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From: First, do no harm: the role of preclinical animal models in predicting adverse events in gene therapy clinical trials for Duchenne muscular dystrophy and X-Linked myotubular myopathy

First, do no harm: the role of preclinical animal models in predicting adverse events in gene therapy clinical trials for Duchenne muscular dystrophy and X-Linked myotubular myopathy

Figure 5. Photomicrographs of H&E (A, B, E, and F) and acidic ATPase (pH 4.3) (C, D, G, and H) stains of the vastus lateralis muscle of GRMD vs. normal dogs at 3 and 12 months, roughly equivalent to 5 and 20 human years based on comparative longevity studies^[126], showing relative stabilization of histopathologic lesions, with minimal fatty deposition in GRMD. Note also the increase in type I (darker stained) fibers with some grouping in GRMD. Although there is minimal open space suggestive of fat in GRMD at 12 months, the endomysial connective tissue separating individual myofibers is increased (see Fan et al., 2014^[132] for GRMD images at 6 months and Figure 6 for comparison to DMD). Courtesy of Drs. Jane Fan and Yael Shiloh-Malawsky, UNC-Chapel Hill.