Table 1.
Experimental therapies evaluated for the treatment of MPS VI. hHSCs = human hematopoietic stem cells, hBM-MSCs = human bone marrow-derived mesenchymal stem cells; hDP-MSCs = human dental pulp-derived mesenchymal stem cells; B4GalT7 = β-1,4-galactosyltransferase.
| Therapy | Description | In Vitro Studies | Animal Studies | Clinical Trials | |||||
|---|---|---|---|---|---|---|---|---|---|
| Model | Ref | Model | Delivery Route | Ref | Phase | EudraCT Number | Clinical Trials.gov Identifier | ||
| Gene therapy | ARSB gene transfer mediated by retroviral vectors | feline muscle cells | [92] | cat | intravenous | [93] | - | - | - |
| ARSB gene transfer mediated by adeno-associated viral vectors | - | - | cat | subretinal | [94] | I/II | 2016-002328-10 | NCT03173521 | |
| cat | intravenous | [95,96,97] | |||||||
| mouse | intravenous | [97,98,99] | |||||||
| ARSB gene transfer mediated by lentiviral vectors | human fibroblasts, feline chondrocytes | [100] | rat | injection into the knee joint | [100] | - | - | - | |
| hHSCs, hBM-MSCs, hDP-MSCs | [101] | ||||||||
| Substrate reduction therapy | Odiparcil (substrate analogue for B4GalT7) | human fibroblasts | [102] | mouse | oral | [102,103] | II | 2017-002158-35 | NCT03370653 |
| Anti-inflammatory drugs | Infliximab (monoclonal antibody against human TNF-α) | - | - | rat | intravenous | [104] | - | - | - |
| Pentosan polysulfate | - | - | rat | oral | [105,106] | - | - | - | |
| rat | subcutaneous | [106] | |||||||
| Stop codon read-through | Gentamicin | human fibroblasts | [107,108] | - | - | - | - | - | - |
| PTC124 | human fibroblasts | [107,108] | - | - | - | - | - | - | |