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Profound Metabolomic Impairments in MPS IIIB Mice and Metabolic Responses to a Systemic rAAV9-CMV-hNAGLU Gene Delivery
Mouse serum samples were analyzed by global metabolomic profiling using mass spectrometry at the age of 2 or 7 months (n = 6/group). (A) Principal-component analyses and (B) heatmap analysis are shown. WT, wild-type mice; IIIB, MPS IIIB mice; IIIB + AAV, MPS IIIB mice treated at age 1 month with an i.v. injection of 5 × 1012vg/kg rAAV9-CMV-hNAGLU; red arrows, outlier.
