Elevated Dkk1 Mediates Downregulation of the Canonical Wnt Pathway and Lysosomal Loss in an iPSC Model of Neuronopathic Gaucher Disease
Gaucher Disease (GD), which is the most common lysosomal storage disorder, is caused by bi-allelic mutations in <i>GBA1</i>—a gene that encodes the lysosomal hydrolase β-glucocerebrosidase (GCase). The neuronopathic forms of GD (nGD) are characterized by severe neurological abnormalities...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2020-12-01
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| Series: | Biomolecules |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2218-273X/10/12/1630 |