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Ultrastructural and diffusion tensor imaging studies reveal axon abnormalities in Pompe disease mice
Pompe disease (PD) is caused by lysosomal glycogen accumulation in tissues, including muscles and the central nervous system (CNS). The intravenous infusion of recombinant human acid alpha-glucosidase (rhGAA) rescues the muscle pathologies in PD but does not treat the CNS because rhGAA does not cros...
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| Pubblicato in: | Sci Rep |
|---|---|
| Autori principali: | , , , , , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Nature Publishing Group UK
2020
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7677380/ https://ncbi.nlm.nih.gov/pubmed/33214573 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-020-77193-w |
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