AAV	Adeno-associated virus
Ab	Antibodies
AIR	Adaptative immune response
AOD	Apoptotic oligodendrocytes
AP	Apoptosis
BBB	Blood–brain barrier
Cas9	CRISPR-associated protein 9
CK	Cytokines
CNS	Central Nervous System
CRISPR	Clustered Regularly Interspaced Short Palindromic Repeats
DMDP	2,5-dideoxy-2,5-imino-D-mannitol
DMSO	Dimethyl sulfoxide
DOAJ	Directory of open access journals
DSB	Double-strand break
ECC	Extracellular compartment
EE	Early endosome
ERT	Enzyme Replacement Therapy
FDA	Food and Drug Administration
GAG	Glycosaminoglycans
GCS	Glucosylceramide synthase
GluCer	Glucosylceramide
GM2-AP	GM2-activator protein
GT	Gene therapy
GVHD	Graft-versus-host disease
HDR	Homologous direct repair
Hex	Hexosaminidase
HLA	human leukocyte antigen
HSCT	Hematopoietic Stem Cell Transplantation
ICC	Intracellular compartment
IIR	Innate immune response
iPS	Induced pluripotent stem
LacCer	Lactoceramide
LD	Linear dichroism
LSD	Lysosomal Storage Disorders
M6PR	Mannose-6 phosphate receptor
MDPI	Multidisciplinary Digital Publishing Institute
MPS	Mucopolysaccharidosis
MUG	4-methlyumbelliferyl-N-acetylglucosaminide
MUGS	4-methylumbelliferyl-beta-D-N-acetyl-glucosamine-6-sulfate
NHEJ	Non-homologous end joining
OD	Oligodendrocytes
PC	Pharmacological Chaperones
PERK	PKR-like endoplasmic reticulum kinase
PGRN	Progranulin
PS	Phosphatidylserine
PYR	Pyrimethamine
RER	Rough endoplasmic reticulum
SD	Sandhoff disease
SRT	Substrate Reduction Therapy
TGN	Trans-Golgi network
TSD	Tay–Sachs disease
UCB	Umbilical cordon blood