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. 2020 Aug 10;21(16):5732. doi: 10.3390/ijms21165732
AGO2 Argonaute 2
ApoB Apolipoprotein B
ASOs Antisense oligonucleotides
BBB Blood-brain barrier
C6-Cer C6-ceramide
CML Chronic myeloid leukemia
CNS Central nervous system
CSCs Cancer stem cells
CSF Cerebrospinal fluid
CTX Chlorotoxin
DAGs Diacylglycerols
DSPC 1,2-distearoyl-sn-glycero-3-phosphocholine
dsRNA Double-stranded RNA
DXR Doxorubicin
EBOV Ebola virus
ERT Enzyme replacement therapy
FD Fabry disease
GAGs Glycosaminoglycans
GD Gaucher disease
GBM Glioblastoma
gSRT Genetic substrate reduction therapy
hATTR Hereditary TTR-mediated amyloidosis
HBV Hepatitis B virus
HF Hemorrhagic fever
HIV Human immunodeficiency virus
HMEC-1 Angiogenic endothelial cells
HS Heparan sulphate
ICV Intracerebroventricular
iPSCs Induced pluripotent stem cells
IT Intrathecal
IV Intravenous
KSP Kinesin spindle protein
LNPs Lipid nanoparticles
LDL Low-density lipoprotein
LSDs Lysosomal storage disorders
miRNA microRNA
MLD Metachromatic Leukodystrophy
MPSs Mucopolysaccharidoses
mRNA messenger RNA
NCAM Neural cell adhesion molecule
NCLs Neuronal Ceroid Lipofuscinoses
nACh Nicotinic acetylcholine
nAChR Nicotinic acetylcholine receptor
n-SCC Non-stem cancer cells
PD Parkinson’s disease
PLK1 Polo-like kinase 1
PEG Polyethylene glycol
PEI Polyethylenimine
PC3 Prostate cancer cells
PKR Protein kinase receptor
P75NTR Neurotrophin receptor 75
RDP Rabies virus derived peptide
RVG Rabies virus glycoprotein
RVG-9r Rabies virus peptide derivative
RME Receptor-mediated endocytosis
rhTPP1 Recombinant human tripeptidyl peptidase 1
RES Reticuloendothelial system
RNAi RNA interference
RISC RNA-induced silencing complex
shRNA Short hairpin RNA
siRNAs small interfering RNAs
SNALP Stable nucleic acid lipid particle
SRT Substrate reduction therapy
TLRs Toll-like receptors
Trf Transferrin
TrfR Transferrin receptor
TTR Transthyretin
T1/2 Elimination/circulation half-time
U.S. FDA United States Food and drug administration
VLDL Very-low-density lipoprotein
ZEBOV Zaire species of EBOV
3’-UTR 3’ untranslated region