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Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
OBJECTIVE: We have recently reported on the pathology of the neuromuscular junction (NMJ) in Pompe disease, reflecting disruption of neuronal and muscle homeostasis as a result of glycogen accumulation. The aim of this study was to examine how the alteration of NMJ physiology contributes to Pompe di...
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| Foilsithe in: | Ann Neurol |
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| Main Authors: | , , , , , , |
| Formáid: | Artigo |
| Teanga: | Inglês |
| Foilsithe: |
2015
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| Ábhair: | |
| Rochtain Ar Líne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4520217/ https://ncbi.nlm.nih.gov/pubmed/25925726 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24433 |
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