fig3

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 3. T2-weighted magnetic resonance images of pelvic limb muscles 8 weeks after AAV9-CMV-mini-dystrophin vector intravenous injection of two GRMD dogs (A-D, E-H) at 4 days of age. Images were segmented and color-coded to outline individual muscles. Signal-intense lesions are particularly pronounced in the vastus heads of the quadriceps and adductor muscles. These changes persisted with fat saturation, suggesting that they most likely represent fluid due to inflammation or edema. From Kornegay et al., 2010[81]. Republished under Creative Commons Attribution License (CC-BY 3.0).

Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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