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. 2021 Feb 27;22(5):2381. doi: 10.3390/ijms22052381
LSD Lysosomal storage disease
FD Fabry disease
Gb3 Globotriaosylceramide
Gal A galactosidase A
ERT Enzyme replacement therapy
CRISPR Clustered regularly interspaced short palindromic repeats
sgRNA Single-guide RNA
Cas9 CRISPR-associated protein
ddPCR Droplet digital PCR
ESCs Embryonic stem cells
iPSCs Induced pluripotent stem cells
CMs Cardiomyocytes
ECs Endothelial cells
HUVECs Human umbilical vein endothelial cells
MVEs Multivesicular endosomes
AVs Autophagic vacuoles
ROS Reactive oxygen species
EMT Endothelial-to-mesenchymal transition