Figure 2.

Myostatin Expression in AAV-cDM1-Treated GRMD Dogs

(A) Mstn levels were measured in blood samples isolated from either WT or GRMD dogs injected via loco-regional route (LR) or systemic i.v. (IV) routes with a high (1e14 vg/kg) or low (2e13 vg/kg) dose of the AAV vector coding a sequence-optimized cMD1. ∗p < 0.05, ∗∗p < 0.01, ∗∗∗p < 0.001, Student’s t test, compared to untreated GRMD dogs. (B) 3- to 4-month-old GRMD dogs (n = 4) were loco-regionally treated with 1e13 vg/kg, and Mstn, Fstn, and Acvr2b were analyzed at mRNA levels in muscle biopsies, 2 and 4 months after treatment. For each mRNA, results are expressed as the ratio, injected leg/noninjected leg. ∗p < 0.05, ∗∗p < 0.01, ∗∗∗p < 0.001, Student’s t test. (C) Mstn (black), Fstn (gray), AcvR2b (ActivinR2b; white), and cMD1 (diamond) mRNA levels were analyzed in untreated or low- or high-dose i.v.-treated GRMD animals at the age of 10 months. The transcript amounts were normalized using Rpl32 dog ribosomal RNA. Treated dogs were AAV injected at the age of 2–2.5 months. The number of samples for each point is detailed in Table S1. Error bars are SEM. A one-way ANOVA, followed by Newman-Keuls test was performed.