Assessment of adeno-associated virus gene therapies efficacy on acid alpha-glucosidase restoration and glycogen storage correction in cardiac muscle of Pompe disease mice using synchrotron infrared and ultraviolet microspectroscopies
Pompe disease (glycogen storage disease type II) is a lysosomal storage disorder due to a mutation in the gene that encodes acid alpha-glucosidase (GAA). GAA deficiency causes the excessive storage of lysosomal glycogen in many cell types, leading to cell and, subsequently, tissue dysfunction. Cardi...
| 主要な著者: | , , , , , , , , , , |
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| フォーマット: | 論文 |
| 言語: | English |
| 出版事項: |
IM Publications Open
2019-08-01
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| シリーズ: | Journal of Spectral Imaging |
| 主題: | |
| オンライン･アクセス: | https://www.impopen.com/download.php?code=I08_a13 |