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Glycogen Reduction in Myotubes of Late-Onset Pompe Disease Patients Using Antisense Technology
Glycogen storage disease type II (GSDII) is a lysosomal disorder caused by the deficient activity of acid alpha-glucosidase (GAA) enzyme, leading to the accumulation of glycogen within the lysosomes. The disease has been classified in infantile and late-onset forms. Most late-onset patients share a...
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| Vydáno v: | Mol Ther |
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| Hlavní autoři: | , , , , |
| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
American Society of Gene & Cell Therapy
2017
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| Témata: | |
| On-line přístup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5589062/ https://ncbi.nlm.nih.gov/pubmed/28629821 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.05.019 |
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