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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 8. Schematic (A) of two German shorthaired pointer muscular dystrophy dogs (Ned and Grinch), in which the opposite cranial tibialis muscles were injected with either µ-dystrophin or µ-utrophin, showing the numbers of blood mononuclear cells reacting over time when cultured with peptides spanning each protein (B), together with the level of µ-dystrophin or µ-utrophin gene expression (C) and resulting histopathologic lesions (D). Note the marked inflammatory cell response in the AAV-µ-dystrophin treated limb (left) contrasted with the absence of inflammation in the AAV-µ-utrophin limb (right). From Song et al., 2019 (Stedman HH, corresponding author)^[118].

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Committee on Publication Ethics

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Committee on Publication Ethics

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