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. 2011 Mar 8;19(6):1025–1033. doi: 10.1038/mt.2011.34

Figure 2.

Figure 2

Distribution of recombinant adeno-associated virus 9 (rAAV9)-mediated rNAGLU expression in the central nervous system (CNS) and somatic tissues of mucopolysaccharidosis (MPS) IIIB mice after an intravenous (IV) rAAV9 vector injection. Tissues from rAAV9-CMV-hNAGLU-treated MPS IIIB mice (6 month postinjection) were assayed for hNAGLU by immunofluorescence. Red fluorescence: hNAGLU-positive cells. Blue fluorescence (4′,6-diamidino-2-phenylindole): nuclei. (a) CNS; A. mouse treated with 5 × 1012 vg/kg vector; B. mouse treated with 2 × 1013 vg/kg vector; C–H. mouse treated with 2 × 1013 vg/kg vector following mannitol pretreatment; I, nontreated MPS IIIB mouse. BS, brain stem; CB, cerebellum; CP, choroid plexus; CTX, cerebral cortex; SC, spinal cord; TH, thalamus. Yellow arrowheads: hNAGLU-positive brain cells; red arrowheads: hNAGLU-positive endothelial cells. Bar = 100 µm. (b) Somatic tissues. Red fluorescence: AAV9, MPS IIIB mouse treated with 2 × 1013 vg/kg vector; NT, nontreated MPS IIIB mouse. A. Hrt, heart; Liv, liver; Mus, skeletal muscle. (quadracep). Yellow arrowheads: hNAGLU-positive cells (hepatocytes, myocytes, cardiomyocytes). B. INT, intestine; ME, muscularis externa; Sm, submucosa; +TB, toluidine blue staining. Yellow arrowheads: rNAGLU-positive neurons of myenteric plexus and submucosal plexus. Red arrowheads: peritoneal surface of intestine. Black arrows: neurons of myenteric plexus and submucosal plexus. Bar = 40 µm. NAGLU, α-N-acetylglucosaminidase.