fig7

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 7. Histogram of comparative GFP transgene RNA expression of WT vs. capsid and genomic optimized (OptX) AAVrh74 vectors injected IV into C57BL mice. Bar graphs show average of WT- and OptX-AAVrh74 △Ct across organs. Statistical significance was determined between WT and OptX (see error bars and P values). From Shoti et al., 2023[159]. Republished under Creative Commons Attribution License (CC-BY 3.0). WT: Wild-type.

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