Extended Data Fig. 9: Brain-specific Treg expansion following PHP.CamKII-IL-2 treatment.
The CamKII promoter restricts gene expression (as assessed using GFP scoring) to neurons (NeuN positive) in adult mouse brain. Off-target expression was not detected when slices were counter-stained for GFAP (astrocytes). Left panel, hippocampus; right panel, cortex. Scale bar, 20 µm. Data are representative images seen in 1 slice from each of 4 independent mice receiving a PHP. CamKII-GFP (control) vector. b, Quantification of GFP colocalization with NeuN and GFAP in PHP.CamKII-GFP-treated mice. c, Levels of IL-2 were measured from tissue samples obtained from wildtype mice administered with 1×109, 1×1010 or 1×1011 total vector genomes of PHP.CamKII-GFP (control) or PHP. CamKII-IL-2 (n = 7,7,5,8,8,8). d, Wildtype mice were administered, intravenously, 1×109, 1×1010 or 1×1011 vector genomes (total dose) of PHP.CamKII-GFP or PHP.CamKII-IL-2 and assessed for the number of conventional T cells (left) and Treg cells (right), 14 days after treatment (n = 5 group) in the perfused brain or e spleen. f, Treg cells as a percentage of CD4 T cells in the brain and g spleen. Statistical analysis was performed using two-way ANOVA with Sidak correction.