Figure 4
Figure 4. Targeting of albumin locus promotes stable supraphysiological activity of GBA in mouse plasma. (A) GBA activity as determined by enzymatic activity assay in wild-type mice 3 weeks after treatment with either 1.2 × 1012 vg AAV8-GBA donor alone (red circle) or in combination with 1.5 × 1011 vg of each individual AAV8-ZFN (green circle). Untreated wild-type mice (▪) shown as controls. (B) Time course of GBA activity in the mice treated in (A). **P < .01, 2-tailed Mann-Whitney test comparing ZFN + donor group to untreated or donor only groups.

Targeting of albumin locus promotes stable supraphysiological activity of GBA in mouse plasma. (A) GBA activity as determined by enzymatic activity assay in wild-type mice 3 weeks after treatment with either 1.2 × 1012 vg AAV8-GBA donor alone (red circle) or in combination with 1.5 × 1011 vg of each individual AAV8-ZFN (green circle). Untreated wild-type mice (▪) shown as controls. (B) Time course of GBA activity in the mice treated in (A). **P < .01, 2-tailed Mann-Whitney test comparing ZFN + donor group to untreated or donor only groups.

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