Table 3.
Correction of Metabolomic Abnormalities in MPS IIIB Mice by an i.v. rAAV9-hNAGLU Gene Delivery
| Groups | Age (Months) | Number of Metabolites Altered (p ≤ 0.05) |
||
|---|---|---|---|---|
| Decrease | Increase | Total | ||
| IIIB/WT | 2 | 17 | 1 | 18 |
| AAV9/WT | 2 | 2a/6b | 0 | 8 |
| IIIB/WT | 7 | 225 | 6 | 231 |
| AAV9/WT | 7 | 1b | 30a/27b | 58 |
At 2 and 7 months of age, serum samples were assayed by global metabolomic profiling for 361 metabolites, using mass spectrometry. IIIB, non-treated MPS IIIB mice; WT, wild-type mice; AAV9, MPS IIIB mice treated with an i.v. injection of rAAV9-CMV-hNAGLU vector (5e12 vg/kg) at the age of 1 month.
a
Number of metabolites unchanged after vector treatment.
b
Number of metabolites altered after vector treatment.