.nlm.nih.gov/pubmed/29295737
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.cellimm.2017.12.011
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