CRISPR | Clustered regularly interspaced short palindromic repeats |
sgRNA | single-guide RNA |
Cas9 | CRISPR-associated protein |
DGJ | 1-deoxygalactonojirimycin |
ERT | enzyme replacement therapy |
FD | Fabry disease |
Gb3 | globotriaosylceramide |
α-Gal A | α-galactosidase A |
KO | knockout |
HEK-293T | human embryonic kidney 293T cells |
HSP | heat shock protein |
LSD | lysosomal storage disease |
PC | pharmacological chaperone |
rhα-GLA | recombinant human α Gal A |