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Report Summary

Single Dose 6-Month Toxicity Study of Adeno-Associated Virus-Cystic Fibrosis (AAV-CFTR) Gene Vector in the Rhesus Monkey


Study Summary

Overall Study Design 
The objective of this study was to determine the potential toxicity of adeno-associated virus-cystic fibrosis AAV-CFTR gene 
vectors following a single intrabronchial administration to rhesus monkeys The study consisted of 4 groups of monkeys 
Monkeys received via a single intrabronchial dose either 1 ml of Ringers balanced salt solution vehicle control group or 1 
ml of Ringers balanced salt solution containing AAV-CFTR vector particles at a total dose of 5 x 10e8 1 x 10e10 or 1 x 
10e11 The animals were sacrificed at 102190 or 180 days post-dose 




 
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