| 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 |