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. 2016 Dec 13;17(12):2089. doi: 10.3390/ijms17122089
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