Enzyme and GAG levels in transduced and cross-corrected cells
| Lysosomal enzyme levels in transduced MSCs (patients A–C, passages P1–P3) . | ||||
|---|---|---|---|---|
| . | MPS-IH A . | MPS-IH B . | MPS-IH C . | Normal (mean ± SEM) . |
| Intracellular IDUA (μM/g.h) | ||||
| Untransduced | 0 | 0 | 0 | 134.8 ± 37.8 |
| Transduced P1 | 254 | 568 | 918 | (n = 5) |
| Transduced P2 | 280 | 346 | 409 | |
| Transduced P3 | nd | 192 | 605 | |
| Extracellular IDUA (nM/mL.h) | ||||
| Untransduced | 0 | 0 | 0 | 0.90 ± 0.37 |
| Transduced P1 | 8 | 141 | 148 | (n = 4) |
| Transduced P2 | 7 | 133 | 167 | |
| Transduced P3 | 6 | 81 | 197 | |
| Lysosomal enzyme levels in transduced MSCs (patients A–C, passages P1–P3) . | ||||
|---|---|---|---|---|
| . | MPS-IH A . | MPS-IH B . | MPS-IH C . | Normal (mean ± SEM) . |
| Intracellular IDUA (μM/g.h) | ||||
| Untransduced | 0 | 0 | 0 | 134.8 ± 37.8 |
| Transduced P1 | 254 | 568 | 918 | (n = 5) |
| Transduced P2 | 280 | 346 | 409 | |
| Transduced P3 | nd | 192 | 605 | |
| Extracellular IDUA (nM/mL.h) | ||||
| Untransduced | 0 | 0 | 0 | 0.90 ± 0.37 |
| Transduced P1 | 8 | 141 | 148 | (n = 4) |
| Transduced P2 | 7 | 133 | 167 | |
| Transduced P3 | 6 | 81 | 197 | |
nd indicates not done.