Figure 1.

GALGT2 expression and adeno-associated viral (AAV) vector biodistribution after vascular delivery of rAAVrh74.MCK.GALGT2. (A) GALGT2 overexpression is shown by Wisteria floribunda agglutinin (WFA) staining in rAAVrh74.MCK.GALGT2-treated gastrocnemius muscle in seronegative (–) or seropositive (+) macaques, of which one cohort was additionally treated with prednisone (Pred). Control represents time-matched WFA staining of an untreated muscle. Scale bar: 50 μm. (B) The percentage of myofibers overexpressing CT glycan was quantified for all muscle segments taken throughout both heads of the gastrocnemius muscle. Data are mean ± standard deviation (SD) for n = 2 (group 5) and n = 3 (groups 1–4) macaques per group. (C) AAV vector genomes were measured by quantitative polymerase chain reaction (qPCR) and normalized to total genomic DNA. Data are mean ± standard error of the mean (SEM) for seven muscle samples analyzed for two to three macaques per group. (D) Relative change in transgenic human GALGT2 mRNA expression, compared to endogenous rhesus macaque GALGT2 mRNA expression, was determined using quantitative reverse transcription PCR (qRT-PCR) with 18S ribosomal RNA as an internal reference. Data are mean ± SEM for seven muscles analyzed for two to three macaques per group. *p < 0.05, **p < 0.01 using one-way analysis of variance (ANOVA) in (B–D).