Generation of an in vitro model for peripheral neuropathy in Fabry disease using CRISPR-Cas9 in the nociceptive dorsal root ganglion cell line 50B11
Fabry disease is a glycosphingolipid storage disorder that is caused by a genetic deficiency of the lysosomal enzyme alpha-galactosidase A (AGA, EC 3.2.1.22). As a result, the glycolipid substrate, globotriaosylceramide (Gb3) accumulates in various cell types throughout the body producing a multisys...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Elsevier Inc.
2022
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| Subjects: | |
| Online Access: | View Fulltext in Publisher |