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. Author manuscript; available in PMC: 2011 Sep 28.

Published in final edited form as: Nat Med. 2009 Sep 13;15(10):1215–1218. doi: 10.1038/nm.2025

Intravenous delivery of epitope-modified virus improves neuropathology in MPS VII mice. (a) Representative sections of cerebral cortex, hippocampus, striatum and cerebellum of MPS VII mice harvested after tail vein injection with either AAV-WT.βluc (left panels) or AAV-PFG.βluc (right panels) expressing β-glucuronidase. Yellow asterisk, denotes region magnified in inset (lower right, all panels) for better visualization of storage vacuoles. Arrows in inset point to vacuoles. Scale bar = 50 μm for all panels. (b) Quantitation of vacuolar storage in various brain regions. Tail vein injection of AAV-PFG.βluc but not AAV-WT.βluc significantly reduced lysosomal storage vacuoles in hippocampus, cortex and striatum (*p<0.001, Tukeys post hoc). (c) Binding of AAV-PFG to cerebral vasculature requires chondroitin sulfate. Purified brain microvessels from heterozygous or MPS VII mice were incubated with the reagents indicated, and bound viral particles quantified. Data presented as mean ± SEM. (*,p<0.001, Dunnett’s post hoc). (d) Binding of AAV-PFG to purified brain vasculature from MPS VII mice in the presence or absence of chondroitin sulfate. Data presented as mean ± SEM. *<0.01, student’s t-test.

Intravenous delivery of epitope-modified virus improves neuropathology in MPS VII mice. (a) Representative sections of cerebral cortex, hippocampus, striatum and cerebellum of MPS VII mice harvested after tail vein injection with either AAV-WT.βluc (left panels) or AAV-PFG.βluc (right panels) expressing β-glucuronidase. Yellow asterisk, denotes region magnified in inset (lower right, all panels) for better visualization of storage vacuoles. Arrows in inset point to vacuoles. Scale bar = 50 μm for all panels. (b) Quantitation of vacuolar storage in various brain regions. Tail vein injection of AAV-PFG.βluc but not AAV-WT.βluc significantly reduced lysosomal storage vacuoles in hippocampus, cortex and striatum (*p<0.001, Tukeys post hoc). (c) Binding of AAV-PFG to cerebral vasculature requires chondroitin sulfate. Purified brain microvessels from heterozygous or MPS VII mice were incubated with the reagents indicated, and bound viral particles quantified. Data presented as mean ± SEM. (*,p<0.001, Dunnett’s post hoc). (d) Binding of AAV-PFG to purified brain vasculature from MPS VII mice in the presence or absence of chondroitin sulfate. Data presented as mean ± SEM. *<0.01, student’s t-test.