Report Summary

Toxicology Study of rAAV-1-CB-hAAT Summary of Findings

Study Summary

Overall Study Design 
The objective of this study design was to determine the potential toxicity of adeno-associated virus expressing the alpha-one 
antitrypsin gene rAAV1-CB--hAAT following a single injection into C57B16 mice The overall study was divided into two 
separate parts the primary toxicology study and the biodistribution study see separate report for the biodistribution study 
The primary toxicology study consisted of 4 experimental arms The mice were injected intramuscularly with either PBS 
vehicle control group or rAAV1-CB-hAAT at a total dose of 1 x 10e10 vg  4 x 10e11 vgkg 3 x 10e11 vg 12 x 10e13 
vgkg or 1 x 10e13 vg  4 x 10e14 vgkg Each arm consisted of 8 mice which included 2 males and 2 females at each 
sacrifice time point of either 21 or 90 days 
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