rAAV2-Mediated Restoration of GALC in Neural Stem Cells from Krabbe Patient-Derived iPSCs
Krabbe disease is a rare neurodegenerative fatal disease. It is caused by deficiency of the lysosomal enzyme galactocerebrosidase (GALC), which results in progressive accumulation of galactolipid substrates in myelin-forming cells. However, there is still a lack of appropriate neural models and effe...
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| Main Authors: | , , , , , |
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| Format: | Book |
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MDPI AG,
2023-04-01T00:00:00Z.
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| Call Number: |
A1234.567 |
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| Copy 1 | Available |