| AAV | Adeno-associated virus |
| BMP4 | Bone morphogenic protein 4 |
| CNS | Central nervous system |
| ER | Endoplasmic reticulum |
| ERT | Enzyme replacement therapy |
| FGF | Fibroblast growth factor |
| GAG | Glycosaminoglycan |
| GlcA | Glucuronic acid |
| GlcNAc | N-acetylglucosamine |
| GPC | Glial restricted progenitor cell |
| HS | Heparan sulfate |
| HSPG | Heparan sulfate proteoglycan |
| IdoA | Iduronic acid |
| iPSC | Induced pluripotent stem cell |
| LSD | Lysosomal storage disorder |
| MPS | Mucopolysaccharidosis |
| NSC | Neural stem cell |
| RNAi | Ribonucleic acid interference |
| shRNA | Short hairpin ribonucleic acid |
| siRNA | Small interference ribonucleic acid |
| SRT | Substrate reduction therapy |