Report Summary

Toxicology and Biodistribution of rAAV1-CB-hAAT Administered in Male and Female Rhesus Macaques by Isolated Limb Infusion

Study Summary

Overall Study Design 
The objective of this study was to assess the safety vector biodistribution and persistence of transgene expression in 
rhesus macaques administered rAAV1-CB-hAAT by isolated limb infusion ILI This route has been show in previous animal 
studies with other AAV vectors to provide more uniform transduction of muscle cells than achieved using intramuscular IM 
injection and reduced immune responses to the vector 
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