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 |