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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 1. Schematic of four quadrants in which cellular and humoral immunity interact with AAV capsid and transgene proteins to cause SAEs. From Hamilton et al., 2021[45]. Republished under Creative Commons Attribution License (CC-BY 3.0). AAV: Adeno-associated virus; SAEs: severe adverse events.

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