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Biodistribution of rAAV9-hNAGLU vector in MPS IIIB and WT mice following a systemic delivery. MPS IIIB mice and their WT littermates, all males, were treated with an IV injection of 2 × 1014 vg/kg rAAV9-CMV-hNAGLU. Tissues were assayed by qPCR at D5 and 6 weeks pi for vector genome (n = 4/group). Data are means ± SD, expressed as 105 vg/μg genomic DNA. *No detectable vector genome in tissues from nontreated control animal mice (<0.001 × 105 vg/μg gDNA).
