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 |