FIG. 1.

Biochemical correction of striated muscle after single or dual administration of AAV vectors containing the liver-specific promoter (LSP) or chicken β-actin promoter (CB) regulatory cassettes. Acid α-glucosidase (GAA) activity, glycogen content, and vector genome quantification were analyzed 18 weeks after AAV vector administration. Means±standard error are shown. (A) GAA activity of highly transduced tissues, 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-knockout (KO) mice were negative controls (n=4). (B) GAA activity of skeletal muscle from groups of GAA-KO mice detailed previously. (C) Glycogen content for striated muscles from groups of GAA-KO mice detailed previously. The p values are indicated as follows: *p=0.03; **p<0.03. EDL, extensor digitorum longus; Gastroc., gastrocnemius.