FIG. 3.

Effective CNS and widespread somatic transduction in NHP after a systemic rAAV9-hNAGLU gene delivery. (a) Tissue NAGLU activity. NAGLU activity is expressed as units/mg protein, 1 unit=release of 1 nmol 4MU/hr. Group 1: treated with 1E13 vg/kg; Group 2: treated with 2E13 vg/kg. (b) Immunofluorescence detection for rNAGLU using a polyclonal Ab against hNAGLU. Red fluorescence indicates NAGLU-positive cells/signals. Yellow arrows indicate neurons; blue arrows, blood vessels; yellow arrowheads, NAGLU-overexpressing hepatocytes; green arrowheads, NAGLU-overexpressing cardiomyocytes. *Subject with high preexisting anti-AAV9 antibodies. CNS, central nervous system; HRT, heart; LIV, liver; TH, thalamus (brain).