24(S)-HC	24(S)-hydroxycholesterol
25-HC	25-hydroxycholesterol
3-MA	3-Methyladenine
AD	Alzheimer’s disease
ALS	Amyotrophic lateral sclerosis
AMPAR	AMPA-receptor
AMPK	AMP-activated protein kinase
Apo E	Apolipoprotein E
APP	Amyloid precursor protein
Cas	CRISPR-associated gene
CBZ	Carbamazepine
CD	Cyclodextrin
CNS	Central nervous system
CoQ10	Coenzyme Q10
cPCs	Cerebellar Purkinje cells
CRISPR	Clustered regularly interspaced short palindromic repeats
ER	Endoplasmic reticulum
GABAAR	GABAA receptor
GD	Gaucher’s Disease
GFAP	Glial fibrillary acidic protein
GlyR	Glycine receptor
HAT	Histone acetyl transferase
HDACi	Histone deacetylase inhibitor
hESC	Human embryonic stem cell
Hex A	Hexosaminidase A
HLC	Hepatocyte-like cell
hSKIN-MASCS	Human skin multipotent adult stem cells
HP-α-CD	Hydroxypropyl-alpha-cyclodextrin
HP-β-CD	Hydroxypropyl-beta-cyclodextrin
HP-γ-CD	Hydroxypropyl-gamma-cyclodextrin
HSEM	Horizontal saccadic eye movement
iPSC	Induced pluripotent stem cell
KD	Knock down
LC3B	Microtubule-associated protein a light chain 3B
LE/LY	Late endosome/lysosome
LSD	Lysosomal storage disorder
M-β-CD	Methyl-beta-cyclodextrin
NDCs	Neuronal differentiated cells
NPC	Neural progenitor cell
NPC1	Niemann-Pick type C1
NPC2	Niemann-Pick type C2
NSS	Neurological Severity Scores
OS	Oxidative stress
PC	Pharmacological chaperone
PD	Parkinson’s disease
PMA	Phorbol 12-myristate 13-acetate
PSCs	Pluripotent stem cells
Rap	Rapamycin
RIP1/3	Receptor interacting protein kinase 1/3
ROS	Reactive oxygen species
S1P	Sphingosine-1-phosphate
SAHA	Suberanilohydroxamic acid
SOD	Superoxide-Dismutase
SphK	Sphingosine kinase
SRT	Substrate reduction therapy
TALEN	Transcription activator-like effector nuclease
Tre	Trehalose
VEGF	Vascular endothelial growth factor
Ver	Verapamil
VSGP	Vertical supranuclear gaze palsy