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. 2015 Aug 5;26(11):743–750. doi: 10.1089/hum.2015.033

Figure 1.

Figure 1.

Biochemical correction by muscle-targeted gene therapy with clenbuterol and anti-CD4 monoclonal antibody (mAb). (A) Acid α-glucosidase (GAA) activity and (B) glycogen content were analyzed to demonstrate biochemical correction of Pompe disease by combinations of clenbuterol (Clen), anti-CD4 mAb, and AAV2/8-MHCK7hGAApA (MHCK7) in GAA-knockout (GAA-KO) mice. Group size was as follows: no-treatment control (n = 6), clenbuterol only (n = 6), MHCK7 only (n = 4), MHCK7 with anti-CD4 mAb (n = 7), triple therapy with MHCK7, clenbuterol, and anti-CD4 mAb (n = 7). Statistical analysis was performed by two-way analysis of variance with Bonferroni's comparison test, using the triple-therapy group as the reference (*p < 0.05, **p < 0.01, ***p < 0.001). Shown are means and standard deviation. Quad, quadriceps; Gastroc, gastrocnemius; Diaphr, diaphragm.