FIG. 9.

Liver transduction and immune responses after single or dual vector administration. (A) Real-time PCR quantification of vector genomes in the liver from groups of GAA-KO mice after administration of AAV-LSPhGAA (2×10¹⁰ VP/mouse; n=5), both AAV-LSPhGAA (2×10¹⁰ VP) and AAV-CBhGAA (1×10¹¹ VP; n=4), AAV2/8-LSPhGAA (1×10¹¹ VP/mouse; n=4), both AAV2/8-LSPhGAA and AAV2/9-CBhGAA (n=5), or AAV2/9-CBhGAA (1×10¹¹ VP; n=3). Vectors were injected intravenously into mice at 3 months of age. Mock-treated GAA-KO mice were negative controls (n=4). (B) RT-PCR quantification of IFN-γ in the liver of groups of GAA-KO mice detailed previously. The p values are as follows: *p=0.03; **p<0.03. (C) Groups of GAA-KO mice were injected intravenously with AAV-LSPhGAA and AAV-CBhGAA as indicated before an immune challenge with rhGAA and adjuvant as shown (Fig. 4A; n=5 per group). (D) Periodic acid–Schiff staining of glycogen in the quadriceps (original magnification, ×400).